US20230142867A1

US20230142867A1 - Aav native-neuro platform and use for neuronal disease gene therapy

Info

Publication number: US20230142867A1
Application number: US17/920,483
Authority: US
Inventors: Senthil Ramu
Original assignee: Aav Gene Therapeutics Inc
Current assignee: Aav Gene Therapeutics Inc
Priority date: 2020-04-23
Filing date: 2021-04-23
Publication date: 2023-05-11
Also published as: US20230171886A1; CN115700016A; KR20210131149A; WO2021215879A1

Abstract

The invention described herein provides methods and compositions for using recombinant AAV-based viral vectors to express any gene-of-interest (GOI) under its native promoter and a proprietary heterologous enhancer, preferably expressing a GOI defective in a neuronal disease or disorder in order to treat the neuronal disease or disorder.

Description

RELATED APPLICATIONS

This application claims priority to U.S. Provisional Application No. 63/014,283, filed on Apr. 23, 2020, the entire contents of which is expressly incorporated herein by reference.

BACKGROUND OF THE INVENTION

Gene-based neurotherapeutics holds great therapeutic potential. Clinical efficacy has already been obtained for central nervous system (CNS) diseases and eye disease through Adeno Associated Virus (AAV) gene therapy. The general lack of current effective neurotherapies for many devastating neurologic diseases portends great future demands for AAV gene-based neurotherapy.
Efficient and specific delivery to a target organ, such as the brain, is a rate limiting step in many protein-based therapeutics. There are multiple barriers to efficient direct protein delivery to the CNS.
One of the great challenges lies in the blood-brain barrier (BBB). Unlike the vasculature in other organ systems, the brain endothelium forms intercellular tight junctions (TJs) that limit greatly the paracellular access of blood-borne components to the tissue parenchyma. As a consequence of this “physical barrier” generated by TJs, gene transfer vehicles attempting entry into the CNS are forced to go through the transcellular route. There, however, specialized lumen facing efflux transporters, which include both the ABC family of transporters and multidrug resistance-related proteins, contribute to the formation of the “transport barrier” that prevents xenobiotic substances from gaining access to the neuronal environment. Another obstacle that partially prevents endothelial crossing of gene-therapy vehicles is the trancytotic/endosomal sorting, which can lead to degradation. Although the endothelium is the greatest obstacle in restricting access to the CNS, the presence of a complex extracellular space, molecular composition and geometry also significantly impede diffusion of viral-based gene delivery systems. That is, even if a therapy can be designed to target one of these receptors and traverse the BMVEC cytoplasm to be released at the basal cell membrane, only the first of many guardian mechanisms at the BBB has been overcome. If neurons are the target cell type, once the BBB endothelium has been passed, the basal lamina, pericytes, microglial ramifications, and the glial limitans comprised of astrocytic endfeet still have to be navigated before surpassing all of the checkpoints at the BBB and encountering a neuron.
As a result, intravascular administration of most viral vectors with the exception of few AAV serotypes has proven unsuccessful in the treatment of neurological diseases, potentially because these gene delivery platforms cannot negotiate their way across the BBB to access their target cells.
Another challenge lies in the promoters used in the gene therapy vectors. With the discovery of new viral vectors, mainly AAV capsids, with remarkable CNS tropism after vascular, CSF administration, or direct intracranial injection, the field of AAV mediated CNS gene therapy is accelerating rapidly towards clinical trials in a number of neurological diseases, and the expedient approach has been to use specific promoters with a proven track record of mediating long-term gene expression given the serious nature of many of these diseases, many of which are pediatric.
The majority of the pre-clinical studies have used strong ubiquitous promoters to provide a broad overview of transgene expression in different tissues and cell types throughout the body after intravascular infusions, or CNS cell types after intracranial delivery.
The CAG/CBA/CB promoter is generally viewed as a ubiquitous promoter as it works in numerous tissues and cell types. However, the assumption that it is highly active in every cell type may not be entirely warranted in the CNS. Studies have been conducted using AAV vectors with the same capsid but carrying different CNS cell specific promoters or the CBA promoter. The apparent discrepancy in CNS transduction profiles of these different AAV vectors suggests that the CBA promoter may not mediate detectable transgene expression in all cell types in the brain. Additional experiments have been conducted using AAV vectors with numerous tropism carrying the cytomegalovirus (CMV), CBA, Rous sarcoma virus (RSV), or human GUSB promoters to drive transgene expression, in the context of intra-parenchymal delivery to different brain structures.
In clinical studies, the majority of AAV vectors for treating neurological diseases have used the CMV immediate early promoter followed by a chimeric intron composed of a CMV splice donor and a human globin splice acceptor, or a version of the CAG/CBA promoter (supra). Two other clinical trials have tested AAV vectors carrying mammalian promoters such as the mouse phosphoglycerate kinase (PGK) promoter, or the rat neuron-specific enolase (NSE) promoter. The choice of promoters is based on the availability of extensive data from pre-clinical studies in different animal models showing that AAV-mediated transgene expression under these promoters is stable and long lasting in the mammalian brain. The stability of AAV gene expression in the human brain has been evaluated in clinical trials using an AAV2 vector encoding aromatic amino acid decarboxylase (AADC) injected into the putamen of Parkinson's disease patients, or children afflicted with AADC deficiency.
An indisputable fact about these AAV-CMV or AAV-CAG/CBA vectors is their ability to drive strong gene expression in many neurons in the CNS of multiple species. The disparate CNS transduction results between cellular promoters and strong non-mammalian promoters raises the possibility that the current generation of AAV vectors express transgenes at exceptionally high levels in a small percentage of all cells that harbor transcriptionally ready vector genomes in the nucleus. A recent report documented considerable toxicity in non-human primates (NHPs) receiving intraparenchymal injections of an AAV2 vector encoding human acid sphingomyelinase. Another safety study on intracranial delivery of an AAVrh10 vector encoding human tripeptidyl peptidase (TPP-I) in NHPs reported white matter edema and inflammation. The interpretation of these results is complicated, but a possible interpretation of the results is that these inflammatory responses are secondary to protein expression overload and non-specific expression in AAV transduced neurons.
The potential for toxicity due to excessive transgene expression is particularly acute in neurological diseases where the expressed proteins are not secreted, and thus the need for high level expression is even less. In one particular case, the development of an AAV gene therapy approach for Rett syndrome represents a significant challenge to the prevalent approach of simply using strong promoters. This disease is caused by mutations in the MeCP2 gene on the X-chromosome. Interestingly, duplication of this gene also leads to neurodevelopmental delays, suggesting that expression of this gene in the CNS need to be tightly controlled, which is a challenge to current AAV gene therapy.

SUMMARY OF THE INVENTION

The invention described herein provides a gene replacement therapy platform to replace mutated genes by AAV Gene Therapy.
According to the invention described herein, about 40 exemplary central nervous system (CNS) disease drug protypes are packaged in recombinant AAV vectors for intravenous administration to a patient, in order to express functional genes in native neuronal cells. The technology is designed for intravenous dosing of AAV to express genes precisely in intended cell types by using endogenous/native promoter fused with viral/heterologous enhancer.
In an exemplary embodiment, the gene cassette in the AAV vector of the invention begins with AAV inverted terminal repeat (ITR), followed by proprietary heterologous enhancer, followed by a native promoter of a gene of interest (GOI), 5′UTR, proprietary codon optimized gene of interest (GOI), 3′UTR, and ends in another AAV inverted terminal repeat (ITR).
Thus one aspect of the invention provides a recombinant adeno-associated viral (rAAV) vector, comprising: (1) a polynucleotide comprising a gene of interest (GOI), under the transcriptional control of the native/endogenous promoter of said GOI; (2) a heterologous enhancer that enhances transcription of the GOI from the native/endogenous promoter; and, (3) a pair of inverted terminal repeat (ITR) flanking the polynucleotide.
In certain embodiments, the GOI is a neuronal gene, or is a gene expressed in neuronal tissues (CNS and/or PNS).
In certain embodiments, the GOI is defective (e.g., loss-of-function) in a neuronal disease or condition.
In certain embodiments, the neuronal disease or condition is a CNS disease or condition.
In certain embodiments, the CNS disease or condition is: Ceroid-Lipofuscinosis, Fucosidosis, Giant Axonal Neuropathy, Globoid cell leukodystrophy, Mucolipidiosis Type IV, Neuronal Ceroid Lipofusinoses, Niemann-Pick Disease, Sandhoff Disease, Sanfilippo syndrome, Tay-Sachs Disease, Hunter Syndrome, Sialidosis, Multiple Sulfatase Deficiency, Childhood Epilepsy, CMT Peripheral Neuropathy, Neuronal Ceroid Lipofusinoses, Aspartylglycosaminuria, Mucopolysaccharidoses, Parkinsons Disease/Symptoms, GM1-Gangliosidosis, Charcot-Marie-Tooth Type 1A, Retts Syndrome, Dannon Disease, Mucopolysaccharidoses (due to GNS, HGSNAT, NAGLU, or ARSB), Sly Syndrome, Biotin basal ganglia disease, Pelizaeus-Merzbacher disease, Neuronal Ceroid Lipofusinoses, Canavan Disease, Beta-Mannosidosis, Wolman Disease, Cystinosis, Alzheimer's Disease/Symptom, Salla Disease, Jansky-Bielschowsky disease, Neuronal ceroid lipofuscinoses, or GM2 gangliosidosis.
In certain embodiments, the GOI is: CLN3, FUCA1, GAN, GALC, MCOLN1, PPT1, SMPD1, HEXB, SGSH, HEXA, IDS, NEU1, SUMF1, GAT1/SLCA1, FIG. 4 , CLN5, AGA, IDUA, GDNF, GLB1, PMP22/MFN2, MECP2, LAMP2, NAGLU, ARSB, GUSB, SLC19A3, PLP1, TPP1/CLN2, ACY2/ASPA, MANBA, LIPA, CTNS, GNS, HGSNAT, NEUROD1, SLC17A5, CLN6, CLN8, or GM2A.
In certain embodiments, the rAAV has a tropism for neuronal cells, or for a cell in neuronal tissue.
In certain embodiments, the rAAV is AAV9, AAV2.5, rAAVrh.39, rAAVrh.43, rAAV7, rAAVrh.8 or rAAVrh.10, preferably AAV9.
In certain embodiments, the rAAV comprises AAV9, AAV2.5, rAAVrh.39, rAAVrh.43, rAAV7, rAAVrh.8 and rAAVrh.10 capsids, preferably AAV9 capsids.
In certain embodiments, the polynucleotide further comprises a 5′-UTR coding region, and a 3′-UTR coding region.
In certain embodiments, the heterologous enhancer is a viral enhancer.
In certain embodiments, the viral enhancer is CMV enhancer.
In certain embodiments, the viral enhancer is SV-40 enhancer.
In certain embodiments, the heterologous enhancer comprises a polynucleotide of SEQ ID NO: 1.
Another aspect of the invention provides a pharmaceutical composition comprising any one of the rAAV vector of the invention.
In certain embodiments, the pharmaceutical composition is formulated for intravenous administration (e.g., to the blood) or intrathecal administration (e.g., to spinal fluid).
Another aspect of the invention provides a cell infected with any one of the rAAV vector of the invention.
Another aspect of the invention provides a recombinant AAV virus comprising any one of the rAAV vector of the invention, wherein the serotype or pseudotype of the recombinant AAV virus is for preferential infection of a neuronal cell or a cell in neuronal tissue.
Another aspect of the invention provides a method of treating a (genetic) disease or disorder in a subject, the method comprising administering the recombinant AAV virus of the invention into the subject.
In certain embodiments, the genetic disease or disorder is a neuronal disease or disorder, and/or wherein the recombinant AAV virus preferentially infects a neuronal cell or a cell in neuronal tissue of the subject, thereby treating the neuronal disease or disorder.
In certain embodiments, the rAAV virus is administered to the subject intravenously or intrathecally.
Another aspect of the invention provides a method of producing any one of the rAAV vector of the invention, the method comprising introducing any one of the rAAV vector of the invention into a packaging cell line that constitutively or inducibly provides rep/cap proteins in trans.
In certain embodiments, the rep/cap proteins comprise one or more AAV9 capsid proteins.
It should be understood that any one embodiment of any one of the aspects of the invention above, including specific embodiments described only in one section of the application, can be combined with one or more additional embodiments.

BRIEF DESCRIPTION OF THE DRAWINGS

FIG. 1 is a schematic drawing (not to scale) showing a generic structure of the subject rAAV vector, as well as an intended mode of action for the vector. The Gene of Interest (GOI) is put under the control of its native promoter. Transcription from the native promoter is further enhanced by the presence of a powerful enhancer, such as a viral enhancer like CMV enhancer, to facilitate the expression of the GOI in the native cells such GOI is normally expressed. The entire recombinant sequence is flanked by two AAV ITR sequences, such as AAV9 ITR sequences for the preferred neuronal cell tropism.

FIG. 2 is a gel image confirming the construction of rAAV viral vectors comprising a number of representative GOI's that are defective in neuronal diseases/disorders. Also shown is a rAAV vector encoding a GFP reporter gene, which can be switched out by any of the GOI's.

FIG. 3 is a gel image confirming the construction of additional rAAV viral vectors comprising a number of representative GOI' s that are defective in neuronal diseases/disorders.

DETAILED DESCRIPTION OF THE INVENTION

1. Overview

The invention described herein provides methods and compositions for using recombinant viral vectors, such as AAV-based viral vectors or lentiviral vectors to express any gene-of-interest (GOI) under the control of its native promoter so that the GOI is preferentially and exclusively expressed in the cells the GOI is intended to be expressed. The GOI is further under the transcriptional control of a powerful heterologous enhancer, such as a viral enhancer like CMV enhancer, which further boosts the transcription and expression of the GOI. Therefore, after a single intravenous treatment, the protein product of interest is expressed at high levels only in the endogenous/native cell types, thereby improving therapeutic safety and efficacy.
Thus in one aspect, the invention described herein provides a recombinant adeno-associated viral (rAAV) vector, comprising: (1) a polynucleotide comprising a gene of interest (GOI), under the control of the native/endogenous promoter of said GOI; (2) a heterologous enhancer that enhances transcription of the GOI from the native/endogenous promoter; and, (3) a pair of inverted terminal repeat (ITR) flanking the polynucleotide.
In certain embodiments, the GOI is a neuronal gene.
In certain embodiments, the neuronal gene is defective (e.g., loss-of-function) in a neuronal disease or condition.
In certain embodiments, the neuronal disease or condition is a CNS disease or condition.
In certain embodiments, the CNS disease or condition is: Ceroid-Lipofuscinosis, Fucosidosis, Giant Axonal Neuropathy, Globoid cell leukodystrophy, Mucolipidiosis Type IV, Neuronal Ceroid Lipofusinoses, Niemann-Pick Disease, Sandhoff Disease, Sanfilippo syndrome, Tay-Sachs Disease, Hunter Syndrome, Sialidosis, Multiple Sulfatase Deficiency, Childhood Epilepsy, CMT Peripheral Neuropathy, Neuronal Ceroid Lipofusinoses, Aspartylglycosaminuria, Mucopolysaccharidoses, Parkinsons Disease/Symptoms, GM1-Gangliosidosis, Charcot-Marie-Tooth Type 1A, Retts Syndrome, Dannon Disease, Mucopolysaccharidoses (due to GNS, HGSNAT, NAGLU, or ARSB), Sly Syndrome, Biotin basal ganglia disease, Pelizaeus-Merzbacher disease, Neuronal Ceroid Lipofusinoses, Canavan Disease, Beta-Mannosidosis, Wolman Disease, Cystinosis, Alzheimer's Disease/Symptom, Salla Disease, Jansky-Bielschowsky disease, Neuronal ceroid lipofuscinoses, or GM2 gangliosidosis.
In certain embodiments, the GOI is: CLN3, FUCA1, GAN, GALC, MCOLN1, PPT1, SMPD1, HEXB, SGSH, HEXA, IDS, NEU1, SUMF1, GAT1/SLCA1, FIG. 4 , CLN5, AGA, IDUA, GDNF, GLB1, PMP22/MFN2, MECP2, LAMP2, NAGLU, ARSB, GUSB, SLC19A3, PLP1, TPP1/CLN2, ACY2/ASPA, MANBA, LIPA, CTNS, GNS, HGSNAT, NEUROD1, SLC17A5, CLN6, CLN8, or GM2A.
In certain embodiments, the rAAV has a tropism for neuronal cells. In certain embodiment, the rAAV serotype is, AAV2.5, rAAVrh.39, rAAVrh.43, rAAV7, rAAVrh.8 or rAAVrh.10.
In certain embodiments, the rAAV serotype is AAV9. In certain embodiments, the rAAV has a pseudotype of xxx. In certain embodiments, the polynucleotide further comprises a 5′-UTR coding region, and a 3′-UTR coding region.
In certain embodiments, the heterologous enhancer is a viral enhancer.
In certain embodiments, the viral enhancer is CMV enhancer.
In certain embodiments, the viral enhancer is SV40 enhancer.
Another aspect of the invention provides a pharmaceutical composition comprising the rAAV vector of the invention.
In certain embodiments, the pharmaceutical composition is formulated for intravenous administration.
Another aspect of the invention provides a cell infected with the rAAV vector of the invention.
Another aspect of the invention provides a recombinant AAV virus comprising the rAAV vector of the invention, wherein the serotype or pseudotype of the recombinant AAV virus is for preferential infection of a neuronal cell.
Another aspect of the invention provides a method of treating a (genetic) disease or disorder in a subject, the method comprising introducing the recombinant AAV virus of the invention into the subject.
In certain embodiments, the genetic disease or disorder is a neuronal disease or disorder, the recombinant AAV virus preferentially infects a neuronal cell of the subject, and treats the neuronal disease or disorder.
Another aspect of the invention provides a method of producing the rAAV vector of the invention, comprising introducing the rAAV vector of the invention into a packaging cell line that constitutively or inducibly provides rep/cap proteins in trans.
With the general aspects of the invention described, further detailed embodiments of the invention are provided below.

2. Treatable Disease or Conditions

The recombinant AAV vector and recombinant AAV virus of the invention, together with the pharmaceutical composition comprising the same, can be used to treat a number of diseases and conditions, particularly neuronal diseases or indications treatable by gene therapy.
Diseases or conditions treatable by the AAV vector/virus/method of the invention are typically characterized by lacking a functional neuronal protein or enzyme, which neuronal diseases or indications can be alleviated (if not cured), treated, or prevented (e.g., at least the onset is delayed), by providing functional versions of the proteins or enzymes. The externally provided functional versions of the proteins or enzymes may be identical in sequence to the wild-type proteins or enzymes that are lacking in a host having such diseases or conditions, or may be a variant of the wild-type proteins or enzymes.
In certain embodiments, the neuronal diseases or conditions can be treated by introducing into the host the subject AAV virus that will infect target host (neuronal) tissues, and stably express the encoded GOI that is defective in the diseases or conditions throughout the life span of the host. The subject AAV virus may only need to be introduced into the host once, or can be introduced into the host multiple times as needed.
A few representative polynucleotide sequences of the invention encoding several genes-of-interest (GOI) are listed below. These polynucleotide sequences can be part of the subject AAV vector sequences. These sequences are for illustrative purpose only and are non-limiting.

Giant Axonal Neuropathy (GAN)

Giant axonal neuropathy (GAN) is a severe, slowly progressive neurodegenerative disorder characterized by progressive motor and sensory peripheral neuropathy, central nervous system involvement (including pyramidal and cerebellar signs), and characteristic kinky hair in most cases. GAN is inherited as an autosomal recessive trait, and is associated with more than 50 different causative mutations in GAN gene (16q24.1), encoding the ubiquitously expressed cytoskeletal protein gigaxonin.
Disease prognosis varies but is usually poor. Most patients become wheelchair-dependent in the second decade of life, and eventually progress to a bedridden state in early adulthood. Secondary complications, such as respiratory failure, may occur. Life expectancy does not exceed the third decade.
Current GAN treatment is symptomatic, focusing on stimulating intellectual and physical development, as well as communication skills, through physical, occupational and speech therapy, starting from the initiation of symptoms. Skeletal deformities and ophthalmoplegia may need surgery.
Thus in one embodiment, the GOI is GAN that encodes gigaxonin, and the disease or condition treatable by the subject AAV vector/virus is Giant Axonal Neuropathy (GAN). The composition and methods of the invention can be used to produce gigaxonin expressed permanently from AAV vectors stably maintained with the host's genome as extra chromosomal material, in native neuronal cells that normally express GAN, thus effectively treating giant axonal neuropathy.
A representative polynucleotide of the invention that can be encoded by the AAV vector of the invention is described below.

	(SEQ ID NO: 2)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	CTCACAGGGATACTGGGTAGGTATCATGGAGAAGA

	TAAGTCCAAGCTGCTTGTCTTGTGTGTTTCTTACC

	TTTTTAAATATATTTTTCGGCCGGGTGTGGTGGCT

	TATGCCTGTAATTCCAGTACTTTGGGAAGCCAAGG

	TGGGCGGATCACAAGGTCAGGAGTTCGATACCAGC

	CTGGCCAACATGGTAAAACCCCATCTCTACTAAAG

	ATGCAAAAGTTAGCCAGGCATGGTGGCACATGCCT

	GTAGTCCCAGCTACTTGGGAGGCTGAGGCAGGAGA

	ACCACTTGAACCCGGGAGGCGGAGGTTGCAGTGAA

	CAGAGATTGCACAACTGCACTCCAGCCTGGGCAAT

	AGAGGGAGACTCCATCTCAAAAAAAAAAAAGAAAG

	AAGAAAATATTTTTCAACATCCACAAAAATATAGA

	AACAGTACAATGAATCTCCACATCCCCATTAAACA

	TCCACAACAATATAGAGACAGTACAACGAATCTCC

	ACATCCCCATTACGTGGCTTTAACAATTAGTAACT

	TGCGGCCACTTTTGACTCATCTACACTCCCATCTG

	CTTCCCCATCCCTTTGTTTTGTTTTGAAGCAAATC

	CTAGACATCTTTATCATTGCATCCGGGAACACTGA

	GCTGCATTTACAGAAGAAAGTGGAAATGCATGTGT

	CTTCCACAGCCGTACCCTCCCCATCTATTCTGCCC

	TGAAAACAGTTAGATACTCGGATTCCTGCCAGTGA

	CTCCAAAGCCTTGGCTGGTTCAGGGTAAATACCCA

	AGACTTTATTGGAGCCTTTCAGTTGGTTGAAGGAC

	AGTGAAAATTCCATCCTGTCTTGCCTGGAATGCTA

	GCCACATTCTTCTGGTCCGGAGTCCTCATACAATT

	GCTAATTTTTTAGAGGCTTATGGAGTCCCTAACGT

	TTGCACAAAATGGTTGCTACATTCCACACTCCCAT

	GTGCCCTGAGCAACCTCCAGGGAAACTTCAGATAC

	CGTGGGGCTTGGCCTGCCAGCTGAGCTGACCCAGC

	GATCCTTTCTATCACAGCTTTGATCTTCACTGGGT

	TGGGAGCCTGTCTGAATCTAAACATCTGCCTCTCT

	ACTGCCCACCCCTTCAGCATTTCCTAGCTCTCCCT

	GAAAGGTGCAAACATGCATGCAGTTACTCAAACAG

	AATATTTATCCTTAACTCCCTCTCCCTCACAATTT

	CTTTTAAAACAATCCACTATTGTCTACCCCCGCCC

	CCGCATCACCCTTTCATCCAGGTGCCCATTCCCTT

	CTCTCTAGTCCTGCAATACCTAAGCATCGATCTGC

	CTTTAGCACATCCATCCCCGCCTTATTCTCCACGC

	AGGAGCTGAGTGACCTGCACAAAATCCCCTGCTAA

	ATACGGTACTTCCATGGTTCTTCACCACTGCAGAA

	ACCAAACTCCTTCCCACACCAAGGAGGCCCTGCAA

	AATTTAAGGCCTCCTTCGGACTTCTCCAGCTTCAT

	CTGCTACATCTCCTTCCTAGCCCGCAACCAAAACC

	ATCTTTTCCGGGTCTGTTTTCTCGGGTTTTTGTCC

	TCTCACCTGGGCAGCTCCTTTTCTCTCATAAATAA

	AGAAAGCTACAGCGACTTCGCCCTTGCTTCCATGC

	ATCATCTCATGAGTCCACAACAACACTAAGAATTC

	GCTTTCATTTTGCAGACGGAGAAACTGAGGATGGG

	CACCTTACCCATGGCCACAGAGCTAGTAAGTGGAA

	CAGCTGCAACCAGGATTCAAACTCGCGGCTCCAGA

	GGCCACAGGCTTAACCACGGAATCCTAGCCCGCCC

	GCACATCCCTCCATCGCGGGAGCGACCTATCTCAT

	CCGTAGACACCTTCCGCTGCACCCAGCCGCAGCTG

	GTCAGGCGGGCGGAGGAAGGAGGCTTCTGATTGGC

	CATCTCCAGCAGCGGAGGCGAGAAGGCGGGGAGCG

	ACGGTGCGGGGGCGTCGCGGAGAAATGACTTCATG

	ACGCGGCTTCTTAAAGGCGCCGGCCCAGCGCGCCG

	CGGA





	ATGGCTGAGGGAAGTGCGGTCTCAGACCCCCAACA

	CGCTGCAAGACTTCTGCGAGCTCTCTCATCTTTCA

	GAGAGGAGTCACGCTTCTGTGACGCACACTTGGTC

	CTTGACGGAGAAGAGATACCTGTCCAGAAAAATAT

	ACTTGCCGCTGCATCCCCATACATTCGCACCAAGC

	TGAACTACAATCCCCCTAAAGACGACGGGAGCACC

	TATAAAATCGAATTGGAGGGCATCTCCGTGATGGT

	AATGCGAGAGATACTGGATTACATATTTAGCGGCC

	AGATCAGGCTGAATGAGGATACGATCCAAGATGTT

	GTACAGGCAGCGGACTTGCTGCTTCTGACGGACCT

	TAAGACCCTTTGCTGCGAGTTCCTCGAAGGGTGTA

	TTGCTGCCGAGAATTGTATCGGAATACGAGACTTC

	GCACTCCATTACTGCCTTCACCACGTTCATTACCT

	TGCGACAGAGTATTTGGAAACACATTTCCGCGACG

	TTAGCTCTACAGAGGAATTTCTCGAACTTAGTCCT

	CAAAAGCTCAAGGAGGTTATTAGTCTTGAAAAGCT

	CAACGTCGGAAATGAGCGGTACGTGTTTGAGGCCG

	TGATACGGTGGATCGCCCACGACACCGAGATCCGC

	AAGGTCCACATGAAGGATGTAATGTCAGCGCTGTG

	GGTATCCGGTCTTGACTCTTCATACTTGAGAGAGC

	AGATGCTCAACGAACCTTTGGTGAGGGAAATAGTG

	AAGGAGTGTAGTAACATCCCCCTCAGCCAACCCCA

	GCAAGGCGAAGCGATGCTCGCGAACTTCAAGCCCC

	GAGGCTACAGCGAATGCATAGTAACAGTGGGAGGT

	GAAGAACGGGTCTCCCGGAAACCCACTGCGGCGAT

	GCGGTGTATGTGTCCCCTGTATGATCCCAATCGCC

	AACTTTGGATCGAGCTGGCGCCGCTGTCTATGCCA

	CGAATAAATCACGGGGTTCTTTCAGCCGAGGGGTT

	TCTGTTCGTCTTTGGAGGGCAGGATGAGAACAAAC

	AAACACTCTCCAGTGGGGAGAAATACGACCCCGAT

	GCAAACACTTGGACAGCTCTGCCACCGATGAACGA

	AGCCCGACACAACTTTGGTATAGTCGAAATCGACG

	GTATGCTGTATATCCTTGGAGGAGAGGACGGAGAA

	AAAGAACTCATCTCCATGGAATGTTACGACATTTA

	CAGCAAAACTTGGACAAAGCAACCCGACCTTACTA

	TGGTGAGGAAGATAGGATGTTATGCGGCAATGAAG

	AAAAAGATATACGCCATGGGAGGCGGTTCTTATGG

	TAAGCTCTTCGAGAGTGTGGAGTGTTATGACCCTC

	GAACGCAGCAATGGACAGCCATATGTCCCCTGAAG

	GAACGGCGATTTGGGGCGGTAGCGTGTGGAGTTGC

	AATGGAGTTGTACGTTTTCGGAGGTGTTCGAAGTC

	GGGAGGACGCGCAAGGGTCAGAGATGGTCACATGT

	AAAAGCGAATTTTATCATGACGAATTTAAGCGGTG

	GATTTATCTGAACGACCAAAACCTCTGTATTCCAG

	CTTCCAGTTCCTTCGTATACGGCGCTGTGCCAATA

	GGTGCGTCAATTTACGTCATAGGCGACTTGGACAC

	TGGTACTAATTATGATTACGTACGCGAGTTTAAGA

	GGAGTACAGGCACCTGGCACCACACAAAACCGCTG

	CTCCCGTCCGATCTGCGAAGGACAGGTTGCGCTGC

	ATTGAGGATTGCTAACTGCAAACTTTTCCGCCTGC

	AACTTCAGCAAGGGCTTTTCCGCATACGAGTCCAC

	AGTCCTGACTATAAAGATGACGACGACAAATA

In the sequence herein, double underlined sequences represent proprietary viral enhancer sequences (SEQ ID NO: 1, see below); bold sequences are native promoter sequences for GAN; italic sequences are coding sequence for the GOI (GAN in this case); the subscripted sequences immediately 5′ and 3′ to the GOI coding sequence are 5′-UTR and 3′-UTR sequences, respectively.
The proprietary enhancer sequences (SEQ ID NO: 1) is listed below.

(SEQ ID NO: 1)

GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGT

ACCTTCTGAGGCGGAAAGAACCAGCGTTACATAACTTACGGTAAATGGCC

CGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGACGTCAATAATGACG

TATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGT

GGAGTATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATA

TGCCAAGTACGCCCCCTATTGACGTCAATGACGGTAAATGGCCCGCCTGG

CATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

CTACGTATTAGTCATCGCTATTACCATG

The GAN protein sequence is:

(SEQ ID NO: 3)

MAEGSAVSDPQHAARLLRALSSFREESRFCDAHLVLDGEEIPVQKNILAA

ASPYIRTKLNYNPPKDDGSTYKIELEGISVMVMREILDYIFSGQIRLNED

TIQDVVQAADLLLLTDLKTLCCEFLEGCIAAENCIGIRDFALHYCLHHVH

YLATEYLETHFRDVSSTEEFLELSPQKLKEVISLEKLNVGNERYVFEAVI

RWIAHDTEIRKVHMKDVMSALWVSGLDSSYLREQMLNEPLVREIVKECSN

IPLSQPQQGEAMLANFKPRGYSECIVTVGGEERVSRKPTAAMRCMCPLYD

PNRQLWIELAPLSMPRINHGVLSAEGFLFVFGGQDENKQTLSSGEKYDPD

ANTWTALPPMNEARHNFGIVEIDGMLYILGGEDGEKELISMECYDIYSKT

WTKQPDLTMVRKIGCYAAMKKKIYAMGGGSYGKLFESVECYDPRTQQWTA

ICPLKERRFGAVACGVAMELYVFGGVRSREDAQGSEMVTCKSEFYHDEFK

RWIYLNDQNLCIPASSSFVYGAVPIGASIYVIGDLDTGTNYDYVREFKRS

TGTWHHTKPLLPSDLRRTGCAALRIANCKLFRLQLQQGLFRIRVHSPDYK

DDDDK

Conservative changes to the protein sequence above (e.g., 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, or 20 conservative residue changes, or up to 1%, 2%, 3%, 4%, 5%, 6%, 7%, 8%, 9%, or 10% conservative residue changes) that result in a functional equivalent version of the protein (e.g., that retains at least 70%, 75%, 80%, 85%, 90%, 95%, 97%, 99% of the wild-type enzyme activity) are also within the scope of the invention.
Likewise, in any of the nucleotide sequence encoding the protein and variants thereof, such as in SEQ ID NO: 2, nucleotide sequence changes that do not result in sequence change of the encoded protein in SEQ ID NO: 3 due to, for example, codon degeneracy, are within the scope of the invention. Further, codon optimization of any of the polynucleotide sequence for optimal expression in a specific type of host cell is also within the scope of the invention (see further details below).

Juvenile Neuronal Ceroid Lipofuscinosis (JNCL or Batten Disease)

Neuronal ceroid lipofuscinosis refers to a family of at least eight genetically separate neurodegenerative lysosomal storage diseases that result from excessive accumulation of lipopigments (lipofuscin) made up of fats and proteins in the body's tissues. The most commonly identified mutations are in the CLN3 gene, located on the short arm of chromosome 16 (16p12.1), found in childhood or juvenile NCLs (JNCLs).
JNCL is an autosomal recessive disorder. The normal function of CLN3 gene is not presently known, but it is known to be a transmembrane protein. Studies of the yeast CLN3 ortholog which encodes Battenin have suggested that the protein may play a role in lysosomal pH homeostasis. Furthermore, recent studies have also implied the protein's role in cathepsin D deficiency; the overexpression of the defective protein appears to have significant effects on cathepsin D processing, with implications suggesting that accumulation of ATP synthase subunit C would result.
All mutations resulting in JNCL have been shown to occur at the CLN3 gene. Of the mutations known to cause JNCL, 85% result from a 1.02 kb deletion, with a loss of amino acids 154-438, while the remaining 15% appear to result from either point or frameshift mutations.
Thus in one embodiment, the GOI is CLN3, and the disease or condition treatable by the subject AAV vector/virus is JNCL. The composition and methods of the invention can be used to produce CLN3 gene product expressed permanently from AAV vectors stably maintained with the host's genome as extra chromosomal material, in native neuronal cells that normally express CLN3, thus effectively treating JNCL.
A representative polynucleotide of the invention that can be encoded by the AAV vector of the invention is described below. Specifically, the proprietary enhancer sequence is in SEQ ID NO: 1. Native CLN3 promoter is known in the art. The coding sequence for CLN3 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

CLN3-DNA

	(SEQ ID NO: 4)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	TTCCCCCAGGGGCGGAAGGGGCCGGATTGGTGGCT

	CTGGGGTGCATCCCCTCACCTATGATTAGGCTTGC

	AAAAAAGAACTTGGCTAAACTTTATATTGTGGATA

	TCTGTGTCTGCCTCCCACCTTCCTGTGACTTCCTT

	GATGGCAGATGCCATGTATGAAACGCTGCTGAATT

	TCTAGAGCCCTGCACGTTAGTAGGATTCCATTTTC

	CATCAGCTGAGTGACCTGGGAGGCACACTAGAGAG

	GGCACATTTCTTTTCTTTTCTTTTCTTTTTTTTTT

	TGAGACAGTCTCACTCTGTCACCCAGGCTGGAGTG

	TAGTGGCGCAGTCTCAGCTCACTGCAACCTCTGCC

	TCCTGGGTTCAAGTGATTCTCCTGCCTCAGCCTCC

	CGAGTAGCTGGGATTACAGGTGCGTGCCACCACAC

	CTGGTTAATTTTTGTATCTTTAGTAGAGACAGGGT

	TTCACCATGTTAGCCAGGCTGGTTTCGAACTCCTG

	ACCTCAGGTGATCTGCCCACCTTGGCCTCCCAAAG

	TGCTGGGATGACAGGCATGAGCCACCACACCCAGA

	CGAGAGGGCACATTTCCAACTGCATTCTGAAGGCC

	TGGTGGTGAGAAAGAGCATATAGAGGTGGCACAGC

	CCATAGCAGAGATGAGGCCAAGGACAAGAAGAGAA

	TGCAGGATGCCGCTTTCCACCTAGAAGAAGGTAAG

	GGTATGATTCCTTATTTGGAACTACATAAAGTGAC

	CTTGCTCTACTTCTCTTCCCCATACCCTCCTTCTC

	CCCTCCCCAGCCCCACTTTCCTATTCCAATTAGGG

	CAATTCTATGAAATCACAGCATCAGAATCTTAGGC

	CTTAAAATCATTTAGTCTGGCCAGGTGCAGTGGCT

	CACGCCTGTAGTCCTAGCACTTTGGGAAGCCAAGG

	CAGGTGGATTGCTTGAGCTCAGGAGTTTGAGGCCA

	CCCTGGGCAACGTGGCAAAATCCAGTCTCTACAAA

	AAACACAAAAATTAGCTGGGCACAGTGGTGCGCAC

	CTGCTGTCCCAGCTACTCGGGAGGCTGAGGTGGGA

	GGATCACCTGAGCCTGGGAGGTCAAGGTTGCAGCA

	AACCAAGATCCTGCCACTGCACTCCAGCCTGGGCA

	ACAGAGCAAAACCCTGTCAAAAAAAAAAAAAAAAT

	CTGGTCTAAGCACCACCTTTGATGGAGGCAGGAAT

	CCTGCAGCAGCCTGGAGCCAACCCAATGGCTCCCT

	GCCTCTGGTGCTCTATTTCTTATCACCATACAGGT

	CTCTAAGGTCACCTTTAGGTCTAACATTGTATGAA

	TGATTGTCAGCAACTTTCCAATTTTTTTCATATAC

	ATATATTTTTTTTATTTTATTTTTTACTTTTTCTT

	TTTTTGAGACAGGATCTCACTCTGTCACCCAGGCT

	GGAGTGCAGTGGCGTGATCATAGCTCACTGTAGCT

	TCAACCTCCTGGGCACAAGTGATCCTCCCACCTCA

	GCTTCCAAGGTACCTGTGACTACAGGAATGCGCCA

	CCATGCCCGGCTAATTTTGTTATATATATATATAT

	ATATATTTGTACCTTGGTTTTCTCATCTGTAACAC

	CAGGGGTTGAAGTGCTGTGATATTTTTTGGTTCTA

	GGATAATTTGGAAATAGGGATCTCTTAACATCTTG

	GATACTGCCAATAGATCATTCACGAAATCCCTTTA

	GATGAGTGTTATGTTTACTTAGGTCTCTAAAACAA

	TTTGTAAGGTCAACAGATGGGGATAAGAATGACCT

	GAAGCTGGTCGTCTTTGTTCGCAACCAGAAACAGT

	CCTGCCTCAAAAAAAAAAAAAAAAAAAAGGGAGTA

	GGCAGGTGGCCATATTTGTTTCTGGCAAGTGAGTG

	CTGAAGGAAAGGAGCTGAAGCCTCCCACAGTCATA

	ACTGGTGCTGGCAGGCTACTGTCTCGGTCTTGGGC

	GCCACTGATCTAAGGTCACGGCTCTGCTTGCTGCT

	CCCACCCGCTCCAGTTTAAAACCTGCGGTTCCAGG

	GTTCTCCAGCCCCTCCCTTTTTCACGCTCCGAAGC

	CGAGAAGGCCCAAAGCGAAGACAGAGAGGACCCGG

	AAGTAGGGAAAACCTCTGAGCACGTGATGGGGGAA

	CACGCGGGTGCTGT





	ATGGGAGGTTGTGCTGGTTCTCGACGCCGGTTCTC

	AGATAGTGAGGGGGAGGAAACGGTGCCCGAGCCTC

	GGCTTCCACTTCTGGACCATCAAGGCGCTCATTGG

	AAGAATGCAGTCGGGTTTTGGCTCCTCGGTCTCTG

	CAACAATTTCTCTTACGTGGTCATGTTGAGCGCTG

	CCCATGACATTCTTAGTCATAAAAGAACCTCAGGC

	AATCAGTCCCACGTTGATCCAGGTCCAACACCTAT

	TCCTCATAACTCTAGCAGCAGATTTGACTGTAATA

	GTGTTAGCACAGCTGCAGTGCTGCTCGCTGATATA

	CTTCCGACGCTTGTTATAAAGTTGTTGGCTCCTCT

	GGGATTGCACTTGCTGCCCTATAGTCCCCGAGTCT

	TGGTTTCTGGCATTTGTGCTGCCGGCTCATTTGTC

	TTGGTGGCTTTCAGTCACAGTGTGGGGACCAGTCT

	GTGTGGAGTCGTGTTCGCATCAATTAGCTCTGGAC

	TGGGCGAAGTAACCTTCCTGTCCCTCACAGCCTTT

	TATCCGCGCGCCGTAATTAGTTGGTGGTCATCTGG

	AACCGGCGGCGCGGGGCTGCTCGGCGCCCTTAGCT

	ACCTGGGATTGACTCAGGCGGGGTTGTCACCACAA

	CAGACTCTCCTTAGCATGCTTGGCATTCCAGCCTT

	GCTTCTGGCTTCTTACTTTCTGCTCCTTACGTCCC

	CGGAAGCGCAGGACCCTGGCGGTGAAGAGGAGGCG

	GAGTCCGCCGCTCGACAGCCACTCATTCGAACTGA

	AGCTCCCGAGTCCAAACCAGGAAGCTCTTCCTCCC

	TCTCCCTGCGGGAACGGTGGACAGTCTTTAAGGGA

	CTGCTGTGGTACATAGTGCCTCTTGTGGTAGTGTA

	TTTCGCGGAATACTTCATAAACCAGGGTCTTTTTG

	AGCTTCTTTTTTTTTGGAATACAAGCCTGTCACAC

	GCCCAGCAGTACCGATGGTATCAGATGCTGTACCA

	GGCGGGGGTTTTCGCTAGCCGATCCTCCCTGCGGT

	GCTGCAGGATTCGATTCACTTGGGCTCTCGCGTTG

	CTGCAATGCTTGAACCTCGTTTTCCTTCTCGCAGA

	CGTCTGGTTCGGTTTTCTCCCTAGTATCTATCTGG

	TCTTTTTGATTATATTGTATGAAGGTCTTCTGGGA

	GGAGCGGCATACGTGAATACTTTTCACAATATCGC

	CCTTGAGACTTCCGACGAGCATCGGGAATTTGCCA

	TGGCGGCGACGTGTATCTCCGATACCCTGGGTATA

	AGCCTGAGTGGACTGCTCGCGCTTCCGCTCCACGA

	TTTTCTGTGTCAATTGAGCGATTATAAAGATGATG

	ATGACAAATA

The CLN3 protein sequence is:

(SEQ ID NO: 5)

MGGCAGSRRRFSDSEGEETVPEPRLPLLDHQGAHWKNAVGFWLLGLCNNF

SYVVMLSAAHDILSHKRTSGNQSHVDPGPTPIPHNSSSRFDCNSVSTAAV

LLADILPTLVIKLLAPLGLHLLPYSPRVLVSGICAAGSFVLVAFSHSVGT

SLCGVVFASISSGLGEVTFLSLTAFYPRAVISWWSSGTGGAGLLGALSYL

GLTQAGLSPQQTLLSMLGIPALLLASYFLLLTSPEAQDPGGEEEAESAAR

QPLIRTEAPESKPGSSSSLSLRERWTVFKGLLWYIVPLVVVYFAEYFINQ

GLFELLFFWNTSLSHAQQYRWYQMLYQAGVFASRSSLRCCRIRFTWALAL

LQCLNLVFLLADVWFGFLPSIYLVFLIILYEGLLGGAAYVNTFHNIALET

SDEHREFAMAATCISDTLGISLSGLLALPLHDFLCQLSDYKDDDDK

Conservative changes to the CLN3 protein sequence above (e.g., 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, or 20 conservative residue changes, or up to 1%, 2%, 3%, 4%, 5%, 6%, 7%, 8%, 9%, or 10% conservative residue changes) that result in a functional equivalent version of the protein (e.g., that retains at least 70%, 75%, 80%, 85%, 90%, 95%, 97%, 99% of the wild-type enzyme activity) are also within the scope of the invention.
Likewise, in any of the nucleotide sequence encoding the protein and variants thereof, such as in SEQ ID NO: 4, nucleotide sequence changes that do not result in sequence change of the encoded protein in SEQ ID NO: 5 due to, for example, codon degeneracy, are within the scope of the invention. Further, codon optimization of any of the polynucleotide sequence for optimal expression in a specific type of host cell is also within the scope of the invention.
Numerous other neuronal diseases/disorders can be similarly treated by the constructs, AAV vectors, and methods of the invention. A representative (non-limiting) list of the treatable diseases and disorders, along with the corresponding GOI's, are provides in the table below.


Target No.	Diseases	Target Gene (GOI)	GOI Size

1	Ceroid-Lipofuscinosis	CLN3	1403
2	Fucosidosis	FUCA1	1487
3	Giant Axonal Neuropathy	GAN	1880
4	Globoid cell leukodystrophy	GALC	2144
5	Mucolipidiosis Type IV	MCOLN1	1829
6	Neuronal Ceroid Lipofusinoses	PPT1	1007
7	Niemann-Pick Disease	SMPD1	1982
8	Sandhoff Disease	HEXB	1757
9	Sanfilippo syndrome	SGSH	1595
10	Tay-Sachs Disease	HEXA	1676
11	Hunter Syndrome	IDS	1739
12	Sialidosis	NEU1	1334
13	Multiple Sulfatase Deficiency	SUMF1	941
14	Childhood Epilepsy	GAT1/SLCA1	1886
15	CMT Peripheral Neuropathy	FIG4	2810
16	Neuronal Ceroid Lipofusinoses	CLN5	1163
17	Aspartylglycosaminuria	AGA	1127
18	Mucopolysaccharidoses	IDUA	2048
19	Parkinsons Disease/Symptoms	GDNF	722
20	GM1-Gangliosidosis	GLB1	2120
21	Charcot-Marie-Tooth Type 1A	PMP22/MFN2	569
22	Retts Syndrome	MECP2	1583
23	Dannon Disease	LAMP2	1322
24	Mucopolysaccharidoses	NAGLU	2318
25	Mucopolysaccharidoses	ARSB	1688
26	Sly Syndrome	GUSB	2042
27	Biotin basal ganglia disease	SLC19A3	1577
28	Pelizaeus-Merzbacher disease	PLP1	920
29	Neuronal Ceroid Lipofusinoses	TPP1/CLN2	1778
30	Canavan Disease	ACY2/ASPA	1028
31	Beta-Mannosidosis	MANBA	2726
32	Wolman Disease	LIPA	1286
33	Cystinosis	CTNS	1289
34	Mucopolysaccharidoses	GNS	1745
35	Mucopolysaccharidoses	HGSNAT	1994
36	Alzheimer's Disease/Symptom	NeuroD1	1157
37	Salla Disease	SLC17A5	1574
38	Jansky-Bielschowsky disease	CLN6	1022
39	Neuronal ceroid lipofuscinoses	CLN8	947
40	GM2 gangliosidosis	GM2A	668

The sequences of the above genes that can be included in the viral vectors as GOI's are provided below.
The FUCA1 protein sequence is:

(SEQ ID NO: 6)

MRAPGMRSRPAGPALLLLLLFLGAAESVRRAQPPRRYTPDWPSLDSRPLP

AWFDEAKFGVFIHWGVFSVPAWGSEWFWWHWQGEGRPQYQRFMRDNYPPG

FSYADFGPQFTARFFHPEEWADLFQAAGAKYVVLTTKHHEGFTNWPSPVS

WNWNSKDVGPHRDLVGELGTALRKRNIRYGLYHSLLEWFHPLYLLDKKNG

FKTQHFVSAKTMPELYDLVNSYKPDLIWSDGEWECPDTYWNSTNFLSWLY

NDSPVKDEVVVNDRWGQNCSCHHGGYYNCEDKFKPQSLPDHKWEMCTSID

KFSWGYRRDMALSDVTEESEIISELVQTVSLGGNYLLNIGPTKDGLIVPI

FQERLLAVGKWLSINGEAIYASKPWRVQWEKNTTSVWYTSKGSAVYAIFL

HWPENGVLNLESPITTSTTKITMLGIQGDLKWSTDPDKGLFISLPQLPPS

AVPAEFAWTIKLTGVKDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for FUCA1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

FUCA1-DNA

	(SEQ ID NO: 7)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	CCCATCTGTATTAAAAATACAAAAATTAGCCAGGC

	GTGGTGGCGTACCTGTAAACCCAGCTACTCGGGAG

	GCTGAGGCAGGAGAATGGCTTGAACCCAGGAGGTG

	GAGGTTGCAGTGAGCTGAGATTGCAGCACTGCATT

	CCAGCCTGGGCGACAGAGCAAGACTGCATCTCAAA

	AGAAAAGAAAAGAAAAAAGAAAAGAAAAGAAAAGA

	AAACAGCTGTGATTCAGCAATAGTCATTTTCTCTC

	CTTGTCACTGCCAGAGTGAGGAAGTGCTGGGTTCT

	GTCAACATAGGCTCTGACTTCTCATGAGGTGCAAG

	AAAGAAGCAGCATGAACAAAGGCATAGATTTTGCA

	CCTGGAGACACGTATTCTAGTCCCACCTCTGACTC

	AAACTAGCTGCATGATCTTGGGCAGCCCCCCAGCC

	CTCTCTGGGCTTCAGTTCCCTCCTCGGTACAATGG

	GAAGGGTAGGGAAGAGGATGGTGCCTAGGATCTTC

	AAAGTCCCAACTCTGATGTGCTGCCATTCTCTCAC

	TGGCATGTGGGAACTGAGTGTGCTACGTGGAGTCA

	TGAGACTTCTTCTTCCTTTTCTTTTTCTTTTTTTT

	TTTTTTTTTTTTTGAGACCGAGTCTCACTCTGTCG

	CCCAGGCTGGAGTGCAGTGGTGCGATCTCGGCTCA

	CTGTAACCTCCACCTCCCGAGTTCAAGCAATTCTC

	CTGCCTCAGCCTCCTGAGTAGCTGGGATTACAGGC

	GCACCCCATGCCCGGCTAATTTTTGTATTTTTAGT

	AGAGATGGGGTTTCACCATGTTGGCCAGGCTGGTC

	TCAAACTCCCGACCTCAGGTGATCTGCCCACCTCG

	GCCTCCCAAAGTGCTGGGATTACAGGCGTGAGCCA

	CCTTGTCTGTTCAGAGACTTCTGACTTCCGTATTT

	CCCTCGCTAATGGCCTCACCTCCCGTTTGAGTATG

	TGCAGCATGTGCAAATTGCAGAGGAACTGGGGAAC

	TTCCCTGCCAGGCAATCCTGGGTCCTCTTGGCTCT

	CCACTTCCTCCTTCCTTCCTTCCTTCCTCCTTCCT

	TCCTTCCTTCCTTCCTTTCTTTCTTCCTTGGTCTG

	GCTCTGTCGCCCAGGGGCTGGAGTGCAGTGGCGGT

	ATCTCGGTACAAACTAAGCTATTTGAAATGCCTCG

	GAATTCCCAAAGAAAGCTCAGTAAATACCAGACAG

	ATGAAACGGTCCTGCCCGCTCTCCCTCTTTCAGTA

	CCCAGTTTTCTCTTTCCTCTCCTTTCATAACGTTT

	CTTCCCATCATCCCTCTACCTCCTTATTTCTGTCA

	GGGCTGTTAGATACCCTTCCGGTCTGTTATCTCCT

	CCTCCAGCCTTGTGCTACAGGACAACTCAAAAGCC

	CCCTATGGCTCCCAAGTGATCACTAAATAAAACTC

	GTGATCTCACTTCAGCCTACCCCTGCAGGATTGCT

	GCGGCAAAATGGACTACTCCCAGAGGCGTGATCAC

	GGTTCGGTTCACTGCAGCCCAGACTTCCCGGGCTC

	AAGCGATCCCGAACCTCCCACCTCAGCCTCTCGAG

	TAGCTGGGACTCCAGACGTGCACCACTACGCCAGG

	CTCATTTTGTTTATTTTTTATAGAGACAGGCTAGT

	CTCCAACTTGTGGGCTCAGAGCGATCCTCCCGCCT

	CGACTTCCGAAAGTGTTAGGATTACAGGCGTGAGC

	CACTGCGTCCGGCTCAATGGACTCCTTCTTAAATG

	TGAGTTCTCCGCACACACTGGGCCTTGCTCACCTC

	CACGATTTAACCTCGCAGTTAAGTCCCTTTTCAAG

	GTTTTTATTCGTCCTTCAGCGAAGCGCTGTCAACG

	CCTCCCTGCGTCTTTCCCTTCTTGAGCACCAGAGG

	GCGCTCCTCCGACCCGAGGAGGAAGCGACTCGGGC

	CAACCTGTCCAGGTTGTCCCACCCCTTTTTCCTTC

	CGGGCCAATCGTTAGTCAGAGTGGGCGGAGCCGCC

	CGCGGGCACCTGCGCGTTAAGAGTGGGCCGCGTCG

	CTGAGGGG



	ATGAGAGCACCCGGCATGCGATCAAGGCCCGCAGG

	GCCAGCTCTCCTCCTCCTGCTCCTTTTTTTGGGGG

	CTGCAGAATCTGTAAGAAGAGCTCAACCCCCAAGG

	AGGTACACTCCTGATTGGCCCTCATTGGACTCACG

	CCCACTCCCGGCGTGGTTTGATGAAGCTAAGTTCG

	GAGTGTTTATCCATTGGGGTGTATTCAGCGTGCCC

	GCGTGGGGGTCTGAGTGGTTTTGGTGGCACTGGCA

	AGGAGAGGGCCGCCCGCAATACCAGCGATTCATGA

	GGGACAATTATCCTCCAGGCTTCTCATACGCTGAC

	TTCGGTCCTCAGTTTACAGCTAGATTCTTCCATCC

	AGAGGAGTGGGCCGATCTTTTCCAGGCGGCTGGAG

	CTAAGTACGTCGTTCTGACGACAAAGCACCATGAA

	GGGTTCACCAACTGGCCAAGTCCAGTGTCCTGGAA

	CTGGAACAGTAAGGATGTCGGTCCTCATCGGGATC

	TGGTAGGCGAACTTGGGACTGCTCTCCGGAAGCGC

	AATATACGCTATGGTCTTTATCACAGTCTGCTGGA

	ATGGTTCCACCCACTGTACTTGCTGGACAAGAAGA

	ACGGATTTAAAACCCAACATTTTGTTAGCGCAAAG

	CATATGCCAGAGTTGTACGACCTGGTAAATTCCTA

	TAAACCCGACCTCATCTGGAGCGACGGAGAATGGG

	AGTGCCCGGATACTTATTGGAACTCAACTAACTTT

	CTCTCCTGGCTGTATAATGACTCCCCTGTGAAGGA

	CGAAGTTGTTGTAAATGACAGATGGGGTCAAAACT

	GCTCATGTCATCACGGGGGATACTATAATTGCGAA

	GACAAGTTTAAGCCTCAATCTCTTCCCGATCATAA

	ATGGGAGATGTGTACTTCTATTGACAAGTTTAGTT

	GGGGATACAGGCGCGACATGGCCTTGTCTGATGTT

	ACGGAGGAATCAGAAATCATCTCCGAGCTGGTACA

	AACGGTGTCATTGGGAGGCAACTACCTGCTGAACA

	TAGGCCCAACGAAAGACGGGCTTATCGTGCCGATT

	TTTCAAGAACGCCTTCTCGCAGTTGGCAAATGGCT

	CTCTATTAATGGGGAAGCTATTTACGCGTCAAAAC

	CTTGGAGGGTCCAATGGGAGAAAAACACCACTAGT

	GTATGGTACACAAGCAAAGGAAGCGCCGTCTACGC

	TATCTTCCTGCACTGGCCAGAGAATGGCGTACTCA

	ATTTGGAATCACCTATAACCACATCCACGACTAAG

	ATCACCATGCTCGGGATTCAAGGTGACCTTAAATG

	GTCTACAGACCCAGATAAAGGTCTTTTTATTAGTC

	TTCCCCAGCTGCCTCCCAGCGCGGTTCCGGCCGAG

	TTCGCCTGGACGATCAAGCTGACCGGGGTTAAAGA

	CTATAAAGACGATGATGATAAATA

The GALC protein sequence is:

(SEQ ID NO: 8)

MAEWLLSASWQRRAKAMTAAAGSAGRAAVPLLLCALLAPGGAYVLDDSDG

LGREFDGIGAVSGGGATSRLLVNYPEPYRSQILDYLFKPNFGASLHILKV

EIGGDGQTTDGTEPSHMHYALDENYFRGYEWWLMKEAKKRNPNITLIGLP

WSFPGWLGKGFDWPYVNLQLTAYYVVTWIVGAKRYHDLDIDYIGIWNERS

YNANYIKILRKMLNYQGLQRVKIIASDNLWESISASMLLDAELFKVVDVI

GAHYPGTHSAKDAKLTGKKLWSSEDFSTLNSDMGAGCWGRILNQNYINGY

MTSTIAWNLVASYYEQLPYGRCGLMTAQEPWSGHYVVESPVWVSAHTTQF

TQPGWYYLKTVGHLEKGGSYVALTDGLGNLTIIIETMSHKHSKCIRPFLP

YFNVSQQFATFVLKGSFSEIPELQVWYTKLGKTSERFLFKQLDSLWLLDS

DGSFTLSLHEDELFTLTTLTTGRKGSYPLPPKSQPFPSTYKDDFNVDYPF

FSEAPNFADQTGVFEYFTNIEDPGEHHFTLRQVLNQRPITWAADASNTIS

IIGDYNWTNLTIKCDVYIETPDTGGVFIAGRVNKGGILIRSARGIFFWIF

ANGSYRVTGDLAGWIIYALGRVEVTAKKWYTLTLTIKGHFTSGMLNDKSL

WTDIPVNFPKNGWAAIGTHSFEFAQFDNFLVEATRDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for GALC and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

GALC-DNA

(SEQ ID NO: 9)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG TCCTCTGTCATCACCTTGGTATTTCCCATC

TAGTCACGGTATTAAGTGGGTCTTATGCGCTTGTTAAGAGCTGGTAAGAGAAGCTTAACATTCACTTG

ATGGAATGGTTACTGTGGCTTGTATCCACAGGAGCTCCAGCTCATGCTTAAAATACTGTGTTAGAACA

AATGGATCAGAGTGAATTCTGTAACAGTAAATCCACAGAACTGAAAATTACTGACTTGCATCATAAAC

TGGTTGCAGTGGCAAGGCAGAGAGTTCAGATTCGTGTTGTACTGGGGAAAGAAGAAACCAAAGAAAAT

AAGTGGTTCATACACAGGGGACTATTTAACTGATCCAAGCCAGAAGACTACTGAGGGTGAAAGGAGCA

TTATCAGTAATTATGTTTAGACAAAAGTTGTGACTGTCCTTAGTAAACCAGGATAGACGTAGAGTCAA

TTCAAAAAGAAAGAAAGAACAAACATATCTAATGTACATTAAGAACCCAGGGATATCGGGTTCTTAAT

GTGCATATTTATTGGATTGCCCAGAGCTTTGACCTTCAAGTATAATTGAGTTCTTGAAACTTAGATTT

AGGTTTGTGGTTAAGGGGCAAAATAATCAGATTCCAAATAGGGTTCTGCAGACAAATCCCACAGGTCC

TAAAGTCTCTGGGTCTTTTCCTTGCCATTGTGGTATCTCAGGGGCTTGTGTAGGCAATGATAACCAGT

TCCATACTTCTGCTAAGAGAAATTTGGGGATCAGAAAATCTTTCTCTGCAGCTCTTCAGAGTCTCTTT

AATTTACTTCAATAACAAGGAAATCAGAAATCTCAGGACAGTGCTGGATGTGGCAGAAATTCAGGGCA

ATTCACAACTTTCCTACCAATTTAGAGTTACCTGTCTGGAATCACTTCAACTGACAGTGGAAGATAGT

TTATAAAATTGTAGGGGAGGCAAAATTTCACCTTCGTCCCTGTAGGGTCCCAGCATGGCCCTAGTATT

AAATTGATTTAAGATATTAATAGACTGACAGGAGACAACCACACAAATGTTTATTTTACATAGCACGG

GGGCCCTCATTAAAAAAAAAAAAGACCTAAAGCAGCAGTTAAGAGCCAATTGCTTATAGAGTGAAATG

GACAAAAAATAGTAGTTGTGAAACATGATAAGGCGAAGGGGCTTGGGGTAAAGAAGCGACTAGAAAGA

TAGGGATTAACAAATTTTGTTTGGACAGATTTCCCTCGACTTCAACTTTCTGTCCTTGAAGATAACAA

TGTTATTTTATAGGGAAGACATCTTTCACATGGGAATTTCATCTCCTCCTTTTAAGACACATAATATC

CATGACAGTGATCTTGCACCTGCTGTTTTTAAGTGCCTTTAATTTAAAATAGTCAATATGCCACAGCA

GCATATTTTGAGGTGGTATATTATTAACTCCTTCAAGGCTAAAAGCTTTGAATATGCCGGGGTAGTTA

CCAGACTGGAGAACCTTCTCAGCTATTTGCCACTTGTGGAATGCATTCAAATGTACTGAGTGCTTATT

AACTTTTCTCTCAGTCCCTCACTCTGCTGCTCTTCTCGCCTCATCTCACATAAGGGAAAACTCAGGCT

CCAAAAGCAAAGTGATTCCACCAAGATGACAATGGAATCGGGACCTGGTGTCCAAGCCTGGTCCTCGC

ACGAGGTAGATACTCCCTGAGATGCTGGGGAAGAGCCATGGTAGGGCCACTCATGGCCCTCTTCCTTT

GGCGGACCTTGGAATCCACCTTCCCTGCGTTAAAGTGCTCCACCAGGTGACGCCGGAAGAGAAGCCAG

CAGTAAGTACATGTGAAATCCAGTAGCTGACCACAGACACGCGAGCGATAAGAAAAAGCTCGCTGCTT

CTTTTCTCAACACTCCGCCAGCTGCGTCAGAGGCCCTTGACCCGCACTCAAAATGGCGGCGGCGGCGT

CAGCATCAGCGGCCTCCTGCCCGTATCTATCGTGGCGGCGACGGGACCCGCCTCCCTGGGCGCCGG

ATGGCCGAATGGCTCCTTTCAGGATCATGGCAGCGCCGA

GCTAAAGCGATGACGGCTGCCGCGGGCTCTGCTGGGAGAGCAGCTGTTCCTCTTCTGCTTTGTGCCTT

GTTGGCACCTGGCGGAGCCTACGTTTTGGATGACTCTGATGGATTGGGTCGCGAGTTCGATGGTATCG

GTGCTGTGTCTGGTGGCGGAGCCACGTCCAGGCTGCTGGTAAATTACCCGGAACCATATCGAAGCCAA

ATCCTTGACTATCTTTTCAAACCTAACTTTGGAGCGAGCCTTCATATTCTCAAGGTCGAAATAGGCGG

AGACGGGCAGACAACTGATGGCACCGAGCCGTCTCATATGCATTACGCTTTGGACGAAAATTACTTTA

GAGGATACGAATGGTGGCTGATGAAGGAAGCCAAGAAAAGAAATCCCAATATTACCTTGATCGGCTTG

CCGTGGTCATTTCCCGGGTGGCTGGGCAAGGGATTTGATTGGCCGTATGTAAATCTTCAATTGACGGC

CTATTACGTAGTTACATGGATAGTAGGTGCCAAACGCTACCATGACCTGGATATTGATTACATTGGAA

TTTGGAACGAACGATCCTATAACGCAAATTACATCAAAATTCTCCGAAAGATGCTCAACTATCAAGGC

CTCCAGAGAGTTAAAATCATCGCGTCAGATAACCTTTGGGAGTCTATTTCTGCCTCAATGCTGCTCGA

CGCGGAACTTTTTAAGGTGGTTGACGTTATAGGGGCGCATTATCCAGGTACCCACAGCGCTAAAGACG

CAAAGCTCACGGGCAAAAAGCTGTGGAGCAGTGAGGACTTTAGTACCTTGAATAGTGACATGGGTGCA

GGGTGTTGGGGTAGGATACTGAACCAGAATTACATCAATGGATATATGACGAGCACTATAGCATGGAA

CTTGGTAGCGAGTTATTACGAGCAGTTGCCGTACGGTCGATGTGGTCTTATGACGGCGCAAGAACCGT

GGAGCGGGCACTATGTGGTCGAGAGTCCCGTTTGGGTAAGCGCTCATACAACTCAATTCACGCAGCCT

GGGTGGTATTATCTCAAGACCGTTGGACACCTTGAGAAAGGCGGGAGTTACGTAGCACTTACCGACGG

CTTGGGGAACTTGACTATTATCATTGAAACAATGAGTCACAAACACAGCAAATGTATTAGGCCTTTCC

TGCCATATTTTAATGTCTCTCAGCAGTTCGCTACTTTTGTGCTTAAAGGAAGCTTCAGTGAAATACCC

GAGCTCCAAGTGTGGTATACCAAACTTGGTAAAACTAGTGAACGATTTCTTTTCAAACAACTCGACTC

ACTTTGGCTGCTTGACTCTGACGGAAGCTTCACTCTCTCTCTTCACGAAGACGAGTTGTTTACTCTCA

CTACGCTTACTACTGGACGCAAGGGTTCATATCCTCTTCCTCCAAAATCTCAGCCATTCCCTTCCACA

TATAAGGATGATTTTAACGTCGATTATCCCTTCTTCTCCGAAGCGCCGAATTTTGCGGACCAAACAGG

TGTATTTGAATATTTTACTAACATTGAGGACCCAGGAGAGCATCACTTCACCTTGCGACAGGTCTTGA

ACCAGAGACCAATCACTTGGGCAGCCGACGCCTCTAATACCATATCAATTATCGGTGATTACAACTGG

ACTAATCTCACAATTAAATGTGATGTCTATATTGAAACTCCTGACACTGGAGGTGTTTTCATCGCCGG

GCGCGTCAACAAAGGAGGTATCCTTATCCGATCAGCTCGCGGTATCTTTTTTTGGATTTTTGCGAATG

GAAGCTACCGAGTTACAGGTGATCTTGCTGGGTGGATTATCTACGCCCTCGGCAGAGTAGAGGTTACT

GCCAAAAAATGGTATACTTTGACGCTGACGATCAAGGGCCACTTCACAAGTGGTATGCTTAACGATAA

AAGCCTGTGGACGGACATTCCAGTGAACTTTCCTAAGAACGGTTGGGCCGCCATTGGGACACACTCAT

TCGAATTTGCCCAATTCGATAATTTTCTCGTGGAGGCAACCAGAGATTACAAGGATGATGATGACAAA

TA

The MCOLN1 protein sequence is:

	(SEQ ID NO: 10)
	MTAPAGPRGSETERLLTPNPGYGTQAGPSPAPPT

	PPEEEDLRRRLKYFFMSPCDKFRAKGRKPCKLML

	QVVKILVVTVQLILFGLSNQLAVTFREENTIAFR

	HLFLLGYSDGADDTFAAYTREQLYQAIFHAVDQY

	LALPDVSLGRYAYVRGGGDPWTNGSGLALCQRYY

	HRGHVDPANDTFDIDPMVVTDCIQVDPPERPPPP

	PSDDLTLLESSSSYKNLTLKFHKLVNVTIHFRLK

	TINLQSLINNEIPDCYTFSVLITFDNKAHSGRIP

	ISLETQAHIQECKHPSVFQHGDNSFRLLFDVVVI

	LTCSLSFLLCARSLLRGFLLQNEFVGFMWRQRGR

	VISLWERLEFVNGWYILLVTSDVLTISGTIMKIG

	IEAKNLASYDVCSILLGTSTLLVWVGVIRYLTFF

	HNYNILIATLRVALPSVMRFCCCVAVIYLGYCFC

	GWIVLGPYHVKFRSLSMVSECLFSLINGDDMFVT

	FAAMQAQQGRSSLVWLFSQLYLYSFISLFIYMVL

	SLFIALITGAYDTIKHPGGAGAEESELQAYIAQC

	ODSPTSGKFRRGSGSACSLLCCCGRDPSEEHSLL

	VNDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for MCOLN1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

MCOLN1-DNA

(SEQ ID NO: 11)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG ACCCTGACCCTGGCTCTGGGCCGGGCACTC

TGCCGGATCCCAGCTCCAAACCCCTCCCCGGCTCCAGATCCACCCCCAGCCCTACTCCTGTGGAATCT

TCTGACCCAAAGGCTGGGCACGACGCTGGTCCCGACCTTGTGCCCAGCCCAGACCTTGATCCTGTGCC

CAGCCCAGACCCTGATCCTGTGCCCAGCCCTGATCCCAACCCTGTGTCCTGCCCTGACCCCTGTTCTC

CCACTCGTGGCACTGTCAGCCCAGCCCTCCCTACCGGCGAGAGTCCAGAGTGGGTACAGGAGCAAGGG

GCACTGCTGGGGCCTGATGGCTGAAGGAGACGCCGGCATCCTCGGGGGCCTGGGGAAGTTGTGTGTTG

TGCAGTCAGTAAAATCCTCCCACTGCCTCCGGGCTCTGTGTCGTGGCTTGCCTTTTGTGCTTTCTTTC

TGAGCACAGCCCCTTGCTCCGGAGATGGGACATTGTGGCCAGGGCCAGCCACAATCTGGAGCTCCAGT

GTCCTGGCTTCCTTCTTGGGCCCACCAGCTAGACTTTCCAGCTTAACCCACAGTGTCTCATCCTGGTC

CATCCCACTAGTCACCACTCTCTGTTCCTCTCACTTACCCCTTGCTCTTCAAAGCCCATACAGTAGGT

ATACAAGTGGACAAAAAAAGTTGCTCATTTATGCAATCAACAAACATCTCTGGATTGCTGGGGTCTCA

GCAGGGAACAAGATAAATATGGCCTCGACCTGCATGGAGCTCATAGATACTAAATTCAGAATACTTAA

AAAATAATTACGGGGTATAGTACATTCTAGGAGAAGCATAACAAGACTTCTGATATAAATGGCAGGCA

GCTTTCTCAATGAAGGATTTTGTAATCCCAATAATCACTAATTTAATAATCAGTACTGTTTGCCCAGC

CTTATGCGATAGTTTTTGCATTCTCTCATTTAATCCTCTCAACAGCCCCAGTAGGTAGATGACTTTGA

ATATCCCCATTTTGCAAATGAGAAAATTGAGGCACATTTTTTTTTTTTTTTTTAGACAGTCTTGCTCT

GTTGCCCAGGCTGGAGTGCAGTGGTGTGATCATAGCTCACTGCAGCCTCGACCTCCTGGGCTCAAGCG

ATCCTCCCACCTTAGCCTCCCGAGTAGCTGGGATTGCCGGTGCATGCCACCGCCCACTGCGCTCAGCT

TGGAGTTGAAGGGACTCTGGAAGATGTAGAAGTGGCATTGTCAGTGCCTAGATTTAAATCCCAATTGC

CCTCCAGGGTCCAAATTCTTAACCATTACGCTCCAGGGCAAAAGTATGCAAAGGCTCTGGGGCTATAG

AAAGATGAGCTTTGGATGGAGGTAGGAGCCAGATCAGAGGGCCCTGATAGACGAGAGTGGGGACTCTG

CCTGTCATTACAGAGCAATGGGAAGCCGAGGGCAGGTTCTCGCAGGAAGGATAGGAATTATTCTTTGA

AGATGCTTGTGGCTGCTGGGTAGAGAGTGGAGTGGAGGGAGGCTGAGATCGGGGAGGAGGTTGCTGCA

AAGATCCAGGCCAGGAATGTTGGAAGACTCTGGGCTGGGGGCCATGGGGTGGGGATAAGTGGTTCTAT

TTGATACATAATTAGGAAATCGTGTTTGCTGAAGATGCGCAGGAGAAGGGTAAAAGGAGTTTCTGGGA

GAAAGAGGAAGACAGCGTTGAGATAGTAGGCAGGGTCATCACCAGGCACCAAGGAGGATAAGGGGTCA

AGCTCTGGACATGGAAGTCACAAGCCTGGCACCGGATTCGGGGCATGGCCGGGAGCCAGGGCAGAGCT

CGTCGTTGCCAAACTCAGAGTCAGCCCATCCCCCGCCACCCAGAGCGCGTCGGCGCTAGGACCTAGCG

ACTGCCTTCGACCCAGAGGGCGCCGGCAGAGGCACGCATGCGCGCTGTTCCGGCAGGGGTTGTCGTGG

CGCAGGGGGCGGGACCAGAGGCGGTCACGTGAGGGGCTCTGGGCTACCGGGTCACGTGACCGAGGC

ATGACTGCGCCGGCAGGTCCACGAGGGTCTGAAACAGAA

CGGCTCCTTACTCCCAATCCCGGCTATGGGACACAGGCCGGTCCTAGCCCTGCCCCACCCACACCCCC

TGAGGAGGAAGATCTGAGGAGACGGCTCAAGTATTTCTTCATGAGCCCGTGCGATAAATTTCGAGCTA

AGGGACGAAAGCCGTGCAAGCTGATGCTCCAGGTGGTAAAGATTCTCGTAGTTACTGTGCAACTCATC

CTTTTCGGTCTGTCCAATCAGTTGGCAGTCACTTTCAGAGAAGAAAACACTATCGCTTTTCGGCATCT

GTTCCTGCTGGGTTATAGTGACGGTGCAGATGACACCTTCGCCGCCTACACTCGCGAACAACTTTACC

AGGCTATTTTCCACGCGGTAGATCAATATCTTGCGCTCCCCGACGTTTCATTGGGGCGGTACGCCTAT

GTTCGCGGAGGAGGCGATCCCTGGACCAACGGCAGTGGTCTCGCATTGTGCCAACGATACTACCACCG

GGGGCACGTAGATCCCGCCAACGACACCTTCGACATAGACCCGATGGTAGTAACAGACTGTATTCAGG

TGGACCCCCCCGAGAGGCCACCTCCACCACCTTCCGATGACCTGACGCTGCTCGAGAGCAGCAGTTCT

TATAAGAATCTTACCCTTAAATTCCATAAATTGGTAAATGTAACCATCCATTTTCGCCTCAAAACTAT

CAACCTGCAAAGCCTCATAAATAACGAAATTCCGGATTGTTACACTTTCTCTGTTTTGATAACATTCG

ACAATAAGGCCCACAGCGGAAGGATTCCGATATCCCTTGAAACACAAGCACACATACAAGAATGCAAA

CACCCTAGTGTCTTCCAGCATGGCGATAATAGTTTTAGACTGCTGTTCGATGTAGTTGTGATCCTTAC

ATGCTCACTGTCATTCCTCCTTTGTGCACGATCACTTCTTCGGGGTTTCCTGTTGCAAAATGAGTTTG

TGGGATTTATGTGGAGGCAACGAGGACGCGTCATATCTTTGTGGGAACGGCTTGAGTTCGTCAATGGA

TGGTACATACTTTTGGTTACATCCGATGTGTTGACTATATCCGGTACAATTATGAAAATTGGCATTGA

AGCGAAGAACCTCGCCAGTTATGACGTTTGCAGTATCCTTCTGGGAACGTCCACCCTCCTCGTATGGG

TAGGAGTCATACGGTACCTGACTTTCTTCCATAACTATAATATACTGATCGCAACCCTTCGAGTCGCG

CTTCCGAGCGTGATGAGGTTCTGTTGCTGCGTAGCTGTTATCTACCTGGGATACTGTTTCTGTGGATG

GATCGTACTCGGCCCGTATCACGTAAAGTTCCGGTCCCTTTCTATGGTCTCCGAATGTCTTTTCAGTC

TCATCAACGGTGACGATATGTTCGTCACCTTTGCTGCGATGCAAGCTCAACAAGGGAGGAGTAGTCTG

GTTTGGCTTTTTAGTCAACTGTACTTGTACTCCTTTATCAGTCTTTTTATTTACATGGTGCTCTCATT

GTTCATCGCACTTATCACGGGCGCCTACGACACGATTAAACATCCGGGTGGAGCCGGCGCCGAGGAAA

GTGAGCTCCAAGCCTACATAGCGCAATGTCAAGATTCCCCGACCTCAGGCAAGTTCAGGAGAGGCTCC

GGAAGCGCCTGTTCTTTGCTTTGCTGTTGCGGGAGAGACCCATCCGAAGAGCACTCCTTGCTGGTCAA

TGACTATAAAGATGACGACGATAAATA

The PPT1 protein sequence is:

	(SEQ ID NO: 12)
	MASPGCLWLLAVALLPWTCASRALQHLDPPAPLP

	LVIWHGMGDSCCNPLSMGAIKKMVEKKIPGIYVL

	SLEIGKTLMEDVENSFFLNVNSQVTTVCQALAKD

	PKLQQGYNAMGFSQGGQFLRAVAQRCPSPPMINL

	ISVGGQHQGVFGLPRCPGESSHICDFIRKTLNAG

	AYSKVVQERLVQAEYWHDPIKEDVYRNHSIFLAD

	INQERGINESYKKNLMALKKFVMVKFLNDSIVDP

	VDSEWFGFYRSGQAKETIPLQETSLYTQDRLGLK

	EMDNAGQLVFLATEGDHLOLSEEWFYAHIIPFLG

	DYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for PPT1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

PPT1-DNA

(SEQ ID NO: 13)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG TGGTCTGGCACAATGGCTCACAGCTGTGAT

CCTAGAACTTTGGGAGGCTGAGGGGGGCGGATTGCCTGAGCTCAGGAGTTCGAGACCAGCCTGGGCAA

CATGGCGAAACCCTGTCTCTACAAAAAACACAAAAAATTAGCGGGGCATGTTGGTGGTGTGTCTGTAA

TCCCAGCTACTCGGGAGGCAGAGGCACGAGAATCGCTTGAACCCGGGAGGTGGAGGTTACAGTGAGCC

AAGATCACGCCACTGCACTCCAGTCTGGGGGACAGAACAAGACCCTGTCTCAAAAAAAAAAAAAAATT

GCATTCAGATTGCTTCACAAGTGTCTTTCAGTTATTGCTCCAGTTTAAAGAAACCTTTAAAGAAATTA

TAAATAGTTCATTAAAGGCAGGAAACTTCATTGAGCGGGCTATATTCACAGAAAAATCCTGAAACTAC

TTCAATTTGTAAGTCTTAAGTTAAAATAAAATGCTTATACAATAAGCTTATTTTTCTTAAAACAAAAC

TGTATACTGTACTGTATAATGCTTTATGATTCTGCATTTTTTTTTCTTTGAGACAGAGTTTCACTCTT

GTTGCCCAGGCTGGAGTGCAGTAGCTCAATCTTGGCTCACTGCACCCTCTGCCTCCTGGGTTCAAGTG

ATTCTCCTGTCTCAGCCTCCTGAGTAGCTGGGATTACAGGCACCCACCACCATGCCCGGCTAATTTTT

TGTATTTTTAGTAGAGATGGGGTTTCATCATGTTGGCCAGGCTGGTCTCAAACTCCCAACCTCAGGTG

ATCCACCTGCCTCAGCCTCCCAAAGTGCGCCCAGTGGATTCTGCATTTTAATCGACTTTTCAACTAAC

AACTTACCTCTAGTCCCAATGGCATGGAATAGAAGAGATTACAGACCGTGGGTTTGTAATCGACTTTT

CTTTTACAACATATTAAGGGCATTTTTCATGTCACTTTATGTAAATTTACCTCATTTTTCTTTTTTGA

GAGAGACAGGGTCTCACTGTTGCCCAGGGCAACAGCGAGATCATAGCTCAGTGCAATGGCGAGATCAT

AGCTCAATGCAGCCTTGAACTCCTGGGCTCAAGTGATCCTCCCACCTTAGCCTCCCCAGTAGCTAGGA

TTACAGGCACATACCACCACCCCTGGCTAATTAAAAAAAAATTTTTTTTTTGTAGAGATGGGGTCTTG

CTATGCTGCCTGGGCTGTACTCTCTTTTTATTTATTTTTTATTCTTATTTTTTGAGACAGAGTTTTGC

TCTTGTTGCCCAGGCTGGAGTACAATGGTATGATCTCAGCTTACCGCAACCTCTGCCTCCCGGGTTCA

AGAGATTCTCCTGCCTCAGCCTCCTGAGTAGCTGGGATTACAAGTATGCGCCACCACACCCGGCTAAT

TTTGTAATTTTAGTAGAGACGGGATTTCTCCATGTTGGTCAGGCTGGTCTCGAACTCGCTACCTTAGA

TGATCTGCCCACCTCAGCCTCCCAAAGTGCTGGGATTACAGGAGTGAGCCACCGCGCCCGGCCCTATA

CTCATTTTTTATAAAACTGGCCTCATATTATTCCATTGTATAGTGGATACACTCTAATTTCTTTATAA

ATTATTTAAACTATTTTTTGATGTTTTGTTGAAAAAAAACTTGAGCAGGGAACTGATATTATGAAGGT

GGATTGAGGGATGAATGTGGCAATAGAGCATAAAATGAATTAAAGAGGAGAGAATGGAAAAGGAAATG

GTTGATGAGAGACCCTTGAAATAATCCAGCTGAGATTAGGCCAGTTAGTGGCCATGGGTTTCCCCCTC

ACTCTTATGCATCCAACATATGCTTTCGTCCGTCTTGAAGTCCCGACATTTCTAGAAAACACAGTTGT

CTTTCTCCTCTCTACCTTAGCTCATCTCAGTCCTTCTGAATTTCATCTTTGCTTGTTGAAATCCTACC

TCTCCTTCAATATCCAGAACAAACCCCACACCCTGCCATGATTGGTCCTGGCTGGAAACCGTCCCTCC

TTCTTTCTTCCTTCCTCTGTGCATGTTTCTTGCAAAGGCAGTACAGGGCAGTGGTGAAGAGCAACGAC

TTTGGAACCACCAATTTGCTATCTCTGTGACCTTGGGCACCTTACTCAGTGCCAATTCGCAGTGCCTC

AGTGTCCTCACCTGTCAGGGGGAGAAGTGCATACAGCATTGTTATGAAGAGTGAATTAGTTAAAGGTT

GTAAGAATAGTGCCTGATAGCATCTGATCAGCACTGAATATATGTTAGCAATTATTTTGATTCACCGC

AGAGGGCGGTCTACGAGAGCGCAGAGCCCCACTCGGCCAGCGGGGTCTGGCGGGGGACCTGTCGCGCT

GAAAGCTCCAGGGTAGGGCCGACGCCCATCAGGCTGGGCATCCGTTCGGGATGCGCAGGTTGCGATCT

GCAACCGGCGGCGCCACGCCCAGGCGGGCGGAGCGCGGTTCCCGGAGTCTCGCGCCCGCGGTCA

ATGGCGTCTCCTGGTTGTCTTTGGTTGCTTGCCGTCGCGTT

GTTGCCTTGGACATGTGCTTCTCGGGCTTTGCAGCACCTCGATCCCCCAGCCCCATTGCCTCTTGTAA

TCTGGCATGGCATGGGTGATTCATGCTGTAATCCCCTCAGCATGGGGGCCATAAAGAAAATGGTAGAG

AAAAAGATCCCAGGCATATACGTACTCTCACTTGAGATAGGGAAGACCTTGATGGAAGACGTTGAGAA

CTCTTTTTTTCTCAATGTTAACTCCCAAGTCACTACGGTATGCCAGGCGCTGGCGAAAGATCCTAAAC

TTCAACAGGGGTATAACGCCATGGGATTCTCTCAAGGTGGGCAGTTCCTTAGAGCGGTCGCCCAGAGG

TGTCCATCCCCCCCAATGATTAATTTGATCAGTGTGGGTGGACAACATCAAGGCGTGTTTGGCCTCCC

CAGATGTCCAGGTGAGTCATCTCATATCTGTGATTTCATCCGGAAGACATTGAACGCAGGTGCGTATT

CAAAGGTAGTGCAGGAGAGGCTGGTGCAGGCTGAGTACTGGCACGACCCTATCAAGGAGGATGTCTAC

CGAAATCACTCAATTTTCCTTGCAGACATCAACCAGGAGCGAGGCATTAATGAAAGCTATAAAAAGAA

TTTGATGGCTTTGAAAAAATTTGTTATGGTCAAGTTCCTCAATGACTCAATAGTCGATCCGGTGGATT

CCGAGTGGTTCGGGTTTTATCGGTCAGGTCAAGCGAAAGAAACAATTCCCCTTCAAGAAACGTCACTT

TATACGCAGGACCGACTCGGCCTGAAGGAAATGGATAACGCTGGGCAACTGGTATTTTTGGCTACCGA

GGGCGATCACCTGCAGTTGAGCGAGGAATGGTTCTACGCTCACATCATACCCTTTCTTGGGGACTACA

AGGACGACGATGACAAGTA

The SMPD1 protein sequence is:

	(SEQ ID NO: 14)
	MPRYGASLRQSCPRSGREQGQDGTAGAPGLLWMG

	LVLALALALALALALSDSRVLWAPAEAHPLSPQG

	HPARLHRIVPRLRDVFGWGNLTCPICKGLFTAIN

	LGLKKEPNVARVGSVAIKLCNLLKIAPPAVCQSI

	VHLFEDDMVEVWRRSVLSPSEACGLLLGSTCGHW

	DIFSSWNISLPTVPKPPPKPPSPPAPGAPVSRIL

	FLTDLHWDHDYLEGTDPDCADPLCCRRGSGLPPA

	SRPGAGYWGEYSKCDLPLRTLESLLSGLGPAGPF

	DMVYWTGDIPAHDVWHQTRQDQLRALTTVTALVR

	KFLGPVPVYPAVGNHESTPVNSFPPPFIEGNHSS

	RWLYEAMAKAWEPWLPAEALRTLRIGGFYALSPY

	PGLRLISLNMNFCSRENFWLLINSTDPAGQLQWL

	VGELQAAEDRGDKVHIIGHIPPGHCLKSWSWNYY

	RIVARYENTLAAQFFGHTHVDEFEVFYDEETLSR

	PLAVAFLAPSATTYIGLNPGYRVYQIDGNYSGSS

	HVVLDHETYILNLTQANIPGAIPHWQLLYRARET

	YGLPNTLPTAWHNLVYRMRGDMQLFQTFWFLYHK

	GHPPSEPCGTPCRLATLCAQLSARADSPALCRHL

	MPDGSLPEAQSLWPRPLFCDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for SMPD1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

SMPD1-DNA

(SEQ ID NO: 15)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG AGCACTTACTAGCTAGCTGTGGGATCTTGA

GCCACTTACTTAATCCTACTTTCCTTCTCCGTAAAATGGGGATAATTATAATACCGACTTCCAAATTT

GTTGTGAGGATTAACGTAAGGCAGAGTCCTCAGAAAAGTACCTACAGAGGGTTATTCGGGCCAAGGGC

CAAGGTTGAGGACTGCGACCCAGGACACACTTGCAAGTTGTCTTGGGGGAGCACTCTGGACAACAAAA

GAGAGGCTCATATTTTCAAAGAAAAAAAGATGAATTAGGAGAGGAAATGAATACAAAAGTTGTTCATC

AGGAATTCTCATTGTTTACAGAAATAACACTGGTTATACATTGTTTAGCGCTTGGCTATACATTGTTG

AACTATAAGGTGTATGGCATTTAATGGCTACTTGGCATAGCAAGTGGTTTCAAGAGATAATTACTTAG

CTCAAAGAAGAGTGAGAGTGACTGTTGCTACATTTTAAATGCCTGTCTGGGCCTGATAATTTAAAGGG

GCTCATCTTCCTTAGATAAAAAGGTTTTGTTTTGTTTTGTTTTTCATAGTACATAATCTTAAAAATGC

ATGCTAATTATTACTGTGAGTACTCTATCTGAGATGATGTCCCCAGAACTGGGGGGAAAAAACACTTG

TAAAAAATTATCAAAGGAAAGAACTGACAGGACTTGATAGCTCTGAGGATTCCTCAGAAGCACTCGTC

AAACAGCTTAGGGTTTAATTGCTATTCTGCCACTTAACTTCTTTGTGCCTCATCCTATCTGTAAAGGA

GGGTAATAATAGTATTTGCAGGGACCAAGGGAAACTTTCCCTTCGCCCTCTGAAATTCTGCTGAAAAA

TCAACCCACAAAAAACAGATTAATTGAAGAAAAGGCATAGAAATTGTATTAATGTGTCCACGGGGAAC

CACAGAGTGACTACCCACTTCCCAGACGAGTTCAGAAGCTTAGGTGTCATTCAGGCAAAGGGGGTTGT

GGGAGTGAGGGGAAGAGGAATTCTGCTGAGGGAGAAGGAATGGATCAGGGAACAGATTAACTAGCACA

TTATCATGTGAAGGGGTCTGTTCAGGTGTGATTATATTCTTGGTTTTGTAGGGAGGGGAAGAAAAAAA

CCGTTGTTCCTTTTGATGGGTCTGGACGCTAGGTAACGGCTTTGGGAGAGAGGGCGCGGGAGTAGGAA

GGCCAGACACACCTTAAGGCTTAACTTAGTAAGTCAAACCACCATATTTTGGGTATGGATTTCTGAGC

CCCAATATAATAGGTACTTCTTAGGGTTGTTGTGAGAATTGAAGATTATTAATTATAAAGTGCCTGGG

TTGGTGCTTGGCACATCATAAACAACTCCATAAATGCTCGCTTTTACTGTCGTGGATACACGGAGGTG

GGAGACCCAATTGGGTTCCCGCCCATGTTATTGGCCCCCACGGCTATATGGTCTTTACGGCTTCCTAG

TCTCTTCACCATCTCTAAGGTTTGTTTCCTCATCTGAATCACAGGTGAGTGATTCTCAGGGTGACTCA

AGAGGGCTGGTGCCAGTGGCAGAGCATGGGGAATCGAGGGAAGGCGCCTTCCTGTCAGAGGCAACACA

CCCAGTGGGAGGATCCTGCCCGCCCTGCTCTCTCCTACCTCCCCTTCCCGGGCTAAACGCTGGGGTCG

GTCTGGCAGCTATTCCGGGAATCTGAGCGCGGATTCTGACAAAGGAGACGTCTTCCACCGACCACATC

ACATGGAGCTCCAAAGCACGAGCACGGCCTCCCGCGGCTGTGACCTCAAGGCGGAGCCCCTGGTGACC

TCAGGGAGAGTCCCCACCCCCGCAGCCCGTGCGCCCGGGGCAGGGCGGGGGCAGGGAGAGGGGGCGGA

ATCGGGGCGGTCCCGGGAGCGCCCCGCCCCCTCCGCCTCCGCAGCGTTGACAGCCGCCCGCCACCGAG

AGATCAGCTGTCAGAGATCAGAGGAAGAGGAAGGGGCGGAGCTGCTTTGCGGCCGGCCGCGGAG

ATGCCAAGGTATGGTGCTAGCTTGAGGCAATCCTGTCCTCG

CTCTGGCAGGGAACAGGGACAGGATGGTACGGCGGGAGCCCCGGGCCTGCTGTGGATGGGGCTCGTGC

TTGCATTGGCGCTCGCCCTTGCTCTGGCGTTGGCACTGTCTGACAGTAGAGTCCTTTGGGCGCCTGCA

GAAGCCCACCCACTGTCTCCCCAAGGGCACCCCGCCCGACTCCATCGAATCGTACCTCGCCTGCGGGA

TGTTTTCGGTTGGGGCAATTTGACCTGCCCAATATGTAAAGGCCTCTTTACGGCCATCAACCTCGGTC

TCAAAAAAGAACCTAATGTCGCTCGCGTTGGCTCAGTTGCCATCAAGCTGTGTAATCTGTTGAAAATA

GCTCCGCCGGCAGTGTGTCAAAGTATTGTGCATCTGTTCGAGGACGATATGGTTGAGGTATGGCGACG

ATCCGTCTTGTCCCCCTCTGAGGCCTGCGGGTTGCTGTTGGGGTCTACATGTGGCCATTGGGACATAT

TTAGTAGTTGGAACATCTCATTGCCGACGGTCCCGAAGCCCCCTCCTAAACCTCCTAGCCCGCCAGCG

CCTGGTGCCCCTGTGTCTCGGATTCTCTTTTTGACAGATCTCCACTGGGATCATGATTATTTGGAAGG

GACCGACCCCGACTGTGCTGATCCTCTTTGCTGCCGCCGCGGTAGTGGACTTCCTCCTGCCTCTCGAC

CAGGGGCGGGCTACTGGGGGGAGTATAGCAAGTGCGACCTTCCCCTCCGAACCCTCGAGTCACTGTTG

TCAGGGCTCGGCCCCGCAGGACCTTTTGACATGGTTTACTGGACAGGAGACATACCAGCACACGACGT

TTGGCATCAGACTCGGCAGGATCAGCTGCGGGCGCTCACTACTGTTACAGCGTTGGTTCGCAAGTTTC

TGGGTCCCGTCCCAGTGTACCCCGCTGTTGGAAACCACGAGAGTACGCCGGTGAACAGTTTCCCCCCT

CCGTTTATAGAAGGAAATCACTCCTCTCGGTGGTTGTACGAAGCGATGGCAAAAGCCTGGGAGCCGTG

GCTCCCTGCAGAAGCATTGCGGACATTGAGAATTGGCGGGTTTTACGCCCTGTCCCCTTATCCAGGCT

TGCGGCTCATATCTTTGAATATGAACTTCTGCTCTCGGGAGAATTTCTGGCTTCTGATCAATTCTACC

GACCCTGCAGGACAACTTCAATGGCTTGTAGGCGAACTGCAAGCGGCCGAAGACCGCGGTGACAAGGT

GCATATAATCGGCCACATTCCGCCTGGGCACTGCCTTAAAAGTTGGAGTTGGAACTACTACCGGATCG

TTGCACGGTATGAAAACACTCTTGCAGCGCAGTTCTTCGGGCATACGCATGTTGACGAGTTCGAAGTA

TTTTACGACGAGGAAACTTTGAGTAGACCACTCGCCGTTGCATTCTTGGCGCCGTCAGCTACAACTTA

CATAGGTCTGAACCCAGGGTACAGAGTATATCAAATAGACGGGAATTACAGCGGCAGTAGCCATGTAG

TCCTGGACCACGAAACATATATACTCAACCTTACCCAGGCAAACATTCCGGGCGCGATTCCTCACTGG

CAACTGCTGTACCGCGCACGGGAAACTTATGGTCTCCCCAACACACTTCCGACAGCCTGGCATAACCT

CGTGTACCGCATGAGAGGGGACATGCAGCTTTTCCAGACATTTTGGTTTCTGTACCATAAGGGGCACC

CCCCTTCTGAGCCTTGCGGTACACCCTGCCGCTTGGCCACCCTTTGTGCTCAACTCTCCGCACGGGCG

GATAGCCCTGCATTGTGTAGGCATTTGATGCCAGATGGGAGCTTGCCTGAAGCCCAAAGTCTGTGGCC

ACGGCCCCTGTTCTGTGATTACAAGGATGATGACGATAAGTA

The HEXB protein sequence is:

	(SEQ ID NO: 16)
	MELCGLGLPRPPMLLALLLATLLAAMLALLTQVA

	LVVQVAEAARAPSVSAKPGPALWPLPLSVKMTPN

	LLHLAPENFYISHSPNSTAGPSCTLLEEAFRRYH

	GYIFGFYKWHHEPAEFQAKTQVQQLLVSITLOSE

	CDAFPNISSDESYTLLVKEPVAVLKANRVWGALR

	GLETFSQLVYQDSYGTFTINESTIIDSPRFSHRG

	ILIDTSRHYLPVKIILKTLDAMAFNKFNVLHWHI

	VDDQSFPYQSITFPELSNKGSYSLSHVYTPNDVR

	MVIEYARLRGIRVLPEFDTPGHTLSWGKGQKDLL

	TPCYSRQNKLDSFGPINPTLNTTYSFLTTFFKEI

	SEVFPDQFIHLGGDEVEFKCWESNPKIQDFMRQK

	GFGTDFKKLESFYIQKVLDIIATINKGSIVWQEV

	FDDKAKLAPGTIVEVWKDSAYPEELSRVTASGFP

	VILSAPWYLDLISYGQDWRKYYKVEPLDFGGTQK

	QKQLFIGGEACLWGEYVDATNLTPRLWPRASAVG

	ERLWSSKDVRDMDDAYDRLTRHRCRMVERGIAAQ

	PLYAGYCNHENMDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for HEXB and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

HEXB DNA

(SEQ ID NO: 17)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG GGAATGAAGTCCTCAAGAACCTGCCTGTCC

AAGGATTCCTAAGGAAGCTGTTTATCCCGCTGTTTATCCCCAGAGGGTAAACTGTGTGGCTGGTTGCA

GTATCTGTTCCAGACACATAACAGCCAGTCCCAAAAGCGCAGCTGAAACTCTGTTTCCTTTAACCATT

CAGACATTTTTAAATGCATATTCTAAGAATTATTAATAAATGCAAATGAATGATTTGCCACAGATTCA

TGGGCTAAAATACTAACCTGCACCTCCCCAACCAGATGTGAGCCCCACTATAAGACTGCACAGAAAAC

CACAAAGGCAGGGGCATGACACATATACCTCATACCATTAGGTGATGCAACTAGTTTTCTTTCCTTTT

TGGAATATAATTTTTTATGATGAGTAAACATGGCTGAGTGGAAGAAAGGAAGCAACAAAGTCCTAGAG

CTCCAGTGTTAGGAAGACTTTACAAAGCACCCAGTAATTTCCACAGTGACTTGAGATATAAAAAATGG

TGAGGAGCCAGCTTCACTCCCACCTCAAGCTAAGCATCTACTGTCTACTCCATTCTAACCAATGCCCA

GTGTTTTGGAGAATGACCATATGAACCTATGGGAATGCATCAGGTTTCCAAAGGGTTAAAACATTCAT

TAGCATTCTCAATGCTCCACCACTGTAGTTGCTATTTTAATTAGTACATTCAATCTGGAGTATCTGGA

GGAAGACTTTAAACCTAAACCTCAATAGACTGTCTACTTACTCTTGTGAATCTGCCGCCTCTCTCAGA

GCAAATGCTGCCCTGAGTGAAGGAAGAATGAGGCTCCCTGAAACTAGGTGCACAGGCACCAAAATGCT

TACACATTGAGCTGGAATCATGCTAGGCCCAGAACACATTTCCTTGAACATTAAATTCAGGATGGTGC

AAAACAACATTCACAACCAGACTTACACCTAAGAAAACAATTTGCTTAGAAGACCTTAGGATATAGGA

TCTACATCTCTCCGCATCCTGTGTTTATGCTCCTGTATGTTCCAGGTTGGCCCAACAAGCCAGAAGGA

AGAGCCTAGCCTGTCATCTTGGATACCACCCAGATGGAAAAGCACGTCACAGTCAAAATCACAATCAA

CATTCTTGCAACAATGTTTGATTAAAATAAAAAGTAAACTTGGAAACCCAAGAGGAAATACTTGCTTT

CTGGTTTAAAACTGTGCAAACCTCAAAATATCAGCAGAAACCATGTATACAAGTCACTGAACACACCT

TCAACATAAAATGAATCATCTTTCGAGCATTTTTAAGCCCCCAGAGAAATCACATTTGCATACCAAGT

AAGTGGCCAAGAAAATGCACACATGTACTTAACCTACGTCTGCAAAGAAGACAGAGAAAGGGCTGCTA

TTACTGTGAACATCAAAAGGGCTTCAGTTACAGTAACTTCAGTTCACTTCATGCTCCTAACTCAGGCT

CTCTGCCTCCAATGCTTAGCGTCCCGCACCCTTCTCCAGGTTTTGAACCTGATCTCAATCCAATCAAA

AGAGAAACTAAGCAGATTTTTTTCTGGGCATTTACATTACCGGTAACAATAGAACATTATAAAGATCT

TGTTAACAAAAGTAGCTTCTTGCCAGAGTCTTTTTATTAACACTTGCAACGCTATTTAACAAGCTGGC

CATGCCTGTGTGACACCAAACCGCATGTGGATAAATTCAGTGTTTCATCTCCAGGCCCACCAATTCCT

ATCCTTTGTAACTAATAATGTTAACACGACAGATCAAGAAACTAGTTTTTCAGAAAGCAGTTTTCATT

CATTCTCTATAGACATTTCCGCCGCAGTGAACATGCCCGCACTCAGGGTGTCTGTGCATGGCCCCAAC

TTTCCTCCAAGCCGGGGAGGATGCAACGTGGGCCAACCATCCCGCGCGCTCGGCCCTGGAGGCTGCGG

GGAGCGACGCAGCGCGACATTCACCCCCAGGAAAGCGCTGCTTTCCGCAGAGGAGAGAGGCTGAC

ATGGAGCTCTGCGGACTGGGTCTGCCTCGCCCGCCTATG

CTTCTCGCGCTTCTGTTGGCCACTCTTCTCGCTGCGATGTTGGCTTTGTTGACACAGGTTGCTCTCGT

AGTCCAAGTGGCAGAGGCCGCCAGGGCACCGTCCGTGAGCGCTAAACCCGGGCCCGCTTTGTGGCCGC

TGCCTTTGTCTGTTAAAATGACTCCAAATTTGCTCCACCTTGCCCCGGAGAACTTTTACATAAGCCAT

TCTCCCAACAGTACCGCAGGGCCGTCATGCACTCTTTTGGAGGAAGCATTCCGCAGATACCATGGATA

TATCTTTGGTTTTTACAAGTGGCACCACGAGCCGGCCGAGTTCCAAGCTAAAACCCAAGTGCAGCAAC

TGCTCGTATCTATTACACTCCAAAGTGAGTGTGACGCCTTCCCCAATATTAGTAGTGACGAGTCATAT

ACCCTCCTCGTTAAGGAGCCCGTTGCTGTTCTGAAAGCTAATCGGGTCTGGGGTGCTCTGCGAGGCTT

GGAGACTTTCAGCCAACTGGTGTATCAGGACTCATATGGCACCTTTACGATTAATGAGTCTACGATCA

TTGATAGTCCCCGGTTCTCCCACCGCGGTATATTGATTGATACATCCCGGCATTACTTGCCAGTCAAA

ATCATTCTGAAAACGCTGGACGCTATGGCGTTTAACAAATTCAACGTTCTTCATTGGCATATAGTAGA

CGATCAATCATTCCCTTATCAGAGCATTACCTTTCCAGAACTGTCTAACAAGGGATCTTACTCACTCT

CACATGTGTACACGCCTAATGACGTTAGAATGGTGATTGAATACGCGCGATTGAGGGGTATTAGAGTT

CTCCCGGAGTTCGACACACCTGGCCATACTCTCAGCTGGGGCAAGGGTCAAAAGGACTTGCTTACACC

TTGTTATTCCAGACAGAATAAACTTGATTCATTCGGTCCCATTAACCCTACGCTGAATACTACATATA

GTTTCCTTACTACGTTCTTTAAGGAGATAAGTGAGGTTTTTCCCGACCAATTCATTCATTTGGGAGGT

GACGAGGTGGAATTTAAGTGCTGGGAAAGCAATCCAAAGATTCAAGACTTTATGCGGCAGAAGGGTTT

TGGCACCGACTTTAAGAAACTTGAATCCTTTTACATTCAGAAAGTTCTGGACATAATCGCCACAATTA

ACAAGGGTAGTATCGTGTGGCAGGAGGTATTCGATGACAAAGCTAAACTGGCCCCGGGAACTATCGTA

GAAGTCTGGAAGGATTCCGCTTACCCGGAGGAACTGAGCCGCGTCACAGCGAGTGGCTTTCCTGTGAT

ATTGTCAGCACCTTGGTATCTGGACCTTATCTCCTATGGGCAAGACTGGCGCAAATATTATAAGGTTG

AACCTCTGGATTTTGGCGGGACTCAAAAACAAAAACAACTCTTCATCGGTGGCGAAGCCTGTCTGTGG

GGTGAATACGTAGACGCTACGAATCTCACTCCCAGACTCTGGCCACGAGCCTCTGCTGTCGGGGAACG

ACTGTGGTCTTCAAAAGATGTAAGGGATATGGACGACGCCTATGACCGGCTGACTAGACACAGATGTA

GAATGGTAGAGAGGGGAATTGCTGCACAGCCGCTTTACGCCGGGTATTGCAATCATGAGAACATGGAT

TACAAAGACGATGACGACAAATA

The SGSH protein sequence is:

	(SEQ ID NO: 18)
	MSCPVPACCALLLVLGLCRARPRNALLLLADDGG

	FESGAYNNSAIATPHLDALARRSLLFRNAFTSVS

	SCSPSRASLLTGLPQHQNGMYGLHQDVHHFNSFD

	KVRSLPLLLSQAGVRTGIIGKKHVGPETVYPFDF

	AYTEENGSVLQVGRNITRIKLLVRKFLQTQDDRP

	FFLYVAFHDPHRCGHSQPQYGTFCEKFGNGESGM

	GRIPDWTPQAYDPLDVLVPYFVPNTPAARADLAA

	QYTTVGRMDQGVGLVLQELRDAGVLNDTLVIFTS

	DNGIPFPSGRTNLYWPGTAEPLLVSSPEHPKRWG

	QVSEAYVSLLDLTPTILDWFSIPYPSYAIFGSKT

	IHLTGRSLLPALEAEPLWATVFGSQSHHEVTMSY

	PMRSVQHRHFRLVHNLNFKMPFPIDQDFYVSPTF

	QDLLNRTTAGQPTGWYKDLRHYYYRARWELYDRS

	RDPHETQNLATDPRFAQLLEMLRDQLAKWQWETH

	DPWVCAPDGVLEEKLSPQCOPLHNELDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for SGSH and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

SGSH-DNA

(SEQ ID NO: 19)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG CACAGTCTCGGCTCACTGCAAGCTCCGCCT

CCCGGGTTCACGCCATTCTCCTGCCTCAGCCTCCCAGGTAGCTGGGACTACAGGCACCCGCCACCACA

CCTGGCTAATTTTTTGTATTTTTAGTAGAGACGGGGTTTCACCCTGTTAGCCAGGATGGTCTCCATCT

CCTGACCTTGCGATCCGCCTGCCTCGGCCTCCCAAAGTGCTGGGATTACAGGCTTGAGCCACCGCACC

CAGCCTAATTTTTGTATTTTTAGTGGAGACGGGGTTTCACCATTTTGGTCGGGCTGGTCTTGAACTCC

TGAGGCCTCCATGAGTCATCTCGCTGGCCCCTCCATAACCAGCTCAGTGTCTGTGTGTGCCAAACAGG

CATAGGAGCCTGGGTCGGCCCAGAGACAATCTGCGTCACACACTGTAAACCAAAGGTTCCTTGGGGCC

CAGGGGCTGGAAACCTGGAATCGCTGATGGTGTCTGGGATTGATACTGTGTATTCTGCACTGTTTCTG

AGATATGGCTGGCAGCAGGGTCAGACGCCTCACCTGGGGCGGGAGTCCAGCCACTTCAGCATAGGCCA

GCGCCTGGGGAATGGCAGTGAGGCCAACTGAGAGGCCGGCGACGAAATCCATCTTCAGCCACTGCAGG

GAGTAGCTGGGCAGCCACGCCAGGATGGGCAGCCTCCTCTGCAGGGCCGCAGGGGAGCAGCAGCAGGC

GCTCGGGGCCATCCCGGGGCCAGAGGACCTGGCCTGACCCAGCGCCGTCACCGAAGAAGGCATCTCTA

CGGTGGGGCTGGGGGGTGCAGGTGACACAGACCAGCAGTCAGAGCGTGGCCTTTGAAGAACCCGGCAA

CACTGGTCAGGGTCGGCGGGTGTCAGGAGAGGTCACTATGGGTCTCCCCTCCCCAGGGGGGCGAACGG

CTCTAAGAGGAAGCCAACAATGTGTGCAAGATGGGGACTGGGCCAAGACTGGAAGGCCCACCCCGTCC

CTCATCACCCTCTCTCCCTGAATCCCCCCCGTGCAAATCCTGGGTGCCTTCTGTCCCTCCATCCTCAA

CTCTCCCAGATAAGAAACTGATAAGCGATTCCTCATTGAGAAGTCTGAAGACTAATTAGGCTCAACAC

TTCTGGCCCCTGGAGAACCGACTTCCTTTGCCTTTTTTTCCGTCTCCCCAGGGAATTTAACGGGGAGC

AGCGCAGAGAGAAGCTGGGCCCGCGGGCGCACCGTAGGCTGGCGGGGGGTGCGGGTGGCAGCCAGGAG

ATCCAGCGGGGCTCCGAGGTTTTCCAACTGCGCACCAAGGCGGGCTCCACCCGCTACGGTGGACTGCG

GCGCTCGAGGGAGAGTGGGGGGGTCCTCCGCTCCTGAGGCCACGCCTCGCTCAGCCCCCACCTGTGGC

TTTCACAGCCCTGGAGTTCTAGTTGCTTCTGGGAGGACATCAGAGGCGCCCGGTTAATTCAGGTGCGC

AGCATCTTCCAGGGCCCCGGCGCCCGCGCGCAGAGCCGCCACCCTCACCCCCAGCCCCCCACGCCCCC

GGCCGCTCCGCTCCGGGCAAAATCCACTCCCCGGCCCCCGGCCCCGCCCACAAGGCCGCTGGCTCCGG

ATCCTGGGGTCTCCGGAGGAGACCCTGCGCCACCTGACCGTCCGCGGTCCCTTGGCCCCGAGCAAGGC

CGAGGGCCGCGGGGACCCGGGTAGGGACGCGGCGGGGGAGTGGTCCCCACGGGAGGCCCCCGCACTTC

AGGACTCCGCGCTGGCCCCCGCGTCGGCTGCCCCCGGCGACCCCGGCCACTGTCCGCTCCCCCTGCCC

CTGGTCCCCGCTCGCCGCCCTCGCGGGCCACGGACTCACCCACTCGGCGATGCAGCTTGGGGTCTCCT

GGCGTCGCCGCAGCTGGGTCCGCAGGAATCGCCGGGGACAGGCCGGGGCGGGGTCTGGCGCGCACGTG

ACCGGGGTCGGGGCAGGGGGCGGGGCCGAGCGGGAGACCAGAGAGCCGGAGCCGGATCCCGATCC

ATGTCTTGCCCGGTACCGGCCTGCTGTGCTCTCCTTCTG

GTCCTCGGTCTGTGTCGCGCTCGCCCCCGAAATGCGTTGCTCTTGCTGGCCGACGACGGGGGCTTTGA

AAGCGGTGCGTATAACAATAGTGCTATCGCAACCCCTCACCTTGATGCACTCGCACGCCGGTCTCTCC

TGTTCAGGAACGCCTTTACATCCGTCAGTTCTTGTTCACCTAGTAGGGCTAGTCTCTTGACAGGCCTC

CCCCAGCACCAAAACGGGATGTACGGACTGCATCAAGACGTGCATCATTTCAACAGTTTCGACAAAGT

TAGATCCTTGCCCCTCTTGCTTAGCCAGGCGGGTGTGAGGACCGGCATAATTGGAAAAAAACATGTGG

GGCCTGAGACTGTCTACCCTTTCGACTTCGCTTACACGGAGGAAAATGGTTCAGTCCTGCAAGTAGGG

AGAAACATTACCAGAATTAAATTGCTTGTGAGAAAATTCCTGCAAACCCAAGATGATCGCCCATTTTT

TCTGTATGTCGCCTTTCATGACCCTCACCGCTGTGGTCACAGTCAGCCTCAATACGGTACATTCTGTG

AAAAGTTTGGCAACGGGGAAAGTGGCATGGGCCGGATTCCTGATTGGACACCACAAGCGTATGATCCA

CTGGACGTCCTTGTCCCGTATTTTGTTCCAAACACCCCGGCTGCTCGGGCAGATTTGGCAGCACAATA

TACTACTGTCGGGCGAATGGATCAGGGAGTTGGTCTCGTTCTGCAAGAGCTGCGGGACGCAGGTGTCC

TGAACGATACGCTGGTAATCTTTACCTCAGACAATGGTATCCCGTTCCCATCAGGGAGGACGAACCTT

TACTGGCCTGGTACTGCAGAGCCTCTGTTGGTGTCCTCCCCAGAACATCCCAAGAGGTGGGGCCAGGT

GTCCGAAGCTTATGTTTCTCTTCTTGACCTTACCCCGACCATACTTGATTGGTTTTCCATCCCCTATC

CGTCTTATGCAATATTTGGTTCTAAAACGATACATCTTACCGGCCGATCTCTTCTGCCGGCGTTGGAG

GCGGAACCCCTGTGGGCCACTGTATTTGGTTCACAAAGTCACCATGAAGTCACGATGAGTTACCCGAT

GCGAAGTGTCCAGCACAGGCACTTCCGACTCGTGCATAATTTGAATTTCAAGATGCCGTTTCCAATCG

ACCAGGATTTTTACGTAAGCCCGACTTTCCAAGACCTGCTGAACCGCACAACAGCGGGTCAACCAACG

GGGTGGTACAAAGATTTGCGCCACTACTATTATCGAGCTCGATGGGAGCTGTACGATCGGAGTCGAGA

TCCTCATGAAACTCAAAATCTGGCGACAGATCCTAGATTTGCGCAGCTCCTGGAGATGCTTCGGGATC

AGCTTGCGAAATGGCAATGGGAAACCCATGATCCTTGGGTGTGTGCGCCAGATGGTGTACTCGAGGAG

AAACTGTCCCCGCAATGTCAGCCCCTGCACAATGAGCTTGATTACAAGGACGACGACGACAAGTA

The HEXA protein sequence is:

	(SEQ ID NO: 20)
	MTSSRLWFSLLLAAAFAGRATALWPWPQNFQTSD

	QRYVLYPNNFQFQYDVSSAAQPGCSVLDEAFQRY

	RDLLFGSGSWPRPYLTGKRHTLEKNVLVVSVVTP

	GCNQLPTLESVENYTLTINDDQCLLLSETVWGAL

	RGLETFSQLVWKSAEGTFFINKTEIEDFPRFPHR

	GLLLDTSRHYLPLSSILDTLDVMAYNKLNVFHWH

	LVDDPSFPYESFTFPELMRKGSYNPVTHIYTAQD

	VKEVIEYARLRGIRVLAEFDTPGHTLSWGPGIPG

	LLTPCYSGSEPSGTFGPVNPSLNNTYEFMSTFFL

	EVSSVFPDFYLHLGGDEVDFTCWKSNPEIQDFMR

	KKGFGEDFKQLESFYIQTLLDIVSSYGKGYVVWQ

	EVFDNKVKIQPDTIIQVWREDIPVNYMKELELVT

	KAGFRALLSAPWYLNRISYGPDWKDFYIVEPLAF

	EGTPEQKALVIGGEACMWGEYVDNTNLVPRLWPR

	AGAVAERLWSNKLTSDLTFAYERLSHFRCELLRR

	GVQAQPLNVGFCEQEFEQTDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for HEXA and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

HEXA-DNA

(SEQ ID NO: 21)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG CCATCTCAAAAAAGAAAAAAATAAAGAGAG

ATGGGATTTCCTCGTATTACCCATGCTGATCTTGAACTCCTGGGCTCAAGTGATCCTCCCATCTTGGC

CTTCCACAGTGCTGGAATTGCAGGTGTGAGCCACCATCCCCAGCCTGTGATCCATTTTTAATTAAGTT

TGTGTATGCTGTGAGGTAGGGGATCATAGTTCATTTTTCCCACATAGGGATATCTATTTTCTTCATCA

CCTTTTATTGAAAATAACTTGCCTTCCCCACTAAATTAAATTGGTGTCTTCATTGAAATGGAGTAATA

ATCAAATGGCTCTTTTCCTGGACCCTCTATTCCGTTCCATCTATTTGTCTGCCATTATGCCAAAACCC

CACTCTAGTGTATAGTAAGGGGGGATATTTTGGAAGATCAACAAATTTATCTTGTGTAACTTACTTTT

ATGTATGCATAAGAATATTTGGTTTAAAAATCTGGTTAAGTCTAAAAGAGCTCTTTCAGTAATAATTA

AATAACTGAATCCCTGTAATCCACCATGCTCAGCCCCATTAGTAATTTTTATAGCGTCTTTATTTGGA

TCTAGCTCTTTCATCTTGTGTAACTCTTTTAAGTAAATGACTCCTCCCTTCTGGTACAGGTTGAATAT

CCATAAAAGGGTGCCTTGGCATGTGCTCAGTCCTGAAATTCTACTTTGATCTACTGCTCTCATCTTTG

AAACTGCTCTAAACACTCCCCCAAATTATTGATCCTGTGGGCCTGATTATGAACTTGGCTCTTCTGAC

AATACTTCTCAAATCCTGTACCTTTGGTAACATCTCTCCTGACTGGCAGAGTACCCAGACACTTAATT

AATGCTGATGAAAATTAAGATTTAAGCAAAGAAGAAATTGGTGTAAGAATGCAAGCCTTAGGATCTGA

AACTTGATGACTGTGGGATCTAATTTCTGCTTCTTTTGCTACAAGCTCTGTTTTTGAATAATCAACTT

TCAAAGACAAATTGCTTGATGAATCTCTGTATTGTCAGAATGTTTCTTTGATTTCCCATCAAAGCCAT

GTATGTGTAACTAGTAATTAGGTACCTACGACCTGAATATAAATTATCATTATTTCAGTAGCCCTTTA

TTTGTTTTTGAGAAAGGGTTTCACTATACTGCCCAGGCTAAACTTGAACTCCTTGGTTCAAGTGATCC

TGCCGCCTCAGCCTCCTGAATAGCTGGGAGTAGCTGGGACTACAGGGATGTGTTATCATGCCCAGTGA

AAGCCCTTTATTTTTAAAAGTTCATTTTCCTGATCTTTACATGTATTTTAATACTAAAATTAAATTTA

AAAATTTTAAAAAGTACCTACTAACCAAATAATGCCACCCAATAACAATAGCATTATTTGTATTATGT

TCTGGTAATATAGTGGTGAACAAGGAAGACAGGGTCTCTGATAACTGTGGAATTCTCCTTGATCACTT

CTCAAGCCTCTCTTCCGGATACTATTTCTTGGATCTACAGCTGTCTTCCTTCCCCACTTCTGTAGTTC

AGTCTCTCATTATTCTCTACCTCCAATAAAAAGGGGCCTCCTAACTGGTTTCTTATGCAACTACTCAA

CATGGTATTTCTAAAATGCAGATCTTTCGGGATGATTTTCAATTGCTCAGAGAATAAATTCCTTGACT

TTCTACGTAAGGCCCTGCAGGACCAGGCCCCTGTCATTATCTCTGAGCACTCTCCATCTTGCACCCAA

AGCCCCAGCAATATCTTACTGCTTTCAGTAAATACACACCGGGCTATGTGGGGTTTTGTCTGGGGAAA

GGGGATGTGAGTTGTAACTAAGAGATTTATAGTCAATCTTGAAGGGAAGAGCTCATATTGGAGGCAAT

GGAATGCCTCAAGCTGGCTCTGAAAGTACACATTTTTTCCAGTCATAAGTAGATCACATTTGTGTGAT

CTTGTGATCCTCTCTCTAGCAGAGCCGTGCAGACTACCTACTTCCGGCCCTTTCTTGCCCCACGAAAT

TTCACATCACAACGACTTGTGGTTTTAATCCTCCGTTTTTCTGCTTCTGAAGTTACTTCAGCCTGGCA

AGTCCTTTACCTCCCCGTAGGCCTGGCGAGCTGCATCACAACATTCAAGATTCACCCTAGAGCCATCT

GGGAAACTTTCTTCTCCAGGTCGCCCTGCGTCCTCGCCTCCCCACCCCGTTCTTCTCGAGTCGGGTGA

GCTGTCTAGTTCCATCACGGCCGGCACGGCCGCAGGGGTGGCCGGTTATTTACTGCTCTACTGGGCCC

GTGAACAGTCTGGCGAGCCGAGCAGTTGCCGACGCCCGGCACAATCCGCTGCACGTAGCAGGAGCCTC

AGGTCCAGGCCGGAAGTGAAAGGGCAGGGTGTGGGTCCTCCTGGGGTCGCAGGCGCAGAGCCGCCTCT

GGTCACGTGATTCGCCGATAAGTCACGGGGGCGCCGCTCACCTGACCAGGG

ATGACGTCTAGCAGATTGTGGTTCAGTCTGTTGCTCGCCGCGGCGTTTGCCGG

CCGAGCCACCGCATTGTGGCCCTGGCCCCAGAATTTTCAAACTTCCGATCAAAGATACGTCCTCTATC

CTAACAATTTCCAATTTCAATATGATGTGAGTTCAGCTGCACAGCCGGGATGTAGCGTCTTGGACGAG

GCGTTCCAAAGATACAGAGATCTCTTGTTTGGCTCAGGCTCATGGCCACGGCCGTACCTTACTGGCAA

GCGGCATACACTTGAAAAGAACGTGCTCGTCGTAAGTGTAGTAACTCCGGGTTGTAATCAGTTGCCCA

CGCTCGAGAGCGTCGAAAACTACACACTCACGATTAACGACGATCAGTGTCTGCTCCTTTCAGAGACC

GTATGGGGGGCGCTGAGGGGCCTTGAAACATTCTCACAGCTTGTTTGGAAATCAGCGGAGGGAACGTT

TTTTATCAACAAAACGGAAATAGAAGATTTCCCCAGATTTCCACATAGGGGCCTGTTGCTGGACACCA

GTCGCCACTACCTTCCACTCTCATCCATACTTGATACCTTGGACGTAATGGCCTATAACAAACTGAAT

GTTTTTCACTGGCACCTTGTAGATGATCCATCCTTCCCTTACGAAAGTTTCACTTTTCCCGAGCTCAT

GCGGAAGGGCTCCTATAACCCTGTTACACATATATATACAGCTCAAGATGTAAAAGAGGTGATCGAAT

ACGCACGCTTGAGGGGGATACGAGTGCTCGCTGAGTTTGACACACCAGGTCACACACTTAGTTGGGGG

CCTGGCATTCCTGGGCTTTTGACACCCTGCTATAGCGGATCAGAGCCCTCAGGGACTTTTGGACCTGT

GAATCCATCTCTGAATAATACGTACGAATTTATGAGCACATTTTTCCTGGAGGTCAGCTCCGTGTTTC

CCGATTTCTATCTGCATCTGGGAGGCGACGAGGTGGATTTTACCTGTTGGAAATCCAACCCCGAAATC

CAGGATTTCATGCGGAAGAAGGGCTTCGGTGAAGATTTTAAGCAACTCGAGAGTTTCTACATACAGAC

CCTTCTTGATATCGTCTCTAGCTACGGTAAGGGGTATGTTGTATGGCAAGAAGTATTTGACAACAAGG

TAAAAATACAGCCCGACACCATAATCCAGGTCTGGAGGGAAGATATCCCGGTTAATTATATGAAGGAG

CTCGAGTTGGTTACAAAAGCAGGTTTCCGAGCGCTTCTCTCTGCTCCTTGGTATCTGAACAGGATCAG

TTATGGTCCGGATTGGAAGGACTTTTATATAGTTGAGCCATTGGCATTTGAGGGGACACCTGAACAAA

AAGCTCTTGTCATCGGAGGGGAAGCCTGTATGTGGGGCGAATACGTAGATAATACGAACCTCGTGCCG

CGATTGTGGCCCAGGGCCGGGGCGGTAGCGGAAAGGTTGTGGTCAAACAAACTTACGTCAGATCTCAC

CTTTGCCTACGAGCGATTGTCCCATTTCCGCTGCGAATTGCTCAGGCGGGGAGTCCAAGCCCAACCCC

TCAATGTAGGTTTCTGCGAGCAGGAGTTCGAACAAACCGATTATAAAGATGACGATGACAAATA

The IDS protein sequence is:

	(SEQ ID NO: 22)
	MPPPRTGRGLLWLGLVLSSVCVALGSETQANSTT

	DALNVLLIIVDDLRPSLGCYGDKLVRSPNIDQLA

	SHSLLFQNAFAQQAVCAPSRVSFLTGRRPDTTRL

	YDFNSYWRVHAGNFSTIPQYFKENGYVTMSVGKV

	FHPGISSNHTDDSPYSWSFPPYHPSSEKYENTKT

	CRGPDGELHANLLCPVDVLDVPEGTLPDKQSTEQ

	AIQLLEKMKTSASPFFLAVGYHKPHIPFRYPKEF

	QKLYPLENITLAPDPEVPDGLPPVAYNPWMDIRQ

	REDVQALNISVPYGPIPVDFQRKIRQSYFASVSY

	LDTQVGRLLSALDDLQLANSTIIAFTSDHGWALG

	EHGEWAKYSNFDVATHVPLIFYVPGRTASLPEAG

	EKLFPYLDPFDSASQLMEPGROSMDLVELVSLFP

	TLAGLAGLQVPPRCPVPSFHVELCREGKNLLKHF

	RFRDLEEDPYLPGNPRELIAYSQYPRPSDIPQWN

	SDKPSLKDIKIMGYSIRTIDYRYTVWVGFNPDEF

	LANFSDIHAGELYFVDSDPLQDHNMYNDSQGGDL

	FQLLMPDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for IDS and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

IDS-DNA

(SEQ ID NO: 23)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG AAATAAGATCACATTCTGAGGTACTGGGTG

TTAGGACTCCAACATACAATTTTTGGGGGGAGGACACTAACACCTAAGTCAAAAAGGACCAAAAGACA

TACCATACAAAGCAAAGGCCCTCTGCCCTGCACCTTCCCTCTTCTGTTCTCATTTTAAATGTTTCTAC

TTTCATTTCTTCTAGTAGATTGTCCCTGTCTTTCTAAATACTATGCTATTTCTATATCAACTTTCTAA

TGTTTTCAACTTTAGACATTGCTGACTTCTTCCTATGATAGATGAGATCACTTATCCATTCCTCTTTT

TATGTACATATTTTTAACCTTGGATTTTGGAAATTGATCAAACCTATACAAGATTACAGAAAATAGTA

TAATAAACCCCAGGTGTTCATCAATGAACTTCATTGGTTATCAACTCATGACAAATTTTGTTTCATCT

CTCCCTGCCATTTTCCCTTCTCCTTTTACTATATAGATTTATTGCCATTTTTAAGTTTTCTATTGTTT

ACTTTTGTATCTTTAAGTAATATCCCTAAATCTGTATTTCTCTTTCTATGAACTGGGGACAGCGACTA

TTGATTTATTATTATTATTATTATTATTTTTTTGCGATAGGCTGGGGCAAAGGAGACAATCCTAAATT

GCTAACCCTAAATTAATGTGCCTACATTTCCTGCCATCTCCCTTCCTCCTCCCAGCTTTTTGTCAACT

GTTGCTGCAGGCCAGGTTCCCAAGGAAGCTGACTAAGATGTAGAGTAATGTGCAGGAAGTTTCTTAGG

GAGTACTCTTAGGATCAACACCTGTAGAAGGGGAGGGGAGGGAAGAAATCAAAATTCAACAGACAGAG

AAAGTGGGCAGTCTCAAAAAAGGCTTCAGTTAACCCCACAGGGAGCTCTGAAGCTGAAATGGCCTTTC

AGAGATGTCCCAAGTTGGGGTAAGGGGCTGCACCTTTGTACCTTCAAGTTGAATCACTGATGTGGGCT

TCCTGAGAACAGGGGTGACCTTGGCTGAGGTACCTATCTTTTTCTAAGACAATTTCTGATGAGGGCTA

ACGACAGCTAACAGCTCTCAAGCCAGCTAGGGAAATAAAATTTTTGGTGTTGAAGGGAGATCTGAGCA

GTGCACCACAGCACCATCACAGCTGTATTTGTACCTTCCCATTGTCCATTGTTGTTTAGTCATAATTA

AGTCATCTGGGCTTTGTTATAGATTGATTCTCAAAGTTGAAAATCAAACTGTGTTTGCAGTATTAGGA

CGGTATAATAGTATTCAGTACAGTACAGTGTAGGGCTAGGATTCCATCTCCCCACAATTGCTGCTCTC

TCTGTTTGGCGCACCCTTCTTGCAGACCTCAATGCGGCTTGCTCCCTCACTTGCTTCAAAATTTTGTT

CAAATGCCACTTCACTAGTGAGGCCCTGCCTGGCCACCCCATAGAAAATAACAAGCATGCCCCGCACC

CACGCTTTATTTTTCTCCAGACCGGGTATGCCCACCGACACTCTCTATATTTTTTTATTGTTTTAAGC

TCCACCAAAGCAGGGACATAGAGCCCCAGTGCCACCAACAGCGCCTGGTATACATATTTGCGATACTT

AATAAGAATTAGTTGAAGAAATGAATAGGAAAGGGAGTCCCTTTAGATGGAGGAAGGGACTTTCTCAA

AAGGCCTCATGGAAGGAGAATGGCATTGAGGCTGGCTTACAAGAAGTGTGTCTTTTATAAAAAGAAGG

AAGGGCAGGGCTTCCAGGTGAAAGGCATAGCACAAGCAAAGGCAAAAAGACGGGTAACTGCTAGTTGT

TAAGGGCAGTGGCTGAATCGCTATACCCCTAGCAGTTCTACACCTAGGGGCCTGCGGGCCGCTGGGTC

TGCGAAGCTTGCAAGGCAACTGGCCCGCCCCCGCTCTGCGCCTGTCTCTCGGCCACGCCTATTGCTGC

AGGATGACGCGCACCTCTAGAACCCGCCCCGGAGGGGAGGGACGCAGGGAAGAGTCGCACGGACG

ATGCCGCCTCCTCGGACGGGGCGCGGGCTTCTGTGGCTC

GGTTTGGTATTGTCTTCAGTGTGCGTGGCCCTGGGATCAGAAACTCAGGCGAACTCCACGACTGATGC

ATTGAACGTATTGTTGATAATAGTTGATGACCTGAGACCCAGTCTCGGCTGCTACGGTGATAAACTCG

TACGAAGCCCCAACATTGATCAATTGGCAAGTCATTCCCTCTTGTTTCAGAACGCTTTTGCCCAACAG

GCGGTTTGCGCACCGAGCAGGGTTTCCTTCCTCACAGGCCGCCGCCCTGACACTACCCGCTTGTACGA

CTTTAATTCATACTGGCGCGTCCATGCCGGTAACTTCTCTACGATACCGCAATACTTTAAGGAAAACG

GTTATGTCACTATGAGCGTAGGTAAGGTATTTCACCCGGGGATCAGTTCCAACCACACCGACGATTCT

CCTTACTCTTGGAGTTTTCCTCCATATCACCCAAGCAGCGAAAAGTATGAGAATACAAAAACTTGTAG

AGGCCCCGATGGTGAGCTTCACGCAAATCTGCTTTGCCCAGTCGATGTGCTCGATGTCCCAGAGGGCA

CTCTGCCGGATAAGCAATCCACGGAGCAGGCTATACAGCTGCTCGAAAAGATGAAAACTTCAGCGAGC

CCGTTTTTCCTCGCGGTGGGTTACCACAAGCCGCATATCCCGTTCCGGTACCCTAAGGAGTTTCAAAA

ACTTTATCCGTTGGAGAATATCACACTCGCGCCCGACCCCGAAGTCCCAGATGGACTTCCTCCGGTCG

CTTATAATCCCTGGATGGACATTCGCCAGAGGGAAGATGTACAAGCTCTCAATATCTCTGTTCCATAC

GGGCCCATTCCGGTTGACTTTCAGAGGAAGATTAGACAATCATACTTCGCCAGCGTATCCTATCTTGA

CACCCAAGTTGGACGCCTGCTCTCCGCGCTTGATGACCTGCAACTGGCAAACAGTACCATAATAGCCT

TTACCAGTGACCACGGTTGGGCACTCGGAGAACATGGAGAGTGGGCCAAGTATAGTAATTTTGACGTG

GCTACCCATGTACCATTGATTTTTTATGTTCCCGGCCGGACAGCTAGTCTCCCGGAGGCTGGAGAGAA

ACTGTTTCCATACCTTGACCCCTTTGATTCCGCGTCTCAGCTTATGGAACCGGGCCGCCAAAGCATGG

ACTTGGTTGAACTGGTTAGTCTCTTCCCGACGTTGGCTGGCCTTGCAGGGCTCCAAGTTCCACCGCGG

TGTCCCGTACCTAGTTTCCACGTTGAACTCTGTCGGGAGGGGAAGAACCTCCTTAAGCACTTCCGCTT

TCGGGATCTTGAGGAGGACCCCTACTTGCCAGGTAATCCTAGAGAGCTGATAGCGTACTCCCAGTACC

CACGACCGTCTGATATTCCACAGTGGAACTCCGACAAACCTAGTCTGAAAGACATTAAGATCATGGGC

TACAGTATTAGAACGATTGACTACAGGTATACCGTGTGGGTCGGCTTTAATCCAGACGAATTTCTCGC

GAACTTTAGCGATATCCATGCGGGTGAGCTTTATTTTGTTGATTCCGATCCGCTCCAGGATCATAATA

TGTATAATGATTCACAAGGAGGTGATTTGTTTCAACTTCTCATGCCGGACTACAAGGATGATGACGAC

AAGTA

The NEU1 protein sequence is:

(SEQ ID NO: 24)

MTGERPSTALPDRRWGPRILGFWGGCRVWVFAAIFLLLSLAASWSKAEND

FGLVQPLVTMEQLLWVSGRQIGSVDTFRIPLITATPRGTLLAFAEARKMS

SSDEGAKFIALRRSMDQGSTWSPTAFIVNDGDVPDGLNLGAVVSDVETGV

VFLFYSLCAHKAGCQVASTMLVWSKDDGVSWSTPRNLSLDIGTEVFAPGP

GSGIQKQREPRKGRLIVCGHGTLERDGVFCLLSDDHGASWRYGSGVSGIP

YGQPKQENDFNPDECQPYELPDGSVVINARNQNNYHCHCRIVLRSYDACD

TLRPRDVTFDPELVDPVVAAGAVVTSSGIVFFSNPAHPEFRVNLTLRWSF

SNGTSWRKETVQLWPGPSGYSSLATLEGSMDGEEQAPQLYVLYEKGRNHY

TESISVAKISVYGTLDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for NEU1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

NEU1-DNA

(SEQ ID NO: 25)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG ATCATGGTGAGTGACTCCCCTCTCTGCTGC

TCCACCCCCAACTCCCCAGAGGAACCCAATAACCCCAACGGGTCATGTCTTCCAGACCTCCATCCTGG

GGGCCTATGTCATCGCCAGCGGCTTCTTCAGCGTTTTCGGCATGTGTGTGGACACGCTCTTCCTCTGC

TTCCGTGAGTGACACCCCCAGCCACCTGTCCCCAACCTTAAAGTACTGAGCCGTTCAAGCATTTTTTT

TTTTTTTTGGAGATTTGGTGCTTCTAGAAAGAATGCCTTTGTGAGCTAAACGAACCAGAGGGAGCTTC

TTGTGACATCCTGAGGCCAGGCGTTTAGGGAAGAAGGAAGAGAGTGAGCCCCCAGGCCGGTAGGCTCT

CCAAAGAAGGAGCATTCTGATGGAGAGGTCTCTGCTATCTTCCCTAAGAAATCTCGGGTAGAAGTTAG

CCATTAGAGGCCAGGCATGGTGGCTCACGCCTGTCATGCCAGCACTTTGGGGGACCGAGGTGGGCAGA

TCACCCTGAGGTCAGGAGTTCGAGACCAGCCTGGCCCACATGGTGAAACCCCTCTCTACTAAAAATAC

AAAAAATTAGCTGGGCGCGGTGGCGGGCGCCTGTAATCCCAGCCACTTGGGAGGCTGAAGCAGGAGAA

TCGCTTGAACTCAGGAGGCGGAGGTTGCAGCGAGCCGAGATTGCGCTACTGCACTTCAGCTTGGGCGA

CAGCAAGACTCCGTCTCAAAACAAAGAAAAAAGATTAAAAAAAAAAAAGAGCCATTAGAGAGACTTCC

CAGCAGGTGGCTGTAGCGGCAGGGGATTTTCTTACTCTACAGCATGCATCCATTCAGGGTTTGCCAGT

CTCCTCCTAAGCGACTGACATGCATTTTTTCCGCTAATTCTTATCATCGCTCTATGCAACATAATATT

CCCGACCCCACTTCACAGGAGGAAACTGAGATTACATAACCTGCCCGGGGGTAAGGGTGGTGGTGCAG

CTTGGATTCCAAATCCCGCAGGCAGCTTGCCTCCCAGTCCCCGATTTCCTGGCCCCAGGGTCCGCTCT

AACCGGTCTCTGTTGCGGCTCAGTGGAAGACCTGGAGCGGAACAACGGCTCCCTGGACCGGCCCTACT

ACATGTCCAAGAGCCTTCTAAAGATTCTGGGCAAGAAGAACGAGGCGCCCCCGGACAACAAGAAGAGG

AAGAAGTGACAGCTCCGGCCCTGATCCAGGACTGCACCCCACCCCCACCGTCCAGCCATCCAACCTCA

CTTCGCCTTACAGGTCTCCATTTTGTGGTAAAAAAAGGTTTTAGGCCAGGCGCCGTGGCTCACGCCTG

TAATCCAACACTTTGAGAGGCTGAGGCGGGCGGATCACCTGAGTCAGGAGTTCGAGACCAGCCTGGCC

AACATGGTGAAACCTCCGTCTCTATTAAAAATACAAAAATTAGCCGAGAGTGGTGGCATGCACCTGTC

ATCCCAGCTACTCGGGAGGCTGAGGCAGGAGAATCGCTTGAACCCGGGAGGCAGAGGTTGCAGTGAGC

CGAGATCGCGCCACTGCACTCCAACCTGGGTGACAGACTCTGTCTCCAAAACAAAACAAACAAACAAA

AAGATTTTATTAAAGATATTTTGTTAACTCAGTAAATCTGTGACTCATTCTCTCCACGACGACAGGCG

GCCTTTTTTTTTCCGCGCCCGGCTCTTTAAAATCGGCCCCGCCCCTGTGACGTGTCCTGGTGCCGCGG

CCAATCAAAAAGCCCAATAAGGATGGTGCCAGTCCCTTCTTCACCCCAGCCAGTCCGCTTCCCGTTCG

GGTCACGCGCTCCGGGCCTCCAAGAGGGCCTCCAAGAGGGCCAATCGGAAGGGCAAGCTTCGAGATGC

TGCGTGATCACGTGGGAGGAGATGAGCTACTTGAAGACCAATTAGAGTCCGGGAAGCGCGGCGGGGCC

TCCAGACCGGGGCGGGCTTAAGGGTGACATCTGCGCTTTAAAGGGTCCGGGTCAGCTGACTCCCG

ATGACAGGCGAACGCCCTAGCACTGCATTGCCTGACAGA

CGGTGGGGCCCTCGAATACTCGGGTTCTGGGGAGGGTGCAGAGTATGGGTTTTTGCGGCCATATTCCT

CCTGTTGAGCCTGGCTGCATCCTGGTCAAAAGCGGAGAACGACTTTGGGCTGGTGCAACCGTTGGTTA

CGATGGAGCAGCTCCTGTGGGTTAGCGGCAGACAGATCGGGTCAGTAGATACGTTCCGAATACCACTG

ATCACAGCCACTCCGCGAGGCACACTCCTTGCTTTTGCAGAAGCCAGGAAGATGAGCTCATCAGACGA

GGGCGCTAAATTCATCGCATTGCGGCGATCCATGGACCAGGGATCTACATGGAGCCCGACGGCATTTA

TAGTGAACGATGGGGACGTGCCGGATGGACTTAACCTTGGAGCGGTTGTATCAGATGTGGAGACAGGC

GTTGTGTTTTTGTTCTACTCACTTTGCGCACACAAGGCGGGCTGCCAGGTTGCTAGCACTATGCTCGT

ATGGTCCAAGGATGACGGCGTGTCCTGGTCTACGCCACGAAATCTCTCACTCGACATTGGTACTGAAG

TATTTGCACCAGGGCCGGGTTCCGGTATCCAGAAGCAACGGGAACCTCGGAAAGGTAGACTTATAGTA

TGCGGACACGGAACTCTTGAGCGGGACGGCGTTTTCTGTCTCTTGTCTGACGATCACGGGGCATCCTG

GAGGTATGGTAGTGGGGTTAGTGGGATACCCTATGGCCAGCCTAAACAGGAGAACGATTTTAATCCGG

ACGAGTGCCAGCCTTATGAACTTCCCGACGGCTCAGTGGTCATAAATGCGCGCAATCAAAATAACTAT

CACTGCCACTGTAGGATTGTGTTGCGGAGTTACGATGCTTGTGATACACTGCGACCGCGAGATGTGAC

ATTCGACCCAGAATTGGTGGACCCCGTAGTAGCGGCGGGGGCCGTGGTCACAAGTTCAGGGATAGTTT

TTTTCAGTAACCCTGCTCATCCCGAGTTTAGAGTGAATCTCACTCTTCGGTGGTCTTTTTCCAATGGG

ACCAGTTGGAGAAAAGAAACGGTACAACTCTGGCCTGGACCGTCCGGTTATTCTAGTCTCGCGACGTT

GGAGGGGTCAATGGACGGAGAGGAGCAGGCCCCGCAACTCTACGTCCTGTACGAGAAGGGGCGGAACC

ATTACACCGAGTCAATAAGTGTCGCTAAAATCTCCGTTTACGGCACATTGGATTACAAGGACGACGAT

GACAAGTA

The SUMF1 protein sequence is:

(SEQ ID NO: 26)

MVPIPAGVFTMGTDDPQIKQDGEAPARRVTIDAFYMDAYEVSNTEFEK

FVNSTGYLTEAEKFGDSEVEEGMLSEQVKTNIQQAVAAAPWWLPVKGA

NWRHPEGPDSTILHRPDHPVLHVSWNDAVAYCTWAGKRLPTEAEWEYS

CRGGLHNRLFPWGNKLQPKGQHYANIWQGEFPVTNTGEDGFQGTAPVD

AFPPNGYGLYNIVGNAWEWTSDWWTVHHSVEETLNPKGPPSGKDRVKK

GGSYMCHRSYCYRYRCAARSQNTPDSSASNLGFRCAADRLPTMDDYKD

DDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for SUMF1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

SUMF1-DNA

(SEQ ID NO: 27)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG ATAGCTGGACCAGGAATCAAAGAAGTTCAG

GTCTCTGAAGCGCAGCTGACACAGCATGTGCTGAGACTGATATTTGAAGGAGGCGAACAAAGATTACC

GTTGACCTAGACCTTGACTATATATTATGCACAGTAAATACATACAACCTTGGTTAAGGCATTGACCT

TTTAGCTTCATCAATTTTGAAATGAGTATAGTAATAATACATTTCTTGCCAAGTGGTTGTAATCTCAA

ATGAGAAAATGTGTGAAAGGCCTTGGCAAACTGTAAAATAGTCAACAAGGAGAAAAGGCCTTTTTTTT

TTTTTTGAGAGATGGTCTCACTTTGTCACCCAGGCTGGAGTGCAGTGGCACAATCATGGCTCACTGCA

GCCTCTGCCTACCAGGCTCAAGTGATCCTCCTGCCTCAGCCTCCGAAGTAGCCAGGACCACCGGTGTG

CGTCACCATGGCCAGTTAATTTTCTTAATTTTTTGTAGAGACAAGGTCTTGCTGTGTTGCCCAGGCTG

GTCTCAAGCTCCTGGCCTCTAGCGATTCTCCTGCCTCGGCCTCCTAAAGTGCTGGGATTACAGGCTTG

AGACACGGTGCCTGGCTGGTCTTTTCTTTATGCTAAAAAAATTCAAACAGATGCTACATTAGAATAGT

GCAATAAACACCCAGATGCCCATCAACCAGCTATGACAGTTATCAACTCATGGCCAATCATGCTTCAT

CTTTAGCCAGACCCTGATTACCCATTTTTTGGGAAGCAAATCTTTGACTGCCTATATTTTTATCCATA

TATATATATGTGTGTGTGTGTGTGTGTGTGTGTGTATACCTCAGTGAGTGTGTGTGTACCTCAGTGAG

GGGATTCTTTTAAAAAATATAGCCACAAATACCATTATTACTCCTAAAAGCCTAAAAACAAAATTTAC

AGCAATTCCTTAATTTTATCAAATATTATAATTTGACTTAGTGGTTAAATTTCCCTGATTATCTCAAA

AATTTTGAACTCAGTAGGTTTGAATTAGTATTTAGATAAGGTTCACATATTGCAGCTGGCTGCTATGT

TTTTTTTTTTTTTTTTTTTTTCAGATGGAGTCTTGCTCTGTCCCCAGGCTGGAGTGCGGTGGCGCCAT

CTCGGCTCACTGCAAGCTCCACCTCCCGGATTCATGCCATTCTCCTGCCTCAGCCTCCCGAGTAGCTG

GGACTACAGGCGCGTGCCACCACGCCGGGCTAATTTTTTGTATTTTTTAGTAGAGATGGGGTTTCACC

GTGTTAGCCAGGATGGTCTCGATCTCCTGACCTCGTGATCCACCCGCCTCGGCCTCCCAAAGTGCTGG

GATTACAGGCTTGAGCCACCGCGCCCGGCCTGCTATGTTGTTTTTTAATCTGTAAGTTTCTCCTTCAT

CTTCTTTTTGTTTTGGTTTTGCATTTTGTTCGATTACGGAACTGTAGAACAGTGGTTCTTAAACTTAA

GTGTGGATTAGAATCATCTGGAGAGCTTGTGAAACCCTGGATTGCTGTGTCCAGTCCCCACGGATTCT

GATTCCGTATTGTATTTCTCTTTTTTTTTTGAGACGGAGTCTTGCTCTGTCGCCGAGGCTGGAGTGCA

GTGGCGGGATCTGGGCTCACTGAAACCTCCGCCTCCCAGGTTCAAACGATTCTCCTGCCTTGGCCTCC

CGACTAGCTGGGACTACAAGCGCGCCATTACTGGGTCTTTGTGAGGATTAAATGAGATTTTAAAAAGT

GAAGTGCTTAGCATAGTACTCTGGCACTTACCAAATGCCCAGCAAATTTGACTGTTATTTTTCAACCA

AGAAATCATAAAGGGGTGCACGGTCTGTGGAAGGCCTTGAGTTTTGTGGAGTTTTGTGTTTTGCTGCG

TTTTGACGTTAGCACAGGCAAGCAGCAGCGCCAAAGAAGTACCTGAAGAAAGGTGAAGAGCAACTCTC

CTGGTCACTTCGTAAGTCAGCTCAAAATTCTGCCCCTTTAAGGAGGGCTCAG

ATGGTTCCAATCCCCGCCGGGGTGTTCACCATGGGGACGGACGACCCACAGAT

TAAGCAGGATGGCGAAGCTCCAGCGAGAAGAGTCACCATAGACGCTTTTTACATGGATGCTTACGAAG

TGTCTAACACGGAGTTTGAAAAATTTGTTAATAGCACAGGTTATTTGACGGAGGCGGAGAAGTTCGGT

GACAGCTTTGTCTTCGAGGGCATGCTTTCTGAACAGGTGAAGACTAACATTCAGCAGGCAGTTGCAGC

GGCACCTTGGTGGCTCCCAGTTAAGGGGGCAAATTGGCGACACCCAGAGGGACCAGACTCTACTATAT

TGCACCGGCCTGACCACCCGGTTCTTCACGTATCCTGGAATGACGCCGTTGCCTACTGCACCTGGGCA

GGAAAACGACTGCCGACCGAAGCAGAGTGGGAGTATTCTTGTAGAGGAGGACTGCATAATCGGCTTTT

TCCCTGGGGTAACAAGCTTCAGCCCAAAGGCCAACACTATGCTAACATCTGGCAAGGGGAATTTCCCG

TCACGAACACTGGGGAGGACGGATTTCAGGGGACCGCACCTGTAGATGCGTTTCCCCCCAACGGGTAT

GGTCTCTACAACATCGTCGGTAATGCCTGGGAATGGACCAGCGACTGGTGGACGGTGCACCATTCTGT

TGAAGAAACATTGAATCCCAAAGGGCCACCAAGCGGAAAGGATCGAGTGAAGAAAGGCGGATCTTATA

TGTGTCATCGGTCCTACTGCTACCGGTATCGGTGTGCTGCTCGCTCTCAGAATACACCCGACTCAAGC

GCAAGCAATCTTGGTTTCCGGTGTGCGGCCGATCGCTTGCCGACAATGGATGACTACAAGGACGATGA

CGACAAATA

The GAT1 protein sequence is:

(SEQ ID NO: 28)

MATNGSKVADGQISTEVSEAPVANDKPKTLVVKVQKKAADLPDRDTWKG

RFDFLMSCVGYAIGLGNVWRFPYLCGKNGGGAFLIPYFLTLIFAGVPLF

LLECSLGQYTSIGGLGVWKLAPMFKGVGLAAAVLSFWLNIYYIVIISWA

IYYLYNSFTTTLPWKQCDNPWNTDRCFSNYSMVNTTNMTSAVVEFWERN

MHQMTDGLDKPGQIRWPLAITLAIAWILVYFCIWKGVGWTGKVVYFSAT

YPYIMLIILFFRGVTLPGAKEGILFYITPNFRKLSDSEVWLDAATQIFF

SYGLGLGSLIALGSYNSFHNNVYRDSIIVCCINSCTSMFAGFVIFSIVG

FMAHVTKRSIADVAASGPGLAFLAYPEAVTQLPISPLWAILFFSMLLML

GIDSQFCTVEGFITALVDEYPRLLRNRRELFIAAVCIISYLIGLSNITQ

GGIYVFKLFDYYSASGMSLLFLVFFECVSISWFYGVNRFYDNIQEMVGS

RPCIWWKLCWSFFTPIIVAGVFIFSAVQMTPLTMGNYVFPKWGQGVGWL

MALSSMVLIPGYMAYMFLTLKGSLKQRIQVMVQPSEDIVRPENGPEQPQ

AGSSTSKEAYIDYKDDDDK

GAT1 DNA

(SEQ ID NO: 29)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG AGGTCGGGTCTGCTCAAGTTCTGCAAAGAT

GGCTGCCAACACCTCTATGCTTAGGAGCTTTGGTGGAGATTGAGTTTCTGTTTGACAATAGTTTTAGC

AAAAGTCCCAGACAGAGTTCTCATTGGCCCAGCATATGTCACACGTCCCCTCATTGAGGTCAGAGTGA

TGTGCTCTGATTATCTGTGCCCTGGGTCACATGTCTGTCTCCAGGAGGAGGGAGCCCCACCCAACCAT

ATTGAATATGAGTGAAGGAGGGGAAGTTTCCCAAATGAAAATTGAGGTTCTGTCACCCAAAGAAGTTG

AAATGAATGTCAGGCAAGCAAAAGTAGCCTTCATTTTTGCTACCCTTGTATCTCCACTCAAAGCTATG

CCCCACATCTCCATCCAGACAGCTCCTACTCATCCTTCAGACCTCAGTTCAAATGGCGCTTCCTTGAG

GAAACCTTCCCAAACCATCCCACACTAGACTGGAATCCCCTGCTGGACTAGAATCCCCTGCATGTGTG

CTCCTGGCAGAGCTTGTCATTCTGGGTTTGTGCGGTTATTTGATGAAAGCCTGTCTCCCATACTGGAC

TGGCATCACCTTGAAAGCTGTGGCCTGTCTTGTCCATCGCTCTGTCCCTGGAGCCTAGGACAGTGCCT

GGCACACAGGTGGAACTCGGGAAATATTTATTTGAAATACCAACGGAAGTCCACAGCACATATGACCT

GGTGATCAGTGGTGGGAGGTGAATGGGTCAAGAAAGACACCAAGATTCCCTGCTCATGAGTGAGAGTG

TGGGGAAACAGAAGAAGTCAGGGTGACCTTGGTGAGCACTGCCCTGGGGGACTCGAGGATGGATGAAA

GCAGGGAGGAGGCCAGATTAGGTGGGGTAGAGGACCTGAGGTCCATCAGCAAAGGCCATCCCTGGGGA

CAAAGAGAGGAGGGGAAGAGTAGGTCTGGGCTGGAGAGAAGGAATCTTTTCTCTGAGTTCCTGGGGAC

CCCAGAGGGAAGGGCAGATGTGGCCCCATTCCCAGAAGCCTTTATAAAAGAGGGTGGGGAGGTCAGGC

CTTGGCAAGGACAAGGGAAGGAGTCCCTACGTGCTGGATTGGGATTGGCTGGAAAGGGGACTTCTCTG

GGAGAATCCCTCAAACCTCAAGAACTGAGAGAAGGGTGTCTGGGGCTCCTGCCACCATCCCTGTTTCC

CTTTTAAGTAATCTGTTTCCCCATCTGTCCATCCATACACACAGCCACTTGTGTCTCCATGACCAACC

GCTGGCAGTGGAAGGGTGTCCTTCCCACCCCCACTCTTACACACACTCCCAGCTGGTACCCAGAGCCT

GGTCACCCCAGGCCAGGCCTGTGTTTCCAGGTGTAACGGGCAGCAGACGCTGCCCTAGGACTAGAGCA

GGGAGGGGGCACGGGCCCACCCCAACCCACAGCGACCCACAGAGGGCGAAAAGAGGACGACCGCAGAG

AGAAACGGAAAGGACAGGCCAACGGAAGCAGTACTGCAAGGCTGGAAGGAGAAAAGCCAGGAGGGGAG

TGCTTGCTGTGAAAGACAGGGAGACAGAGACCAAGACGGACAGGCAGACAGGCTGGTGACCCAGGATG

AGGCCGGAAAGAGCCATCAAAGGAAGGAGAAGGAAGGAGAGAGATTGGAGCGGGACGGCGGGGCAGGC

GAGGGAAGGAGGGGGTGGGGAGAGGGAGGGAGGAAGAGAGGGGAGAAAGAGGGAGGAAGAGAGGGGAG

AAGGAGGGAGAAGAGAGCGGGAGAATGCGAGAGGAAAGAAGGGAGAGGGGAGGCGTAGAAGGGGAGAG

GAGGTGAAGGGAAAAGGAGAGAGCCTGCTGGCGGCGAAGCTGCAAGAGGCAGCTGCGGAGGGAGCGCG

CGGCGGGCCTGGGGGAGCGCTGGGCGGGGGCGGGCGGTGCGGGCAGGGCTATACCCGAGCTGGGCGGG

CTCCGGGCGCCGCGGGCCCTGCCCTCCCCCTCCATCCCTCCGGACTCGCTCCCCCCTCCTCTCCCTTC

CCCGCGACCCTCCGCC ATGGCAACTAACGGTAGTAA

AGTCGCGGACGGTCAGATCTCCACAGAAGTTTCTGAAGCGCCGGTCGCAAATGATAAACCAAAGACCC

TCGTAGTCAAGGTACAGAAAAAAGCTGCTGACCTTCCTGATCGAGATACCTGGAAGGGCAGGTTTGAT

TTCCTCATGTCATGCGTTGGGTACGCCATCGGCCTCGGAAACGTATGGAGGTTCCCCTACCTGTGCGG

TAAGAATGGTGGCGGAGCCTTCCTGATACCATACTTTTTGACTCTCATATTCGCAGGCGTGCCACTCT

TCTTGCTCGAGTGCAGCTTGGGCCAGTACACGTCTATAGGCGGCTTGGGCGTATGGAAGCTGGCGCCC

ATGTTCAAAGGTGTTGGCTTGGCCGCTGCTGTACTGTCCTTTTGGCTGAACATTTATTACATAGTGAT

CATATCCTGGGCAATTTACTACCTCTACAATTCTTTTACGACAACACTGCCCTGGAAGCAATGCGATA

ACCCATGGAATACGGACCGATGCTTTTCCAACTATTCAATGGTAAACACCACGAATATGACGTCTGCG

GTAGTCGAATTTTGGGAACGGAACATGCACCAGATGACCGATGGTCTCGATAAACCGGGTCAGATTAG

ATGGCCACTCGCAATAACTCTTGCCATCGCATGGATACTCGTATACTTTTGCATCTGGAAAGGTGTCG

GATGGACAGGCAAAGTAGTCTATTTTAGTGCTACATACCCTTATATCATGTTGATTATACTGTTTTTC

AGAGGAGTCACACTTCCTGGGGCGAAGGAGGGCATTCTCTTCTATATCACGCCAAACTTTCGAAAGCT

GAGTGACTCAGAGGTTTGGCTCGATGCTGCAACTCAGATATTCTTCTCATACGGCCTTGGTCTGGGTA

GTCTCATAGCCTTGGGTTCCTACAATTCCTTTCACAATAACGTGTACCGAGACAGCATTATAGTATGT

TGTATTAATTCTTGCACTTCAATGTTCGCCGGTTTCGTCATTTTCAGCATTGTCGGATTTATGGCGCA

TGTTACCAAAAGGAGCATTGCGGATGTGGCGGCTAGTGGGCCAGGACTCGCTTTCCTCGCCTACCCAG

AAGCAGTTACTCAGTTGCCGATTAGCCCACTGTGGGCTATCTTGTTCTTTTCTATGCTCCTTATGCTT

GGGATAGATTCCCAGTTTTGCACGGTAGAGGGTTTCATAACGGCACTTGTCGATGAATATCCGCGACT

CCTGAGAAATAGACGCGAACTCTTTATAGCAGCGGTATGTATAATCTCATATTTGATAGGCTTGTCAA

ACATAACACAAGGGGGAATATACGTTTTCAAATTGTTCGACTACTATAGTGCCTCCGGGATGTCACTT

CTTTTCCTGGTATTTTTCGAGTGCGTTTCAATCAGTTGGTTTTATGGCGTGAATCGATTTTATGACAA

CATTCAGGAGATGGTAGGAAGTAGACCGTGCATATGGTGGAAACTCTGTTGGTCTTTTTTCACGCCCA

TCATCGTGGCCGGCGTGTTTATATTTAGCGCAGTGCAAATGACTCCTCTCACTATGGGGAACTATGTC

TTCCCCAAATGGGGCCAAGGGGTAGGCTGGCTCATGGCGTTGAGTTCCATGGTCCTTATTCCAGGTTA

TATGGCTTACATGTTCCTGACACTGAAAGGGAGTCTGAAACAACGGATTCAGGTGATGGTTCAACCTA

GTGAAGATATTGTCAGACCTGAGAACGGTCCCGAGCAACCTCAGGCGGGAAGTAGTACCTCTAAGGAG

GCTTATATAGACTATAAAGACGACGACGATAAATA

The FIG. 4 protein sequence is:

(SEQ ID NO: 30)
MPTAAAPIISSVQKLVLYETRARYFLVGSNNAETKYRVLKIDRTEPKDLVII

DDRHVYTQQEVRELLGRLDLGNRTKMGQKGSSGLFRAVSAFGVVGFVRFLEG

YYIVLITKRRKMADIGGHAIYKVEDTNMIYIPNDSVRVTHPDEARYLRIFQN

VDLSSNFYFSYSYDLSHSLQYNLTVLRMPLEMLKSEMTQNRQESFDIEEDEG

LITQGGSGVFGICSEPYMKYVWNGELLDIIKSTVHRDWLLYIIHGFCGQSKL

LIYGRPVYVTLIARRSSKFAGTRFLKRGANCEGDVANEVETEQILCDASVMS

FTAGSYSSYVQVRGSVPLYWSQDISTMMPKPPITLDQADPFAHVAALHFDQM

FQRFGSPIIILNLVKEREKRKHERILSEELVAAVTYLNQFLPPEHTIVYIPW

DMAKYTKSKLCNVLDRLNVIAESVVKKTGFFVNRPDSYCSILRPDEKWNELG

GCVIPTGRLQTGILRTNCVDCLDRTNTAQFMVGKCALAYQLYSLGLIDKPNL

QFDTDAVRLFEELYEDHGDTLSLQYGGSQLVHRVKTYRKIAPWTQHSKDIMQ

TLSRYYSNAFSDADRQDSINLFLGVFHPTEGKPHLWELPTDFYLHHKNTMRL

LPTRRSYTYWWTPEVIKHLPLPYDEVICAVNLKKLIVKKFHKYEEEIDIHNE

FFRPYELSSFDDTFCLAMTSSARDFMPKTVGIDPSPFTVRKPDETGKSVLGN

KSNREEAVLQRKTAASAPPPPSEEAVSSSSEDDSGTDREEEGSVSQRSTPVK

MTDAGDSAKVTENVVQPMKELYGINLSDGLSEEDFSIYSRFVQLGQSQHKQD

KNSQQPCSRCSDGVIKLTPISAFSQDNIYEVQPPRVDRKSTEIFQAHIQASQ

GIMQPLGKEDSSMYREYIRNRYLDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for FIG. 4 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

FIG. 4-DNA Sequence

(SEQ ID NO: 31)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG GTAGCAGCTGCTATTAGTGCCTCCCCCATA

TCCACTTGACATACACCTTTTCTATGCACGACCAGCCCAACTTCCAAAATGTTAGCATGTGCGACTTT

TTGCCTAAGGGCTTTCTCTGGCTGCCAGAACCTACTCTCCCTGAACACAGGGTAGACTGGAAGTTCTA

GGGCATCAATGTTCTGCCCTATCAGGCTTCATCCAATAACTGATGGAAGTTGGCGAATAAATACTCCA

GCTTCCTTGCCCCTCAAGAGGGGATAACTGAGACATGCCTCCCAGTATTCCCTAGTGAATTCAGCCCA

CAGAGAAATTTCTCTGGTAATATTTCCTTTATTAGTTTTCTTCCCCTCCCTGCTGCTGTTTCCTGGGA

TTACGTCTCATATAAACAACCTGCCTTCAAAGTTTTGTCTTAGGATCTGATTCCGGGTGAAATAAACT

AAGATAAAGTTAAAATGGAAGTGCTAAACTATAATAATTGAAAACTAGAATCCTTCAGGTATTTTAAG

AGTTGCTATCCTAGATAAAAACACTGTGCAAACATTTAACTAATATTCAGAAGAGTTGGTTTCATTAT

GAAACAGAACACAGCTAACTATTTTTCCCAGTTAAAAGATCCAAGTCCAAAATGCCAGAACGGACACG

GCCATCCTTCTAGTTCTTAATAAAAGGAGTACCAAACTACAGGGGGAAAGAAGCGCAAAATAACATAG

ACTTAAGTATTTCTTTACAATTTTTGTAATTTCTCAAATTTTAATTTACAATATGATCTTTGGTATTA

CATGAATGTTATCTGGGTATGCTATAATTCTATAATTTTTTTTTAAAGTTTCCTTACACTTAGCAGTT

GAGAAGCCCTCTAGCAGAGGTTTCTGAGGTTTAGAGGTTTCCTGGGAAACCTAGAATTGCCGCCTAAG

ATAGGATAGCTGCCTCTCCCCTACATCTTATACTCTTGCAGTAGGTGTTCAGTAAATATTTGTGGAAT

AAATACTTCAGTCGTTGAAAGGAGACGTGGTTTTAAAAAGTGGATTTACAAAGAGACCGTTTTGCAAG

AAAAGAAGCTGGTCTGGGGGTCCCCGCAGGAGGCGGGGTTCCGTGCAACAGATACCAAGATGCCTCCA

GGGCGGACCACGACCCAGCGGGCCGCCGGAAGGCTCCTTACCTGGCCGCAGCCGGACCCTCCTCAGGG

AAGCAACTGCCTCCAGGGACTCCGCGGGGCTGTCGGGCGGTTGCCGGGGAGACCTCTGGCCGCCACGC

ACCTGCTGCTTGGTGGGGCGGGGCCTCGAGAGGCGGGGCTGTAGTTCTTTGTCATTTCTTTTCCAATT

CGAAATAGTCACTTTTGTACCTAATTTGGCATTTATAATTATAATACACTCATTTTCACAACTATGGA

AGAGAGGGTACGCGCATGCCCATTTATGTCGCACACATGAAATGAAGAAACCTCATTTCAAGCGCGGC

TCCGCCACACTGCGCTAGTCTCTGTCGACAACTTCAAGTGGCTAGCCTCACGCACAAACGTGTGGCAA

AGCCACACTTTCCAGCTTTAGGTGATTTTAAAACAATTTCTTGGGCCACACAGCGTAGGTACAACATG

GATTAGAGGTGAGGGGAAAACTGACACTCAGTGAAGGACCGTCAGAGGGCCGGCTTCCAGGAAAATGG

GCCGCAGCAGGCAGGGAAAACACAAGGATCTATCCTGCAACTCGGAGAACCCTTTTCAAAAGAAGCTA

CCCAATCCCAAACCTTCCGCTTTTTTCCAAAGAGAGGGAGGAGATAAATAGACTCAATTAATCCGTCG

ACTTTTTCTCCGCCCATGTTTTCCTCCAATCCTTACCAAAGATGGTGCCCTTCGCTTCCTCTCTCGGC

AGCACGCAGGTCCCGGGAGCCTCTACCCGACCTCTCTATGATACGTCCTCCAGCCCCGCTCCCGACGT

GAGGGGCGGGGCTTGCCTGGAGGCGGGGCGCAGGGATCCGGAAACACC

ATGCCCACGGCAGCGGCTCCAATAATCTCTTCCGTTCAAAAGCTTGTTTTGTATGA

AACTCGCGCGCGGTATTTTCTTGTAGGTTCAAATAATGCGGAAACAAAGTACAGAGTACTGAAGATAG

ACAGAACTGAGCCTAAGGACCTTGTCATAATCGATGATAGACATGTTTATACGCAACAGGAAGTGCGG

GAACTCTTGGGCAGATTGGATTTGGGGAATAGAACAAAAATGGGGCAGAAAGGTAGCTCTGGGTTGTT

CCGAGCTGTCTCAGCTTTCGGCGTAGTAGGCTTTGTGAGATTTCTGGAGGGCTATTACATAGTGCTTA

TTACCAAAAGGCGCAAGATGGCGGATATAGGCGGCCATGCGATTTACAAGGTTGAGGATACAAACATG

ATATACATACCAAATGATTCAGTGCGAGTCACCCATOOTGATGAAGCTCGCTACCTGCGCATATTTCA

GAATGTCGATCTGTCTAGTAACTTCTATTTCTCCTATAGTTATGATCTTAGTCATAGTCTCCAATATA

ATCTCACCGTCCTGCGGATGCCCTTGGAGATGCTCAAATCCGAGATGACACAGAACCGACAAGAAAGC

TTCGATATTTTTGAAGACGAAGGTTTGATCACACAGGGAGGTAGCGGTGTATTCGGAATTTGTAGTGA

ACCCTATATGAAGTACGTATGGAATGGCGAGCTCCTGGACATTATAAAAAGCACGGTCCACAGAGACT

GGTTGCTTTACATCATTCATGGCTTCTGCGGCCAGTCTAAGTTGCTGATTTACGGGCGGCCTGTGTAC

GTAACTCTGATCGCAAGGCGATCTTCTAAATTTGCGGGCACCAGATTTCTCAAGCGAGGCGCCAACTG

CGAGGGAGACGTAGCTAACGAAGTTGAGACTGAACAAATTCTGTGCGACGCGTCCGTTATGAGTTTTA

CAGCAGGTTCCTACTCCTCCTATGTACAAGTACGGGGCTCAGTTCCTCTCTACTGGAGTCAAGACATT

AGCACTATGATGCCCAAACCCCCAATTACCCTCGACCAGGCCGACCCGTTCGCTCACGTTGCGGCCCT

GCATTTTGATCAAATGTTCCAACGATTCGGCAGCCCGATTATCATTCTGAATCTTGTGAAGGAGCGGG

AAAAGAGGAAGCACGAGCGGATCTTGTCTGAGGAATTGGTGGCAGCCGTGACATATCTGAACCAATTC

CTGCCACCGGAGCACACGATAGTATATATTCCTTGGGACATGGCTAAGTACACAAAATCTAAATTGTG

CAATGTGTTGGATCGCCTTAACGTGATCGCGGAAAGCGTAGTCAAGAAGACAGGTTTTTTCGTAAACA

GGCCCGATTCATACTGTTCAATTCTCCGACCTGACGAGAAATGGAACGAACTGGGGGGATGCGTGATA

CCTACTGGTCGCCTGCAGACTGGTATTCTCAGGACGAATTGTGTAGATTGCCTCGATCGGACGAATAC

AGCGCAATTTATGGTAGGGAAGTGCGCGCTGGCCTACCAGCTGTATTCACTGGGGCTGATAGACAAGC

CGAATTTGCAGTTCGACACCGATGCGGTGCGCCTCTTTGAGGAGCTCTATGAGGACCATGGAGATACA

CTCAGTCTCCAGTATGGAGGGTCACAACTCGTACATCGAGTTAAAACATACAGGAAGATCGCCCCATG

GACACAACATAGTAAAGATATTATGCAAACCTTGTCCAGATATTATTCCAACGCCTTCAGCGATGCCG

ATAGGCAAGATTCAATCAATCTCTTTCTGGGGGTGTTCCATCCCACTGAAGGTAAGCCCCACTTGTGG

GAGTTGCCTACAGATTTTTACCTTCATCATAAGAACACCATGAGACTGCTCCCAACACGGCGGTCTTA

TACGTACTGGTGGACACCTGAAGTTATTAAGCACCTCCCCCTCCCGTATGACGAGGTGATCTGCGCGG

TTAATCTCAAAAAATTGATTGTGAAGAAATTTCATAAATACGAAGAGGAAATTGATATCCATAACGAA

TTTTTTCGCCCATACGAACTGTCCTCTTTTGATGATACATTCTGTCTTGCCATGACTAGTTCTGCGAG

GGATTTTATGCCTAAAACGGTCGGAATCGACCCGAGTCCATTTACCGTTAGGAAACCTGACGAGACGG

GGAAGTCAGTATTGGGGAATAAGAGCAACCGAGAAGAGGCGGTGTTGCAGCGGAAGACCGCAGCTTCT

GCTCCTCCGCCCCCATCCGAGGAGGCTGTGAGTAGCTCTTCAGAAGACGATTCCGGCACCGACAGAGA

GGAAGAGGGATCAGTTAGTCAGAGAAGCACACCGGTGAAAATGACTGACGCGGGCGACTCCGCTAAGG

TAACTGAAAACGTCGTGCAACCAATGAAAGAGCTCTACGGTATCAACCTCAGCGACGGCCTTTCAGAA

GAGGACTTCTCTATCTACAGCAGATTTGTTCAACTCGGTCAAAGCCAACACAAGCAAGACAAAAATTC

CCAACAACCCTGCTCAAGATGCTCCGACGGCGTAATTAAACTGACTCCGATTAGCGCGTTTTCACAGG

ATAACATTTACGAAGTACAACCTCCACGAGTTGACCGGAAAAGTACGGAGATATTTCAGGCTCACATC

CAGGCCTCTCAAGGAATCATGCAACCCCTCGGAAAGGAGGATTCCTCAATGTACAGAGAATATATACG

CAATCGATATTTGGATTACAAAGATGACGACGACAAGTA

The CLN5 protein sequence is:

(SEQ ID NO: 32)

MAQEVDTAQGAEMRRGAGAARGRASWCWALALLWLAVVPGWSRVSGIPS

RRHWPVPYKRFDFRPKPDPYCQAKYTFCPTGSPIPVMEGDDDIEVFRLQ

APVWEFKYGDLLGHLKIMHDAIGFRSTLTGKNYTMEWYELFQLGNCTFP

HLRPEMDAPFWCNQGAACFFEGIDDVHWKENGTLVQVATISGNMFNQMA

WVKQDNETGIYYETWNVKASPEKGAETWFDSYDCSKFVLRTFNKLAEFG

AEFKKNIETNYTRIFLYSGEPTYLGNETSVFGPTGNKTLGLAIKRFYYP

FKPHLPTKEFLLSLLQIFDAVIVHKQFYLFYNFEYWFLPMKFPFIKITY

EEIPLPIRNKTLSGLDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for CLN5 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

CLN5 DNA

(SEQ ID NO: 33)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG CTTGGTTATGTCAGTTCCTGTGTGTAGACA

GTAAGAAAAAAAAAGCCATGCTATGTGTGACATTTTTTTCCAATGTTTATTTCCCACCAAAAAGTAAA

TGCATTTTAACCCATTCTGTGGTTCACTGTAGCTTAAGGAAACCAAGCATATAGATGCATTAGTGATT

TTGTTTATATTATGTAAAATGTAACGATCTATCTTAGAAATACCACAGTTTGTATTTTTCTTTAAGGA

GTAAAGATTTGTCTTTAAAAATAACTTGATATTTTCCTGGCTTTCATTTAATACAATGGAAAATAAAG

TATTACACGAAAAAAAAATTTTTTCTTAGAGACAGTATCTCATTATGTTACCCAGGCTGAAGTGCAGT

GGCTATTCACAGGCACGATTATAGCTCACTATAGCCTCAAAGTCCTGGGTTCAAGCCTCCTGAGTAGC

TAGAACTTCAGGTACATGCCACCTTGCCTGGCTTCCTGCTTTCTTATTCACTAAGAAACTTTGTTCTC

TAAAATCATAGATTAGAGACAACTTCGTGTTTGAAATTACATCAGTAACAATAAAATATCCCACCTTT

GCTTGAAGGACCTCAGTCATTCCACACAGAGAATTGGCCTCAATTTACAACCCAGATGTCAAGCTTCT

GATAAGGGGTTTCCAGACACAAAACTCCATCGCTATTTTAACTTCATAGTCTCCAAGGAAATAGGCCC

TTCATCCACTTTGCAGTCTAGACCTAGAGCCTGTGTGTTGATGGCAGCAGCAGGCCGTCTGGAGCAGC

CACTACCATCATTGACATACAGCCTTGTTTTGCTTTGAGTCTACCAAAATACTGCTTATTTAAATTTC

ATCTCAACTCCACACTTCCCCAGAATCCTATCACTCTATCTGGGGAGATGCCATGTGGTCCAGGCAGT

CTTAGGCTGAATGGGCAAGGATCCAAGGGAAACCAATTAGATTGAAATTAGTTCTGTCTGTGGGCCCT

CAAGATTTAAAAGCCATGGACAAGGGGGAAGCAACCAGTATCGACCAGCGATAATCTATGAGCACCTT

CAGCTTGGGGTGTGGGGTCATAGACAAAAAGAATGGGGTTTAATCAGTGTTCCCATCCAAGGCAAAAC

CAGACCAGAAGAATGGGAGCTACCAGGAGGCTGAGTTAAGACCAATACTAGGGCCAGGCATGGTGACT

ACAGCCTGTAATCCCAGCACTTTTGGAGGCCGAGGCAGGTGGATTACTTGAGGTCAGGAGTTCGAGAC

CAGCCTGGCCAACATGGTGAAACCTCGTCTCTACCAAAAAAAAAAAAAAAAATTACAACTGGCACACA

CTTGTACCCAGCCACTAGGGAGGCTGAGGCAGGAGAATTGCTTGAACCCGGGAGGCGGAGGTTGCAGT

GAGCCGAAATCACATCACTGCACTCCAGGCTGGGTGACAGAAGGAGACTCCGTCTCCAAAAAAATAAA

AAATAAAAACCAATACTAGGAAGTGCTTTCTTACCATGAAAGCTCCCTGCACACAGAATGAAGAGACA

GATGGCAGAAGCAAGACCAGAGGCCTCCACCACTGAACAAGGTCAACCTGAATTTGTTTTTGTAGCTC

TGGGAAGTCCTGAACTGAATGGGAGGTTCTACTGTGGCCCTCTACTAACAGTAGATGGAACCTGGAAC

CATCTACTGTTAATTGTCCTTTTAGTAGCCAAGACATGAAGTACCTATCTCCCTTCTTTGTATTTGGT

GAAAAGATTTGAACTCGAGCCATCTCTGCTAAAGGCATGACTTTAGTCACCCCACTGCAGTTGCTTCT

CTTTGCCTCCAAGAAAGTCCAATCTGATGGGCAGATTGTGCTTGCAGATGACTGGATTAAACTGCAAA

TATCTCATGAAGCTGCTGCCTGCATCACCGCTCTCCAGGCAGCCACGGAGGCTCTGGTTGTTGAGGCC

ATCTACTGTGGCCCTCATACATCATACTGTGTATGGTTCCAGGATTTGTAAATGCTCTAATCAATGTT

TTTTTTTTTTTTTAAAGCTGTTCCTGCTGATTGCTTTTTAAGCTAGGCTCAAGTGTGGGGCCACGGAA

TCCTTCAGGTGCCCTTCAAATCAGACATTACATTTCAGCTCTCAGGGCTAGAATGCTTCCCCCTCTTA

TCTTGGGCAGGTCTTTGAAGAAGCCAGTTTAGCCTTGGTAATTTTGAATTTGGCCATAGTATACGGAG

GAGAGCTAGGTAGCTATCAAAACGCTTAGACAAACGTATGGTAAAATGCAGCCCAGCCTGAACCTCCG

CAAAACCCCCAAACCTGGAGCAGCAAGTGGGCAAAGGAGTGTGTGTTTGGGGCAGGGTACTGAGTCCC

ATATGTAATCAAGAGACCTGGAGGGCCCAAGGAAGTCAGTGACACCCCCACAAGCTACATATCCTCCA

ATGGATTGTGGTGGTCATCTCCGGCTACCGAAGCCACCTTGCTTATTCCCATAGGCTTACAAACATTA

TGATGCTTCACACGAGTTGTGGGTGAAGTGAAGGAATTGGAAAGTCCCGGTCTAAGGCCCTGCAAAAC

TGCTGTCTGGTAAAAACAAGTGACTATGGCAAGGCCCTGAATGTACTACGTATAGACTTAAGTCAGTA

TTTGTGCCTAACAGCAAAGCATGCCACAAGAGGCTCCTCAAAAGCCGGGTAATTGATGCCATTGTTTT

CTTGACCTAGTTGAAGAGTCAAAGAAGAGAAGGTGATGCAGAAACTTCTTTCCAGTGAAGTGACAGAT

CCAAGCAAGGAACACCTGCCTGTGAGGGGAGCGGTAGGGGAGGGGGTAGATTCGGCAGCACTCGGGTA

GTGGCAGGGACCACATATTGTCGGTGTTAACCACTGTTATCACTGGCCCAGGTCGAGTGAGGACGAGT

CATTTCCCCACTAAGCACTTGGGGAGTGGCAGGGACCACATATTGTCAGTGTTAACCACTGTTATGAC

TGGCCCAGGTCGGGTGACGACGAGCCATTTCCGCACTATTGTCCCTTCTTCCTCACTGCACCCCCAGC

CCTGCACCCTCAGCTCCAAATACACATTCTCGTTCACTCAACGTTTGTTGACACCTAGCGTGGGCCCT

GTGGGATGGGGTAAAAGTTCGGTGTTGGGAGGAGCCCCGGTGACAGATTTTTGGTTTTGGTGGTGGCT

TTTGGGCAAGGACAGGTCAATTCATAAAAGATGACGCCCCAGGTCTGGATCGAATTCTCCTGGGAGCC

AGACCAAAGCACCTTCCTGGACTGCAAAGTGTGGAAGCCGCCGCGGGCCGGGCGCGGGGAGGTGTCAT

GCGCCGGAACCTGCGCTTGGGGCCAAGCTCTGGAGCTGACGCGCAGGGGCAAGGCGCCCCGCGTCCCG

GACTGGCGGCTCCGCGCATGCTCCTCCCACCGGCGTCGCAGGCCTCGAGAGGCTCCGG

ATGGCTCAGGAGGTCGATACAGGAGAAGGGGGAGAGATGGGGAGGG

GAGCGGGAGCGGCGCGAGGCAGGGCAAGCTGGTGTTGGGCACTCGCACTGCTCTGGCTGGCGGTTGTC

CCGGGATGGTCTAGAGTGAGCGGCATTCCAAGTAGGAGGCATTGGCCAGTGCCCTACAAACGATTTGA

TTTCAGACCCAAGCCAGACCCGTACTGCCAAGCAAAGTATACATTCTGCCCCACTGGCAGCCCCATCC

CTGTGATGGAGGGGGATGATGATATAGAGGTCTTTCGGCTCCAAGCCCCAGTATGGGAGTTTAAATAT

GGAGATCTCTTGGGCCATCTGAAAATTATGCACGATGCGATCGGGTTTAGATCAACATTGACAGGCAA

AAATTACACCATGGAGTGGTATGAACTCTTTCAGTTGGGAAACTGCACTTTTCCGCACCTTCGACCTG

AAATGGATGCCCCTTTTTGGTGCAATCAGGGCGCGGCTTGTTTCTTTGAGGGTATAGACGATGTACAC

TGGAAGGAGAATGGCACACTTGTACAGGTGGCAACAATCTCCGGTAATATGTTCAACCAAATGGCTAA

ATGGGTTAAACAGGATAATGAGACTGGGATATATTACGAAACCTGGAATGTAAAGGCTAGCCCAGAAA

AGGGTGCAGAGACGTGGTTTGATAGCTACGACTGCAGCAAGTTCGTCCTGCGGACCTTCAACAAATTG

GCGGAGTTTGGAGCAGAGTTTAAAAACATAGAGACTAATTACACACGCATTTTTCTTTATAGCGGCGA

GCCCACTTACCTCGGTAACGAGACATCAGTCTTCGGGCCCACCGGAAACAAGACCCTTGGTTTGGCGA

TTAAAAGATTTTATTATCCATTCAAACCTCATTTGCCAACTAAGGAATTTCTCCTCTCTCTCCTGCAG

ATATTCGATGCGGTCATTGTTCACAAGCAATTCTACTTGTTCTACAACTTCGAATATTGGTTCCTTCC

AATGAAATTCCCATTCATTAAAATCACATATGAGGAGATTCCCTTGCCCATAAGGAACAAGACTCTCT

CAGGGCTCGATTACAAAGATGATGACGACAAATA

The AGA protein sequence is:

(SEQ ID NO: 34)

MARKSNLPVLLVPFLLCQALVRCSSPLPLVVNTWPFKNATEAAWRALA

SGGSALDAVESGCAMCEREQCDGSVGFGGSPDELGETTLDAMIMDGTT

MDVGAVGDLRRIKNAIGVARKVLEHTTHTLLVGESATTFAQSMGFINE

DLSTTASQALHSDWLARNCQPNYWRNVIPDPSKYCGPYKPPGILKQDI

PIHKETEDDRGHDTIGMVVIHKTGHIAAGTSTNGIKFKIHGRVGDSPI

PGAGAYADDTAGAAAATGNGDILMRFLPSYQAVEYMRRGEDPTIACQK

VISRIQKHFPEFFGAVICANVTGSYGAACNKLSTFTQFSFMVYNSEKN

QPTEEKVDCIDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for AGA and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

AGA-DNA

(SEQ ID NO: 35)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG6

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG TGTCTCTACTTAAAATACAAAAATTAGCCG

GGCATGGTAGTAGGCAGTAGCAATCCCAGCTACTCAGGAGGCTGGGGCAAGAGAATCGCTTGAATGCA

GGAGGTGGAGGTTGCAGTGAGCTGAGATTACACCAGTGAACTCCAGCCTGGGCGATAGAGCAAGACTC

CATCTCAAAAAATAAATACATACATACATACATACATACAAATATCCATGGAAAAGTTGATATATTGA

GACCTTCTATCAGCCATTCCCACTATAGTGTAATTTAGCTGTTTCTCTGACAGGAATTTTTATTTAAG

AAGAAAGAATTTGGAGAAATAGACATAGTAGGAGGTAACAGATCAGGCTCTTTCCACCAGAAGAAACA

CAAATACCTTAAAAAACAGGAAGAGGCTAGAGAGATCAGTGGGGTACTAAGACCAATTTTCCATGTAT

TGTATTTTACAATTTTTGCTAAAGGCATGTCCTAATTCCTTAAGGCTGCATAAAAATACTCAGCCCAG

GAGCTAAGGAAAATTTACTCAGAGGACAATGTATGTTTTTCTGGCTTTCTTACCCTGTTTCACCCAAG

TTTCCAGTTTCACCTGCCATTGGGTTATGATTACTCCAGCACAGATGACTTGTTCCTCTTCCAAACCC

CATGAAAGGAACTTAGAAAATTTGTCTTTATTATATCTTTTAATTAACACTTCCCCAACGGGATAATG

ATACTTCTGGGCTATCTTATGTTCTTTAAATTATGTATTTAATTTCCTAGCAATCAATAAGAGCCTTG

AAGGTTTTGTAAGTCTCACAACTCCTACCTAGTTTATATTTTTACATTTATTTATTTATTTATTTTTG

AGACAGAATCTCACTCTGTTGTCCAGGCTGGAGTGAAGTGGTGCGATCTCGGCTCACCACAATCTCCA

CCTCCCGGCTTCAAGCAATTCTGCCTCAGCCTCCCAAGTAGTTGGGACTACAGGCATGCACCACCACG

CCCGGCTAATTTTTGTATTTTTAGTAGAGACGGGTTTCACTATGTTGGCCAGGCTGGTCTCGAACTCC

TGACCTCGTGATCCACCCACCTCAGCATCCCAAAGTGTTGGGATTACAGGCGTGAGCCATCGTGCCCA

GCCTAGTTCATATTTTTAAAGTTTGTATAGATTTATAGATGCTTTTAAAAATTATGTGATTATGTGTC

TTGCCTTGGCAACCTGGGCAGGCATTCCAGACACCCAAAGGGATTATTAGTTCATGTTCGCACGTCTG

CCCAGCAACAAAACTGAGCAGCCTCCACCAAGCACGTAAAATCCCCATTCCTCCTCTTATTCATTTAT

TTAGCACTTAATAGTGCCAGGCAGGAAGGTAAAAATACATTTAAAAAGGAATGGAGGTAGCCGTCTTG

CCCTGGAGGTGCAGGAAGCTGCACTTCAGTGTGCTAGGTAGAGGGTACATAGGCCGTTTCTGTTTTTC

TTCCAGCTCAGGGATTTATAATGAGAGGAAGTGCTACAAAAAGCTGTGGTGCGAAAATGACGGACGCT

ATGAATGGAAAAAAAATCTAAAATCAATCCGGTCCTAAGCAAACACGCTTCACCTACACTCATAACTA

TTGCAGACCTCCGAGGCCTGGGATCCCCAAGAATATACTGAGTTTGACAAACTTTTCAACTTCAACTT

TAAATTAAAAAGACAGTAAAAGAACCAAATCCATAGTACACAGCAATCGGCTAAAGTTCCTGGGGCCC

TGCAACCCAGAGTTGAATAATTTTGTATTAAATTCCCAATATCAAGCTAAATCTATTTTAACCACAGG

AAAAGCAGCAACTTGTCTGGCTATTTTAAAAATCTGAACAGCACTTAGGAAGAAGCACCTTAGCGCAG

GGAACAGCTCAGTGCCCCGTGACACAACTCTCCCGCGGGCCCAGGGACGCCTCGTCTCGCGAGAGTTG

AGGGACGCCTGAGCGAACCCCCGAGAGAGCGGGCGTGGGCGCCAGGCGGGCGGGGCACTGGGGAT

ATGGCCCGCAAAAGTAACCTTCCCGTTCTCCTGGTACCG

TTCTTGTTGTGTCAAGCACTTGTACGCTGCTCTTCACCGCTCCCGCTTGTCGTTAACACCTGGCCTTT

CAAGAATGCAACAGAGGCGGCATGGCGAGCACTCGCATCTGGCGGGTCTGCCCTCGATGCAGTAGAGT

CCGGCTGTGCCATGTGCGAAAGGGAACAGTGTGACGGGAGTGTGGGATTTGGCGGCTCTCCAGATGAG

TTGGGAGAAACGACCCTTGATGCGATGATTATGGATGGAACAACCATGGATGTGGGGGCGGTCGGTGA

CCTCCGCAGGATAAAAAATGCTATTGGGGTGGCGAGGAAAGTCCTGGAACACACCACCCATACTTTGC

TCGTTGGTGAGTCTGCAACAACGTTCGCGCAAAGCATGGGCTTTATAAATGAGGACCTGTCCACAACT

GCCTCTCAGGCTTTGCACTCTGACTGGTTGGCGAGGAATTGCCAACCCAATTACTGGAGAAATGTTAT

TCCCGATCCATCCAAGTATTGCGGGCCTTATAAGCCACCGGGAATCCTTAAACAAGATATACCTATTC

ATAAGGAAACCGAGGATGACCGGGGACATGATACGATCGGAATGGTAGTGATCCACAAGACAGGGCAT

ATTGCGGCGGGGACCAGCACAAATGGTATCAAATTCAAAATTCACGGTCGAGTCGGTGACTCCCCCAT

ACCTGGTGCCGGGGCTTATGCTGACGACACAGCTGGAGCTGCTGCCGCGACAGGTAATGGTGATATCC

TGATGCGGTTTCTCCCATCATACCAGGCTGTCGAATATATGAGACGGGGTGAGGACCCCACTATAGCC

TGTCAAAAGGTTATTTCTCGGATACAGAAGCATTTTCCGGAGTTTTTCGGTGCAGTTATCTGCGCCAA

CGTTACTGGTAGCTATGGAGCTGCATGTAACAAGTTGAGTACGTTTACCCAATTTTCATTCATGGTTT

ATAACTCCGAGAAAAATCAGCCGACTGAGGAAAAGGTTGACTGTATTGACTACAAAGACGACGACGAT

AAATA

The IDUA protein sequence is:

(SEQ ID NO: 36)

MRPLRPRAALLALLASLLAAPPVAPAEAPHLVHVDAARALWPLRRFWRST

GFCPPLPHSQADQYVLSWDQQLNLAYVGAVPHRGIKQVRTHWLLELVTTR

GSTGRGLSYNFTHLDGYLDLLRENQLLPGFELMGSASGHFTDFEDKQQVF

EWKDLVSSLARRYIGRYGLAHVSKWNFETWNEPDHHDFDNVSMTMQGFLN

YYDACSEGLRAASPALRLGGPGDSFHTPPRSPLSWGLLRHCHDGTNFFTG

EAGVRLDYISLHRKGARSSISILEQEKVVAQQIRQLFPKFADTPIYNDEA

DPLVGWSLPQPWRADVTYAAMVVKVIAQHQNLLLANTTSAFPYALLSNDN

AFLSYHPHPFAQRTLTARFQVNNTRPPHVQLLRKPVLTAMGLLALLDEEQ

LWAEVSQAGTVLDSNHTVGVLASAHRPQGPADAWRAAVLIYASDDTRAHP

NRSVAVTLRLRGVPPGPGLVYVTRYLDNGLCSPDGEWRRLGRPVFPTAEQ

FRRMRAAEDPVAAAPRPLPAGGRLTLRPALRLPSLLLVHVCARPEKPPGQ

VTRLRALPLTQGQLVLVWSDEHVGSKCLWTYEIQFSQDGKAYTPVSRKPS

TFNLFVFSPDTGAVSGSYRVRALDYWARPGPFSDPVPYLEVPVPRGPPSP

GNPDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for IDUA and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

IDUA-DNA

(SEQ ID NO: 37)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG TCCAGGGAAGGTGTACTTTTCTGTCCACAG

GTGTCCCGGTGAGCGGCTCACCTTAATTCTCTCAGGGCTTGAGGTGATTTAAGGCTGGCTTCAGTCCC

CACAAAGACAATTCTGTTGCTGGTTTACCTTTGCTTTTAGGGGATAGAACTTTGGGGTCCCAAGAAAA

ACCCAAAGTCTTTACCAGGCCCCCCGCCTGAGCCTCGACTTCTGTCCTGTGGCCACAGTGTGAGGAAC

GAACGCAGCAGCGTGTCGCCATGTCTTGTGTGGCTGGGATACGCTGCAGGGGTGTGGAGTCGCCGCCC

CAAAGCGGGCCCCGCCCCCTCCCGGCTTGTCCCTGCAGAGCCCCGAGGCCTCAGAGGCTCCTCAGAGC

TCCCAGTGCCCTGGACGGTTCCGTTCAGCGCTCTCCAGTTCTGATGGTTGCTCTCAGGAGGCGGCTGG

TCTGAAACAATTCAGTTTCCATTGCTGTGTCCTGCACCACGGTGTGGGACTGGTGGTTTCTTACTGTT

TTTCTCAGTTCTCTGACGCTTTGAGGAATTGTTTTTAAAGCGAGTTATCCATCATTTTAGTTGTTTTC

TCTGGGGTCGTTGGACTGAGGGTCTTACCCCGCTGTTCCTGGAGACAGTTCTGTACTTGCATGGTTTC

TCTCGCCTTTCCCTCCCTTTCTTACTTTTTAGTTTCTTAATGAATTTTTTTGTATTATGTTTTTCCTC

CTTGCTAATTTGGAATATATACACACTATTTCACTTTTAAATGGCTAATCACTTTTAATGCAATTTCA

ACATAAGAAGACCTAATGTTAAGCAATATTTTAACCCCTCTCCCCAAAATAAAAAGACCCAGAACACT

GTAACTCTAATTATCCTTCTCCCAACTTAGAGAGGATTATTGTCCAGTATTTTCGTTTTGTTAGTAGA

CTGTTCTTTTTTTCTTTTCTTTTTTTAGAGATGGGGTCTCATTTTGTCAACCAGGCTGGAATGTAGTG

GTGTGATCATGGCCCGTCTCAGCCTTGAACTCCTAAGCTCAAGCAATCTCCCACCTCAATTTCGTGGG

TAGCTGGGCCTCACAGGCATATACCATCGCATCTGGCTAAATTTTTTTTTTGTACAGATGGGGTCTTG

GTACATCTCCCAGGCCGATTTCAAACTCCAGGCCTCAAGTGATCCTCCCGTGTTGGCCTGCAATTCCA

AAGTGCTGGGATTGCAGGTGTGAGCCACCGCACCAAGCCTGTTCTTTCCATTATTATTTTTATACAAT

GTTTGCTTGGATTCACACATGCATACATGGACTACACATGTACTACAGTTGTCCCATGGTATCCTCGG

GGTATTGGTTCCAGGACCCTCCCAGATACCAAAATTTGCAGGTGCTCAAGTACTTGATATAAAATGGC

ACAGTATTTGCACATTACCTACGCACATCCTACTGTGTAGTTTAAATCAAGATTACTTATAGTCCCTA

AAACAATGTAAATGCTCTGTAAATAGCTGTTATACTGTATTGTTTTAAAATATGTATTATTTCGTATT

GCTTTGGCTTTAAAACAATATTTTCAGTCTGCTTGGTTGAATCCATGGATCTGAAGCCAAGTGTAAGG

AGGGCCCAGTGTGCTCTTCTGGGGTAATTCTCCTTCCTGCTAAAGCGCACGCTTTACTCAGGAGGCTG

GGGTGAGAAAATCGCTGAAGCCCCGGAGATGGAGGTTGCAGTGAGCTGAGATCGCGCCACTGCACCTC

AGCCTGGGCGACAAAGCAAGACTCTGTCTCAAAAACACACAAAAACAGAGAAAAACAAGACAGTAATG

GCTCAACTCACATAGCACCAACGGGCGAAGCGTTCTTCTGAGCGCTTTCCGAGTCATCGGTCCTCAGA

GCAGCCCCTGAGGCCCGCAAGGAAGCGGGGCTCCAAGCCCTGCCGTGCTCCCGGCTCCCCGAGGCTCC

CCGAGGCCACCCAACCCCTCCCACCCGGCCATCGCCCCCTCACCAAGGCCCCGCCCCGCGGCGGCGGT

CACATGGGGTGCGCGCCCAGACTCCGACCCGGAGGCGGAACCGG

ATGAGGCCACTGAGGCCAAGAGCTGCACTTCTGGCGCTCTTGGCGAGTCTTCTCGCCGCA

CCTCCCGTTGCCCCTGCGGAGGCACCACATCTCGTTCATGTTGACGCTGCGAGGGCTTTGTGGCCACT

TAGGCGATTCTGGAGAAGTACGGGCTTCTGTCCACCGCTGCCGCACTCACAGGCAGACCAATATGTCT

TGAGCTGGGATCAGCAACTCAACTTGGCGTATGTGGGAGCTGTCCCGCATCGCGGTATTAAGCAAGTG

CGAACGCATTGGTTGCTTGAGTTGGTAACCACGAGAGGTTCCACTGGGCGAGGGCTCTCCTATAACTT

CACACACCTCGATGGGTATCTGGACCTGCTGCGAGAAAATCAACTCCTTCCCGGGTTCGAACTGATGG

GCTCCGCATCTGGCCACTTCACGGACTTCGAAGATAAGCAGCAGGTCTTCGAATGGAAAGATCTGGTG

TCAAGTCTGGCTCGCAGGTATATTGGACGATACGGTCTGGCCCACGTCAGTAAGTGGAATTTTGAAAC

GTGGAACGAACCAGACCACCATGACTTTGATAACGTATCTATGACTATGCAGGGATTTCTCAATTACT

ATGACGCTTGCTCTGAAGGACTCCGGGCTGCAAGTCCAGCTCTCCGCTTGGGCGGGCCTGGGGATAGT

TTCCATACACCTCCCCGATCCCCGTTGAGTTGGGGGCTCCTCCGACATTGTCACGACGGGACGAACTT

TTTCACCGGTGAAGCGGGAGTTCGGCTCGATTACATCTCTCTGCATCGGAAGGGCGCAAGAAGCTCCA

TAAGTATCTTGGAGCAAGAGAAAGTCGTGGCGCAGCAGATCAGACAGCTCTTCCCGAAGTTTGCGGAT

ACACCTATATACAATGATGAGGCTGACCCACTGGTCGGCTGGAGCCTGCCCCAGCCGTGGAGGGCTGA

CGTAACATACGCCGCGATGGTGGTTAAGGTAATAGCCCAGCACCAGAATCTTCTGTTGGCAAATACCA

CGAGTGCATTCCCTTATGCTCTGTTGTCAAATGATAATGCCTTCTTGTCTTACCACCCGCATCCTTTC

GCTCAACGCACGCTGACCGCCCGCTTTCAAGTAAATAACACTCGCCCACCCCACGTGCAGCTCCTGAG

GAAGCCCGTTCTGACTGCTATGGGGCTCCTCGCGCTGCTGGATGAGGAGCAGTTGTGGGCTGAGGTAT

CTCAAGCCGGGACTGTATTGGACTCCAATCACACTGTGGGGGTTCTTGCTAGCGCGCACCGCCCTCAA

GGACCTGCCGACGCCTGGCGCGCTGCAGTGTTGATATATGCCAGTGATGATACGCGAGCCCACCCGAA

TAGGTCTGTTGCCGTGACGTTGCGCCTCAGAGGCGTTCCCCCTGGTCCAGGACTCGTCTATGTTACTC

GATATTTGGATAACGGCCTCTGTTCCCCTGACGGTGAATGGCGGAGATTGGGGAGACCTGTGTTTCCG

ACAGCGGAGCAGTTCCGGCGCATGCGAGCGGCAGAAGACCCTGTTGCAGCTGCTCCACGACCGCTCCC

TGCAGGCGGCAGGCTTACTTTGAGACCAGCTTTGAGGTTGCCGAGCCTCCTGTTGGTCCATGTATGCG

CGCGGCCAGAGAAGCCACCCGGGCAAGTAACCCGGCTTCGCGCCCTCCCGCTCACCCAAGGCCAGCTC

GTGCTTGTATGGAGCGACGAGCATGTTGGATCTAAGTGTTTGTGGACTTACGAGATACAATTTAGCCA

GGATGGAAAGGCGTATACCCCTGTGAGTCGGAAGCCTTCTACATTCAACCTCTTCGTGTTCAGCCCCG

ACACAGGTGCTGTTAGCGGGTCCTATCGGGTGCGGGCGCTGGATTACTGGGCACGGCCCGGGCCGTTT

TCCGACCCTGTTCCCTACCTGGAAGTTCCCGTTCCGCGAGGCCCACCTAGCCCTGGGAATCCAGACTA

TAAGGACGACGACGATAAATA

The GDNF protein sequence is:

(SEQ ID NO: 38)

MKLWDVVAVCLVLLHTASAFPLPAGKRPPEAPAEDRSLGRRRAPFALS

SDSNMPEDYPDQFDDVMDFIQATIKRLKRSPDKQMAVLPRRERNRQAA

AANPENSRGKGRRGQRGKNRGCVLTAIHLNVTDLGLGYETKEELIFRY

CSGSCDAAETTYDKILKNLSRNRRLVSDKVGQACCRPIAFDDDLSFLD

DNLVYHILRKHSAKRCGCIDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for GDNF and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

GDNF-DNA

(SEQ ID NO: 39)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG AAACCCTCTTAAGACGGAAGGGAAATCTAG

TGCCTCTGTGGCTTCCTGCTGGCGGGCACCGCGATTTGGAATGGCGCATTAACCCCGCGCCTCCATGC

TCCTCCGGTCGGACCGCCAGCCATTTCGCACGCACCCGGGAGCTGGCACCGGCGGGGGGCTTGAGGGG

GGAAGCTCGCGTTCCCCAGGTCCTAGCTGCCAAAGTACCTTTCTGGGCTCATTTTGCATGGCCTGGTG

CAGTTTTCCTGTGTCTGCACATCGCGACCCAGAACCTAGCTTTTTCCCGAGTTTGCAAACCAGCCCGC

GAGGCAAGAGGCGCTCGGTGCTGCAGGTCACTAGGAGTTTCTAGTCCCTACACCCTCTCGAGCCCAAC

AGCTGCATAGCGAACAAACAGACGCTCAGGAGATGTAAAATGCATTAGTCTCTGCAGAGGTGGGAGGT

GGCGGCCGAATTAAAGGCTTCCCCGGGTTGCCTGCACCGGGACAGGGAGTGGGGTTCCGGGTAGAGAT

GAGGACTGGAACCCTGGAATGGAGGCGGGGGTGCCTGTGAACTAATGGCTAGGAAAGGCACTGAGGTT

TTTGCATTAACCTGGGTTTTGCATTTCCCTCCCGCATGGGAAAAAAAAAATCGAGTTTTTCCTAAGAA

CCGTTTTATGGAGCCGGTCGAGGGGATAGGATAATCGACTGCACGAATCTCGCAGATTCCGCTTGAGG

AGATTCTCTCTAGGTCACTAGTGCCCTGGAGACGAACCCTGGGATTAGGAAGGCACTGGATAACACTG

CACCCCAAGATGCCTCGGTTCTTCCCCGTCTCCTCATTGGTTGGAACTCGCCCCCAAATTTACAGCCC

CTTTTTCTAACCCACCCCCAGCTGCGTGGCCCTCCTTTGTAGGGGTGTGAGGATTGAGAAGCCAAACA

GAGCCCACCTCGGGCTGAAAAGAGCTGAACCCCCTACTCTGCGCCGTACCACGGTCTAGGCCTTCCAG

TGCCAGAGCACC ATGAAGCTCTGGGATGTCGTGGCG

GTGTGTCTTGTCCTTCTCCATACTGCGAGTGCGTTTCCACTGCCAGCTGGGAAAAGACCCCCAGAGGC

CCCTGCAGAAGACCGATCTCTTGGGAGGAGGAGAGCACCGTTCGCTCTGAGCAGCGACAGTAATATGC

CGGAGGATTATCCCGATCAGTTCGACGACGTAATGGATTTTATCCAGGCAACGATAAAGCGGCTGAAA

CGGAGTCCGGACAAACAAATGGCAGTGCTCCCGCGCCGGGAACGAAATAGACAGGCCGCCGCGGCAAA

TCCGGAAAATAGTAGGGGCAAAGGAAGACGGGGGCAGAGAGGCAAAAACAGGGGCTGTGTATTGACTG

CCATTCATTTGAACGTAACGGACCTGGGGCTTGGTTACGAGACTAAAGAAGAACTGATATTTAGATAC

TGCAGTGGATCTTGCGATGCCGCTGAAACCACCTATGACAAGATTTTGAAGAATCTTTCTCGAAACAG

AAGACTGGTTTCTGATAAGGTTGGTCAGGCGTGTTGCCGACCCATCGCGTTTGACGACGACCTTAGTT

TTCTCGACGATAATCTTGTCTATCATATCCTTCGCAAGCACTCCGCTAAGAGGTGCGGATGCATTGAT

TACAAAGATGATGATGACAAGTA

The GLB 1 protein sequence is:

(SEQ ID NO: 40)

MPGFLVRILPLLLVLLLLGPTRGLRNATQRMFEIDYSRDSFLKDGQPFR

YISGSIHYSRVPREYWKDRLLKMKMAGLNAIQTYVPWNFHEPWPGQYQF

SEDHDVEYFLRLAHELGLLVILRPGPYICAEWEMGGLPAWLLEKESILL

RSSDPDYLAAVDKWLGVLLPKMKPLLYQNGGPVITVQVENEYGSYFACD

FDYLRFLQKRFRHHLGDDVVLFTTDGAHKTFLKCGALQGLYTTVDFGTG

SNITDAFLSQRKCEPKGPLINSEFYTGWLDHWGQPHSTIKTEAVASSLY

DILARGASVNLYMFIGGTNFAYWNGANSPYAAQPTSYDYDAPLSEAGDL

TEKYFALRNIIQKFEKVPEGPIPPSTPKFAYGKVTLEKLKTVGAALDIL

CPSGPIKSLYPLTFIQVKQHYGFVLYRTTLPQDCSNPAPLSSPLNGVHD

RAYVAVDGIPQGVLERNNVITLNITGKAGATLDLLVENMGRVNYGAYIN

DFKGLVSNLTLSSNILTDWTIFPLDTEDAVCSHLGGWGHRDSGHHDEAW

AHNSSNYTLPAFYMGNFSIPSGIPDLPQDTFIQFPGWTKGQVWINGFNL

GRYWPARGPQLTLFVPQHILMTSAPNTITVLELEWAPCSSDDPELCAVT

FVDRPVIGSSVTYDHPSKPVEKRLMPPPPQKNKDSWLDHVDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for GLB1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

GLB1-DNA

(SEQ ID NO: 41)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG AATCAATACACTAATACAAAACTATTATTT

AATATTTAGACCGTGTACCATTATCAAAATCAGAAAGTTAACATTGATGCGGCCGGGCACAGTGGCTC

ATGCCTGTAATCCCAGCACTTTGGGAGGCCGAGGTGGGCAAATCACTTGAGGTCAAGAGCTCCAGACC

AGCCTGGCTAACATGGTGATACCCCATCTCTACTAAAAATACAAAAATTAGCCAAGCCTAGTGGTGGG

TGCCTGTAATCCCAGCTACTTTGGAGGCTGAGGTGGGAGAATCACTTGAACCTGGGAGGCGGAGGTTG

CAGTGAGCCGAGATTGTGCCACTGCACTCCAGCCTGGGTGACAGAGTGAGACTCTGTCCCCCCACCCC

CCCAAAAAGGAAATTAACATTGATGCATGGTACTAATCTAACCTACAAGTCTAATTCTGATTTCATCA

ACTGTCCCACTAACATCCTGTATATACCAAGCTTCTTATCGTCATCATCTCTGGTAATTTCCCCAATT

ATCCAGTGAATTTGATATTATTAGCCCTGCTTTACCAACAAAGAAACTAAGCTCAGAGAGGTTAAGTA

ATTTGCCCACAGTTACACAGACAGTAAGTGGCAGAGCTGGAATTCCAACTCAAGTGTACTTCCTCCAG

AGCCCTTGTTCTTTAATACTGAACTAGAATTGCAGAGCTTGGAAAGGACTTCACAGTGCAAGGACACG

GAATTGGTACTACAGGCATGAAAAGAAGCCAGGCACGGTGGCTCATGTCTGTAATTCCAGCATTTTGG

GAGTTCCAGGCAGGTAGATTGCTTGATCCCAGAACTTCAAGACCTGCCTGGGAAACGTGGTGAGACCC

TGTCTCTACAAAAAATACAAAAATTAGCCGGGTGTGGTGGCGCACGCCTGTAGTCCCAGCCACTTGGT

AGGCTGAGACGGGAGAATTGCTTGAGCCCAGGAGGCCAAAGCTGCAGTAAGCCGTGATTGCGCCCCAC

TCCAGCCTGGGTGACAGAGTGAGACCCTGTCTCAAAAAACACAAAAACAAAACAAAAAAACAACCCCC

CAAACCAAAAAACAAAAACCTAAGAAAAGCCAGTAACTGATACTTTCTCAATCTCTCAATGAAATGTC

CTTTCTGATACTTTCTCAATCTCTCAAGGACATGTCCGGTAGGAAAGGAGAAAGGAGTGAACAAATAG

AATTTTGGCTACTTTGTTATACAATGTAAAAAGGCTTTTTGGAACACCAAGGCATAAACTAAGGTTAT

TTTAAAAAAGAAAATTTTTTTTTGATACGGAATTTCGCTCTTATTGCCCAGGCTGGAGCGCAATGGCT

CGATCTCGGTTTACTGCAACCTCCACCTCCCGGGTTCAAGCGATTCTCCTGTCTCAGCCTCCTCAGTA

GCTGGGATTACAGGCGCCTGCCACCACGCCCGGCTAATTTTTGTATTTTTAGTAGAGACGGGGTTTCA

TCATACTGGTCAGGCTGGTCTGGAACTCCTGACCTCAGGCGATCCGCCCGCCTCGGCCTCCCAAAGTG

CTGGGATTACAGGCGTGAGCAGGATTTTCATTCTAACAAGTTCCAGGTGAGTTGATACAGTGGCTCCA

GGGACCGACCACATTTTGCTAACCCCCGGCTTAGAGTTATTCAAAGAGCCCGTATATGAGACGCGGAT

TCCATCTAGGGCGTTTAGGTTTAATGATTAACAATTTCCCTCTTCTGCTCTCTCAAGGCAGCCAGGGA

ACAGGGAGACCATGATTCATGTCCAATCCCCGAGGCGCGTTATCAAGCTGCTGAAAGCAGGCCCTCCG

GACTGCAGTTCCAAAGGGTCCCTTCCCAGGGAAGACGCCTGCAAAACCCAGATAGTACTATCCTGGAG

TCACGCGGCGGCGCGCAGCCTCCTAGCCGCCCCCACCCGCCCGGCTCGG

ATGCCGGGGTTCCTGGTGAGAATACTTCCCCTGTTGCTTGTGTTGCTCCTGCTTG

GTCCCACCAGGGGTTTGAGAAACGCGACACAAAGGATGTTTGAGATTGATTATAGTAGGGACTCATTC

CTCAAAGACGGGCAACCATTCCGCTACATCTCAGGCTCCATTCATTACAGCCGAGTACCTCGCTTCTA

TTGGAAGGATCGGCTGTTGAAGATGAAAATGGCCGGGCTCAACGCGATCCAAACTTATGTCCCCTGGA

ATTTTCATGAACCGTGGCCTGGCCAGTACCAGTTCTCCGAGGACCACGATGTGGAATACTTTTTGAGA

CTTGCTCATGAGCTTGGCTTGCTTGTAATTTTGCGGCCGGGGCCTTATATATGTGCAGAGTGGGAGAT

GGGCGGACTTCCAGCGTGGCTTCTTGAAAAAGAATCAATTCTGCTCCGGAGCTCAGACCCTGATTATC

TTGCCGCTGTTGACAAATGGCTGGGGGTCTTGCTCCCTAAAATGAAACCACTCCTTTACCAGAACGGA

GGTCCGGTAATTACCGTTCAGGTTGAAAACGAATACGGTTCCTACTTCGCCTGCGACTTCGACTACCT

CCGCTTTTTGCAAAAGCGATTTCGGCACCACCTTGGAGATGACGTCGTCTTGTTTACGACTGACGGGG

CTCACAAGACATTTTTGAAGTGTGGCGCCTTGCAAGGTTTGTATACCACCGTTGACTTTGGCACCGGT

AGTAATATCACTGATGCATTTCTCTCTCAGAGAAAGTGTGAGCCAAAGGGTCCTTTGATCAATTCAGA

GTTCTATACTGGATGGCTGGACCACTGGGGACAGCCTCATAGTACGATCAAGACTGAAGCCGTCGCGT

CTAGCCTTTACGATATCCTCGCACGCGGAGCCTCCGTTAATCTGTACATGTTCATCGGTGGGACGAAC

TTTGCGTACTGGAACGGAGCAAATTCTCCGTATGCTGCACAACCCACTAGCTATGATTATGATGCTCC

TCTGAGTGAGGCGGGCGACCTTACAGAGAAATATTTCGCTCTCAGGAACATCATCCAGAAATTCGAGA

AAGTTCCAGAGGGGCCCATCCCACCTTCAACACCAAAGTTCGCCTATGGGAAAGTCACACTGGAAAAA

TTGAAAACAGTAGGAGCAGCTCTCGATATACTGTGCCCTTCCGGTCCGATAAAGTCTCTGTACCCACT

TACATTCATTCAAGTTAAACAGCATTATGGTTTTGTGTTGTACAGGACTACCCTTCCCCAAGATTGTT

CAAATCCTGCACCGTTGAGCTCACCACTTAATGGGGTGCATGATAGAGCGTATGTCGCGGTGGACGGT

ATCCCCCAAGGGGTTTTGGAGCGAAATAACGTCATTACGCTCAATATAACTGGTAAGGCTGGGGCCAC

CCTGGATTTGCTTGTAGAGAACATGGGGCGGGTTAACTATGGCGCGTACATCAACGATTTCAAGGGTC

TGGTGTCAAACCTTACGCTGAGCAGTAACATACTGACTGATTGGACTATCTTTCCTCTTGATACGGAG

GACGCTGTTTGTTCTCACCTTGGGGGCTGGGGACATCGCGACTCAGGGCATCATGATGAAGCTTGGGC

ACATAACTCCTCTAACTACACGCTCCCCGCCTTTTACATGGGCAACTTCAGTATACCAAGTGGCATCC

CCGACCTCCCTCAAGATACCTTTATTCAATTCCCAGGCTGGACAAAGGGTCAGGTCTGGATCAATGGT

TTTAATTTGGGAAGATATTGGCCAGCCCGGGGACCCCAGCTGACTTTGTTTGTGCCCCAACATATTCT

GATGACGAGCGCTCCCAACACTATAACCGTTCTCGAATTGGAGTGGGCCCCCTGTTCCAGTGACGATC

CTGAGCTCTGCGCAGTCACCTTTGTAGACAGGCCAGTCATTGGTTCCTCTGTGACTTATGACCATCCA

TCCAAGCCTGTCGAGAAGAGATTGATGCCGCCTCCTCCGCAGAAGAATAAGGATAGCTGGCTCGACCA

TGTAGACTACAAAGACGACGACGATAAGTA

The PMP22/MFN2 protein sequence is:

(SEQ ID NO: 42)

MLLLLLSIIVLHVAVLVLLFVSTIVSQWIVGNGHATDLWQNCSTSSSGN

VHHCFSSSPNEWLQSVQATMILSIIFSILSLFLFFCQLFTLTKGGRFYI

TGIFQILAGLCVMSAAAIYTVRHPEWHLNSDYSYGFAYILAWVAFPLAL

LSGVIYVILRKREDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for PMP22/MFN2 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

PMP22-DNA

(SEQ ID NO: 43)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG GGAGTTTTCCTCACCTGACTCTGGGAAGGG

AAGAGAGTCTGGCCCAGATGGGTAAAGACAAGTGTGTTGGCATCTGGTAGACAGGGCCAGTGGCACAC

AACAGTGAACAGCTCTACTGCTGACAGCCAACAGGGACTTCGGGGTTTGGCTGCTGGAGATGGAGGAC

CTGGGCAAAAGAGACGTGAGGCCAAGAGAGCAGGTCTCAGAATGTGGAGCTCATGGAAGGAGAGAGGG

AGGGCTAATCAGACTGCTCCTATCTGGCCAGAAGCCATGCCCCACACTCACCAGGCCCTACCCCTTTT

CCTCTGAATCTTTAGCTCTTAGATCAGAAATAATCTTACGTGTCTTCAGGCTTGATCCTGGCCTGACG

GACAGCAGCCTCTGTCGTGTAGCCTTTGGAGCTGATCATGGCAGTACCCAGGAAAAGACTGTCTGGAG

AGGCTGCAAAAACCATGCTCACTGTTCCCAGTCCCCGTACAGACCATGTCAGAGGAGGACTGAGGGAG

GCTCCTCTCTCTCTCTCTCTGTCACACACACGCACACGCGCACACACACACACACAGATACACGCGCA

CACACACACACCGTTTTGCCTGGCAGAACAGACTTATAACTTGCATAATACTGCAGCAGCCCACACTG

TACCTCATCTCCATTTGGGTTCCACAACAGAAGGGAGCTGGAAGGAAATCAGCGACAATCTAACTCCA

CATGACAACCTGGCTGGACAACGAAACATCCAGCGGGGAGAAAGACACTGGAAATTACCCATACCTGA

GCCCCATCCCAAAACACCTGAGTCAGCTTCTCTGACCATGGCCCAGGCTAGGTCATTTGCAAAGCTCT

TGAGTGATTCTAAAGAACCACTGCTGTGAAAAATAAAAATGATTTCAGCAAAGAGAGAAAGGTGAAAC

TATTTTATTGGCACTGGCTGTGATGCTGGCTGTGAGCAGATGCTGGCTGTGGGCAGAACAGGGGAGAG

GGCCATTGAGAGGCCCTTGTGACTGGAGAGTCCAATCAAACGTAAAGTGTGTGACAGGGAGGGACTCC

TCCAAATTCTGCAGGTCTCTCCAGACTGGGGATCCCTAACACAAGAGGAAGTCTTATGCCCCAAGTGC

CAGAATTCCAGGAAACTCATGCAATGAAACCAGTCCTCTTGTGTCAGAGTTTGCAGCTAGAAGCGGTT

CAAGGGATGGAGGACTCTCTGGGAGCTGGAGTGGTACGCTGCAAAGAACCTAAATATGCCATAACTTC

CTTTACTTCCCTCTCCAAGAATTTCCAATTGCTCTTCCAGCCTATTCTGCCTGACAGGTTAGAAAGTT

TTCAATAGTTTTCAATAGAGGTTCGTTTACTTGCATTGAATTATCTCATGAAGGTCAGCTTGATTCGG

GCACATTAAAGACAGAGTCGGAAGAACACATTTCCCTAAGTTTTATTCTAATGTACAACACATGTGAT

CATTTGCGATGGGGTCTAGGCAGACAAGGCAGGTAACAGGGAGTCCTTCCAACCAGGGGTTGGAACAA

AGGAGGCTTGATTTGGTGACTCTTGAGACATTTGGCTCACTGCTGTGATGCTGTGAGAGATTAGCTGT

GCAATGTTTGGGCTCCTTAAAAGGAAGTTTATTTAAAATAAAACTTACCTGCACGTATGTAACACTGT

AGACACAGATCCTTCCCACAGTATATTTAATCTCTGCAGAATTCACTGGGAGGGGAGGGGAGCCAGTG

GGACCTCTTGGCTATTACACAGGTTGGCACTTCCAGAGAGAACAGTCTTGGCATCACAGGCTTCAGGC

ATACTCAAAGCTCTTCTCCCTTCTGATTCCAGTTTCTCCATGCCCTGCAGGGCCTCTTGGGATTATTG

TATTCTGGAAAGCAAACAAAGTTGGACACTGTCTCTTTAAATAATAGAGGCTGAGAACCTCTCAGGCC

ACCATGACATATCCCAGCATTGGACCAGCCCCTGAATAAACTGGAAAGACGCCTGGTCTGGCTT

ATGCTTTTGCTGCTTCTCTCCATAATAGTGTTGCACGTAGC

AGTCTTGGTCCTGCTCTTTGTTTCTACCATTGTATCACAGTGGATTGTGGGTAACGGCCACGCTACCG

ATCTGTGGCAAAATTGTAGCACGTCCAGCTCTGGTAATGTACACCATTGTTTCAGCAGTTCACCTAAC

GAGTGGCTCCAGTCAGTCCAAGCCACAATGATCCTGAGCATAATTTTTAGCATCCTCAGCCTTTTCCT

GTTCTTCTGCCAGCTCTTTACATTGACAAAGGGTGGACGGTTCTACATTACAGGGATATTCCAAATTC

TTGCGGGTCTGTGCGTGATGTCCGCCGCCGCCATCTACACCGTTAGGCACCCTGAATGGCATCTTAAC

TCCGACTATAGTTATGGTTTCGCCTATATCCTCGCATGGGTGGCTTTCCCTCTTGCCCTGCTTAGCGG

CGTAATCTATGTGATTCTCAGGAAGAGAGAAGATTATAAGGATGACGATGACAAATA

The MECP2 protein sequence is:

(SEQ ID NO: 44)

MAAAAAAAPSGGGGGGEEERLEEKSEDQDLQGLKDKPLKFKKVKKDKKE

EKEGKHEPVQPSAHHSAEPAEAGKAETSEGSGSAPAVPEASASPKQRRS

IIRDRGPMYDDPTLPEGWTRKLKQRKSGRSAGKYDVYLINPQGKAFRSK

VELIAYFEKVGDTSLDPNDFDFTVTGRGSPSRREQKPPKKPKSPKAPGT

GRGRGRPKGSGTTRPKAATSEGVQVKRVLEKSPGKLLVKMPFQTSPGGK

AEGGGATTSTQVMVIKRPGRKRKAEADPQAIPKKRGRKPGSVVAAAAAE

AKKKAVKESSIRSVQETVLPIKKRKTRETVSIEVKEVVKPLLVSTLGEK

SGKGLKTCKSPGRKSKESSPKGRSSSASSPPKKEHHHHHHHSESPKAPV

PLLPPLPPPPPEPESSEDPTSPPEPQDLSSSVCKEEKMPRGGSLESDGC

PKEPAKTQPAVATAATAAEKYKHRGEGERKDIVSSSMPRPNREEPVDSR

TPVTERVSDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for MECP2 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

MECP2-DNA

(SEQ ID NO: 45)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG TCCTTGTTTCTTCCCTTGCTCTATGCCCTA

CATCCAATCCTTTTGTTCAGTTCTGAAAACATATCTTGAATCTCACAACTTCTCAGAGCTGCACTGCT

GCCACCCTAGTTAAAGCTACCATCAACTCTTTCTCCTAGACTGTTCCAGGGCCTTGCAACTAGCCTTG

TGCTGTAGTTTTGTTTCATCACGTCCAGTTCTCCACTCTACACCTGCAACATAGATCAGACAGCTCCT

GGCTCAAAATCCTCTGAGGGCTTCTCATCTTAGAATAAACTCTCGGTTCTGGCCGGGTGCGGTGGCTT

ACGCCTGTAGTCCCAGCACTTTGGGAGTCCGAGGCGGGCGGATCACTTGAACTCAGGAGTTTGAGACC

AGCCTGGGCAACATGGTGAACTCCCATCTCTATCAAAAATACAAAAACTTAGCCAGGCGTGGTGGTTC

GCATCTGTGGTCCCAGCTACTTAGGACGCTGAGGAGGGAGGATCGCTTGAGCTCAGGGTGGACGTTGC

AGTGAGCCAAGATTGCGCCACTGCACTGCAGCCTGGGTGACAGAATGAGACCCCATCCCCACCCCCCC

CAAAAAAGAATGAACTCCCAGTTCTCATAGTGGCCCCAGCTGCCTTTCCAATCACATTCCCTACCACT

CTCCAGCAACACTGACTTCCTCGTTAGTCCCCAACATGCCAGGCATAGTCTCTCCTCATGTCCTTTGA

ACTTGCCTGGAATGTTCTTTCCCCAGATATTCATATGAGGGAGTAAAATGAGGGTGAAAACCAGCAGA

TATCTAAATAGCACCCCCTTCACTTAGTTTATCTTTCTCAAAGCCCTTATCACTATGTGAAATGATAT

ATTATACTTATTTGTATGCTAGTATGAATCTTCCCGGCAAGAATGTTAGTTTGCTGTCTGTTCAGTAC

CGTGCATCCAGAGCCTGGAAGAGTGCCTGGCACATAGCAGGTAGTCAATAAATGAATGGGGGCAAGCA

GCCAAATCAGAATCAGGTTTTCTTGCTAAGCATAGAACTAACAGAAGGATCATTGAATGGATTGGATA

ATGACTGGCATCAGGGTAAGGTCCCCTTAACAAACACTCCTGTCCTGAACACCTGGTTAGCTAACAGT

TTTCTCATACTCTTATTTTCCCAAAACACAATTGCTGGATCTCAGCTCCAAATCAACTCTTCTAGGAA

AGTGAAAAATTGCTGGATCTCAGCTCCCAAATCATATCTTCCAGGCAGAGCTAACATTGCCCCTTATT

CACACCTCCACCAAACCATCTGATCCAACAGTGACAGGTGTCACGAGGCCTTGGCATGCACTCTCTTC

CCCCGCCAGAGTTCTGCGAAAGCCAGGGTTGCGATTTGTTGTCAGTTTATTCCCCGCCTCTATGAGAG

TGTGAGCACTGGGCAGGCTCGGATGAAATAATGCATTGAGTAGGCCTCTGAAACCAAGGCCCCTCAGC

TGGGGCAACGTCAGGCTCCAGGGTGGGCAACTTTGCTGCTTCTGCCGAAGATAGTGATATTGAGAAAA

TGTGGGTGCAATGAAACGCTTATTGCAGCGCACTCGGTGCATCTGTGGACAGAGGGTCAATCGCCCCT

CAGAGCAGCGCAAACAGGCGTCCCAAGCCTAGGCCTTCACTTGCCCCAGCATCCGCAAGGGTCCATTA

ATCCTTAACATTCAAATTCCGCCCACTAAACCAGTCCCTCCGCGCCCAAGCCGCCTCTTTTCCCCAAA

CGACGGCCGAAAGCAGCCAATCAACAGCTGGAGGGGTCCGCCCCCTTTTCCCTGGCCGAAATGGACAG

GAAATCTCGCCAATTGACGGCATCGCCGCTGAGACTCCCCCCTCCCCCGTCCTCCCCGTCCCAGCCCG

GCCATCACAGCCAATGACGGGCGGGCTCGCAGCGGCGCCGAGGGCGGGGCGCGGGCGCGCAGGTGCAG

CAGCGCGCGGGCCGGCCAAGAGGGCGGGGCGCGACGTCGGCCGTGCGGGGTCCCGGCGTCGGCGG

ATGGCCGCCGCTGCTGCCGCGGCTCCCAGCGGCGGCGGT

GGAGGTGGTGAAGAAGAAAGACTTGAGGAGAAATCAGAGGACCAGGATCTCCAAGGTCTCAAGGATAA

GCCGTTGAAGTTCAAGAAGGTGAAGAAGGACAAGAAGGAAGAGAAAGAGGGCAAACACGAACCGGTCC

AACCTAGCGCTCATCATTCCGCCGAACCAGCAGAAGCGGGCAAAGCAGAAACGAGTGAAGGATCAGGT

AGCGCTCCTGCAGTTCCGGAAGCGAGCGCTTCACCGAAACAGCGCCGGTCTATCATCCGGGATCGGGG

ACCTATGTACGACGATCCAACGCTTCCGGAGGGATGGACGAGAAAGTTGAAGCAACGCAAGTCCGGAA

GAAGTGCTGGCAAGTACGATGTGTACCTGATTAACCCCCAAGGAAAGGCTTTCAGGTCTAAAGTAGAG

CTCATAGCTTATTTTGAAAAAGTGGGCGACACCTCTCTCGACCCTAACGATTTCGATTTCACCGTCAC

TGGCAGAGGCAGTCCTTCTAGACGCGAACAGAAACCCCCTAAAAAACCCAAGAGCCCCAAAGCCCCCG

GAACGGGACGAGGTCGCGGCCGCCCAAAGGGTAGCGGAACAACAAGACCAAAAGCGGCTACATCCGAA

GGTGTGCAAGTTAAGCGCGTGCTGGAAAAAAGCCCAGGAAAGCTCTTGGTCAAAATGCCATTCCAGAC

CTCACCTGGTGGCAAGGCAGAAGGTGGGGGCGCGACAACGTCCACCCAGGTAATGGTGATCAAAAGAC

CAGGCCGCAAGCGAAAGGCAGAGGCAGATCCGCAAGCGATCCCCAAAAAACGGGGCCGAAAGCCAGGA

AGTGTTGTCGCTGCTGCCGCGGCCGAAGCAAAGAAAAAAGCTGTCAAAGAGAGCTCCATTAGGTCAGT

ACAGGAAACCGTGCTGCCAATCAAAAAAAGAAAAACTCGGGAAACCGTCTCCATAGAAGTAAAGGAGG

TTGTCAAGCCCCTTCTTGTTTCCACCCTGGGAGAAAAATCAGGAAAAGGTCTCAAAACGTGTAAAAGC

CCGGGTCGAAAGTCAAAAGAATCATCACCAAAAGGCCGGAGTAGTTCAGCCAGTTCTCCGCCAAAGAA

AGAACACCACCACCATCACCACCACTCAGAATCCCCTAAGGCCCCAGTGCCTTTGCTCCCACCCCTTC

CTCCGCCGCCGCCAGAACCGGAGAGTTCTGAAGACCCCACTTCACCTCCGGAGCCACAGGACCTGTCC

TCTTCAGTTTGCAAAGAGGAGAAGATGCCTCGAGGCGGATCTCTGGAATCTGACGGGTGCCCGAAAGA

ACCCGCCAAGACGCAACCGGCGGTAGCAACCGCAGCAACCGCGGCCGAGAAATATAAGCATCGAGGAG

AAGGAGAACGCAAAGACATCGTTAGCTCTTCTATGCCACGGCCAAACCGGGAGGAACCAGTGGATTCA

CGAACGCCGGTCACAGAACGGGTCAGCGATTATAAGGACGATGACGACAAATA

The LAMP2 protein sequence is:

(SEQ ID NO: 46)

MVCFRLFPVPGSGLVLVCLVLGAVRSYALELNLTDSENATCLYAKWQMNF

TVRYETTNKTYKTVTISDHGTVTYNGSICGDDQNGPKIAVQFGPGFSWIA

NFTKAASTYSIDSVSFSYNTGDNTTFPDAEDKGILTVDELLAIRIPLNDL

FRCNSLSTLEKNDVVQHYWDVLVQAFVQNGTVSTNEFLCDKDKTSTVAPT

IHTTVPSPTTTPTPKEKPEAGTYSVNNGNDTCLLATMGLQLNITODKVAS

VININPNTTHSTGSCRSHTALLRLNSSTIKYLDFVFAVKNENRFYLKEVN

ISMYLVNGSVFSIANNNLSYWDAPLGSSYMCNKEQTVSVSGAFQINTFDL

RVQPFNVTQGKYSTAEECSADSDLNFLIPVAVGVALGFLIIVVFISYMIG

RRKSRTGYQSVDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for LAMP2 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

LAMP2-DNA

(SEQ ID NO: 47)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG GTTTGTTAAAGAAGTGATTAATAAGACTGC

CACAGACCTACTGTTTCAGAATCTCTGGGGAATGAGGTACAGGAATCTGTATGTTAAAAAAAAAAAAC

CCAAAACCAGATGGTTCTTTTGGAAACTAACACTTGAGAATCAGTGCTTTATTCCAACTCAAGATTTT

TAAATGCTTTCATCTGTTGAATATTTTAGGTTTTGTCTGTTCATCTATCAAGTATAGCAAACAGTATG

CTTAGTATGAACTCATTACAGAATATTTGTGTGTATGTACATAGGAAAAATATCTGGAAGTATAGATG

TGGGTGTGTGTATATATGTGTGTAAATATATATAATAATGTTAATAGTAGGAATACAGATGATCTCTA

TTTTTCCGCTTTATCCTTCTGTTTTATTTCTCTCATTTTTTTCTTCCTGTGTTTAAAAAAGATGCTTC

TTGTTTCTGAGCTCAATTCCCCCACCTGAGAACAGGGTGCTGTAACTAGGTGAATTTCAAGATCATGT

CTTGTTCCGATGGTCCGTGACTCCATACTACCCCCTAGGTTCCCTCGTCCATCTCACAGCATGAACTG

CATAGGTAACAACAGTGTTTGGAGAATAAGCATGTATTATTTTTGTATGTATTACGTTTGAGATGCCT

AGCAACCGTTTTTGTGCCCGCAAAATTTTTCTCTTCTTTGTCAAGGAGCCAAAACGGAAATAGTCTAC

TATCTACGGCCCTCAAAAGTTCCTCCTCTCTCCTTAGCCTTAGAAAATACCGTCCGCCATAGGCATGG

GCAAAGACCTCATGGCTAAAACACCAAAAGCAATCGCAACAAAAGTCAAAATTGACAAATGGGATCTA

ATTAAAAGAACTTCTGCACAGCAAAAGAAATTAGCATTAGCGTGAACAGGCAACCTGCAGAATGGGAG

AAAATTTTTGCAATCTACCCATCTGACAAAGGTCTAATATCCAGAATCTACAAGGACCTTAAACAAAT

TTACAAGAAAAAAACAACCCCATCAAAAAGTGGGCAAAGGATATGACCAGACACTTCTCAAAAAAAGA

CATTTATGCAGCAACAAACATGAAAAAAAGCTCATTATCACTGATCATTAGAGAAAGGCAAATCAAAA

CCACAATGAGATACCATCTCACGCCAGTCAGAATGGTGATTATTAAAAAATCAGGAAACAATAGATGC

TGGCGAGGCTGAGGAAAAATAGGAATGCTTTTACACTGTTGGTGGGAGTGTAAATTAGTTCAACTATT

GTGGCAGACAGTGTGGCGATTCCTCAAAAATCTAGAACCAGAAATACCATTTGACGCATGCACACGTA

TGTTTATTGCAGCACTATTTACAATAGCAAAGACTTGGAACCAACCCAAATGCCCATCAATGATAGAC

TAGATAAAGAAAACGTGGCACATATATGCCATGGAATACTATGCAGCCATAAAAAAATGAGATCATGT

CCTTTGCAGGGACATGGATGAAGCTGGAAGCCATCATCCTTAGCAAACTAACACAGGAACAGAAAACA

AAACACTGCATGTTCTCACTCATAAGTGGGAGTTGAACAATGAGAACACATGGACACAGGGAGGGGAA

CAACACACACGGGGGCCTGTCAAGGGGTGGGGGCCAAGGGGAGGGAGAGCATTAGGAGAAATACCTAA

TGCATGTGGGGCTTAAAACCTAGATGACGGGTTGATAGGTGCAGTAAACCACCATGGCACATGTATAC

CTATGTAACAAAGCTGCACATTCTGCACATGTATCCCAGAACTTAAAGTAAAATTAAAAAAAAAAAAA

AAAAAGAAAGAAAAAGAAAAAGAAAATGCCGTCCGCGCATATCTCTCAGGAGCATAGGAAGGGCTGTG

AACCAAAAAGCCAGGGCGTGGTCGCCCCGCCCACCGGCCGCTACATCACCACCCCTCTCGCCGCTTTC

AAGCGCGGAGATTGGCTGTAAGCAAGAAAGAGCCCCGCCCC

ATGGTCTGCTTTAGACTGTTTCCAGTACCAGGTTCTGGACTCGTGCTGGTGTGTCTTGTACTG

GGTGCAGTCCGATCTTACGCCTTGGAACTTAACCTCACTGATTCTGAGAATGCCACTTGTCTTTACGC

AAAGTGGCAGATGAACTTTACGGTGCGGTACGAGACCACGAATAAAACTTATAAGACCGTAACGATAA

GCGATCATGGGACCGTCACTTACAACGGCTCCATCTGCGGGGACGATCAAAATGGCCCAAAAATCGCT

GTCCAATTCGGCCCAGGGTTCAGTTGGATAGCCAACTTCACTAAAGCTGCATCAACCTATAGCATAGA

TTCTGTATCCTTCTCTTACAATACCGGAGATAACACAACGTTTCCCGATGCAGAAGACAAAGGCATAC

TCACCGTGGACGAGTTGCTTGCTATTAGGATACCGCTTAACGACTTGTTTCGCTGCAATAGTTTGAGC

ACCCTTGAAAAGAATGATGTGGTTCAGCATTATTGGGACGTACTCGTTCAAGCTTTTGTCCAGAATGG

TACGGTTTCAACCAATGAATTTCTCTGCGATAAGGACAAAACTTCCACAGTTGCCCCAACTATTCACA

CGACCGTTCCAAGCCCCACAACGACCCCGACTCCGAAAGAAAAACCAGAGGCCGGAACCTACTCAGTG

AATAATGGTAATGACACATGCCTCCTTGCTACGATGGGGCTCCAGTTGAACATCACACAAGACAAAGT

TGCGTCTGTCATAAATATTAACCCCAATACCACCCATAGTACCGGGTCCTGTCGATCTCATACTGCGC

TTCTCAGACTTAATAGCAGCACCATCAAGTACCTGGATTTCGTTTTCGCTGTAAAAAACGAGAATCGG

TTTTATCTCAAAGAAGTAAATATTAGTATGTACTTGGTAAATGGTAGTGTGTTCTCTATAGCGAACAA

TAACCTCTCATACTGGGATGCGCCTCTTGGTAGTTCCTACATGTGCAACAAGGAGCAAACAGTGAGTG

TCAGTGGGGCATTTCAAATCAATACGTTTGATCTTAGAGTCCAGCCATTCAATGTGACTCAGGGGAAG

TACTCTACGGCGGAAGAGTGTTCCGCGGACTCCGATCTTAATTTTTTGATCCCTGTCGCCGTGGGGGT

CGCCCTGGGATTTCTGATAATTGTCGTTTTTATAAGCTACATGATAGGCAGGAGGAAGAGTCGGACGG

GATACCAGTCTGTGGATTACAAAGATGATGATGACAAATA

The NAGLU protein sequence is:

(SEQ ID NO: 48)

MEAVAVAAAVGVLLLAGAGGAAGDEAREAAAVRALVARLLGPGPAADFS

VSVERALAAKPGLDTYSLGGGGAARVRVRGSTGVAAAAGLHRYLRDFCG

CHVAWSGSQLRLPRPLPAVPGELTEATPNRYRYYONVCTQSYSFVWWDW

ARWEREIDWMALNGINLALAWSGQEAIWQRVYLALGLTQAEINEFFTGP

AFLAWGRMGNLHTWDGPLPPSWHIKQLYLQHRVLDQMRSFGMTPVLPAF

AGHVPEAVTRVFPQVNVTKMGSWGHFNCSYSCSFLLAPEDPIFPIIGSL

FLRELIKEFGTDHIYGADTFNEMQPPSSEPSYLAAATTAVYEAMTAVDT

EAVWLLQGWLFQHQPQFWGPAQIRAVLGAVPRGRLLVLDLFAESQPVYT

RTASFQGQPFIWCMLHNFGGNHGLFGALEAVNGGPEAARLFPNSTMVGT

GMAPEGISQNEVVYSLMAELGWRKDPVPDLAAWVTSFAARRYGVSHPDA

GAAWRLLLRSVYNCSGEACRGHNRSPLVRRPSLQMNTSIWYNRSDVFEA

WRLLLTSAPSLATSPAFRYDLLDLTRQAVQELVSLYYEEARSAYLSKEL

ASLLRAGGVLAYELLPALDEVLASDSRFLLGSWLEQARAAAVSEAEADF

YEQNSRYQLTLWGPEGNILDYANKQLAGLVANYYTPRWRLFLEALVDSV

AQGIPFQQHQFDKNVFQLEQAFVLSKQRYPSQPRGDTVDLAKKIFLKYY

PRWVAGSWDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for NAGLU and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

NAGLU -DNA

(SEQ ID NO: 49)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG GGACAACATGGTGAAATCCCATCCCTACAA

AGGTTAACTGGGCTTGGTGGCACGTGCCTGTAATCCCAGCTACTCAGGAGACTGAGACAGGAGAATGG

CTTGAACCTGGGTGGCTGAGGTTCCCGTGAGCTGAGATTAAAAAAAAAAATCATACTTCCCTAGTATC

TAAAGTTGATATCTGATTCCAGGCCTCTTTCAACTTTTTTTGTTTGTTTGTTTGTTTTTTTGATATGG

AGTCTCGCTCTGTCATCCAGGCTGGAATGCAAATGGCACGATCTTGGCTCACTGCAACTTCCACCTCC

AGCGTTCAAGCGATTCTCCTGCCTCAGCTTCCCAAGTAGCTGGGACTACAGGCGTGTGCCACCAAGCC

TGGCTAACTTTTTTTCTTTTTTTTTTTTTTGAGAGGGAGTCTTGCTCTGTCCCCCAGGCTGGAGTGCA

GTGGTGTGATCTTGGCTCACTGCAACCTCCGCCTCTCCGGTTCACGCCATTCTCCTGCCTTAGCCTCC

CGAGTAGCTGGGAGTACAGGCGCTCGCCAACACGCCCGGCTAATTTTTTGTATTTTTAGTAGAGATGG

AGTTTCACCGTGTTAGCCAGGAGGGTCTTGATCTCCTGACCTCGTATCCGCCCGTCTTGGCCTCCCAA

AGTGCTGGGATTACAGGCGTGAGCCACCGCGCCCAGCACCTGGCTAACTTTTGTATTTTTAGTACAGA

CGGGGTTTCACTGTATGTTGGCCAGGCTGGTCTCAAACCCCTGACTTGAGGTGATCTGCAAGCCTCAG

CCTCCCAGAGTGCTGGGATTACAGGCGTAAGCCACCGCTCCTGGCCTAAGGTTGGCTATTTTTATGGT

TATTTCTTGATTATATGATAAACAAGGGGTGGGTTAGTAATGAATTTTTCAGAAAAGGGGTGGGGATC

CCCCCCAACTGAAGGTTCCTCCACTGTTTAGACCATATAGGGTAACTTCTGGACGTTGCCATGGCATT

TGTAAACTGCCTGGCGCTGCTAGGAGTGTCTTTAGCATACTAATGCATTATAATTAGCGTATAATGAG

CAGTGAGGACGATCAGAGGTCACCTTCCTGTCTTGGTTTTGGCAGGTTTTGACCAGTTTCTTTGCTGC

ATTCTGTTTTATCAGCGGGGTCTTGTGACCTTTTATCTTGTGCTGACCTCCTGTCTCATCCTGTGACG

AAGGCCTAACCTCCTGGGAATTCAGCCCAGCAGGTCTCTGCCTCATTTTACCCAGCCCCTGTTCAAGA

TGGAGTCGCTCTGGTTGGAAACTTCTGACAAAATGACAGCTCCTGTTATGTTGCTGCTGCTGCCGCCA

ATGGACAGCCTTTAACGTGCCCGCCAGCCCTGCTCCACCGCCGGCCTGGGCTCACATGGCCCCATCCC

TCCTCGAACCTCCTAGCCTGTTAGTTACTCAAATCTGCAAGCTCTCTGCCTTCTCAGGGCCTTCAATA

AATGCATTTCTTCTGTCTGGAAGGCTCTTCCTTTCCCTCTTCTAGCCAATTCCTATTCATCCCTGAGT

TTCAGATTAAAAGTCACTTCCTTTGGAAACCTTACTTCGCTACTTCGCTACTTACTGCACTACTTCGC

AGCATCACAACTATGATGGAAATCCTTACTTACGTTAAATATCTGGTTTCTAGGTCACCTCCCTGACG

GGGACGGTAGGGACCGTCTTCTCGTTCATCAGTAGGGAAGTAGCTATGGCAGTGCCTGATACAAAATA

AACTCCAAATGTGTATTTATTAGATGGTTGGATGGAAGTTATTTGCGTGTGAAAGCGCGTTTTACCCG

AAGGCGCTCTGTGAGGGCCAGCGGGTCCCCTTCGGCCCTGGAGCCGGGGTCACACGCTCCCCACCGCG

TGCGGTCACGAGACGCCCCCAAGGGAGTATCCTGGTACCCGGAAGCCGCGACTCCTGGCCCTGAGCCC

GGGCTTAGCCTTCGGGTCCACGTGGCCGGAGGCCGGCAGCTGATTGGACGCGGGCCGCCCCACC

ATGGAGGCGGTAGCCGTTGCCGCTGCCGTCGGAGTACTT

TTGCTCGCAGGAGCAGGTGGTGCAGCTGGTGACGAGGCTCGGGAGGCAGCGGCCGTACGCGCACTGGT

AGCTCGGCTGCTGGGGCCAGGTCCCGCAGCTGACTTTAGCGTGAGTGTGGAGCGGGCCCTCGCTGCGA

AGCCAGGCCTTGATACCTACTCTCTGGGCGGGGGAGGAGCGGCACGCGTGAGAGTTCGGGGAAGTACC

GGGGTGGCTGCGGCGGCGGGCTTGCATCGGTATCTTAGGGACTTTTGTGGGTGTCACGTCGCCTGGTC

AGGTAGCCAACTCAGGCTGCCACGACCTTTGCCAGCAGTACCCGGCGAGTTGACGGAAGCGACGCCAA

ATAGGTATAGGTACTATCAAAACGTCTGTACCCAGTCTTATTCTTTTGTATGGTGGGATTGGGCTCGC

TGGGAACGAGAAATAGACTGGATGGCACTTAACGGCATCAATCTTGCGTTGGCCTGGAGCGGGCAGGA

GGCAATCTGGCAGCGAGTGTATCTCGCATTGGGGCTCACACAGGCTGAAATAAATGAGTTTTTTACCG

GTCCCGCCTTCTTGGCTTGGGGCAGGATGGGTAATCTCCATACGTGGGATGGACCGCTCCCACCTTCA

TGGCATATAAAGCAGCTGTACCTGCAACATAGGGTGTTGGACCAAATGAGGAGCTTTGGAATGACACC

GGTACTTCCGGCATTCGCCGGTCACGTTCCTGAAGCGGTCACGCGAGTATTTCCGCAAGTGAATGTAA

CAAAGATGGGTTCATGGGGACACTTCAATTGTTCATATTCATGCTCTTTCCTGCTTGCGCCGGAAGAT

CCTATTTTTCCTATCATTGGAAGTCTGTTCCTTCGGGAATTGATCAAGGAGTTTGGAACAGATCACAT

ATATGGAGCAGACACTTTTAACGAAATGCAACCTCCGTCTAGCGAGCCCTCTTATCTGGCAGCTGCGA

CCACGGCGGTGTATGAAGCGATGACAGCTGTCGATACGGAAGCAGTCTGGCTCCTTCAAGGTTGGCTG

TTTCAACACCAACCGCAATTTTGGGGACCAGCCCAGATTAGGGCTGTGTTGGGTGCTGTGCCCCGGGG

TAGGCTCTTGGTGTTGGATCTTTTTGCGGAGAGTCAGCCTGTGTATACCCGAACGGCGAGCTTTCAAG

GTCAGCCGTTTATCTGGTGTATGCTTCATAACTTTGGCGGGAACCATGGTCTTTTCGGTGCCCTTGAA

GCCGTAAATGGGGGGCCAGAGGCTGCTCGATTGTTTCCTAACAGCACTATGGTAGGAACAGGTATGGC

ACCCGAGGGTATTAGTCAAAATGAGGTCGTATACAGTTTGATGGCAGAACTTGGGTGGCGCAAAGATC

CCGTACCTGATCTGGCAGCGTGGGTTACGAGCTTCGCGGCTCGAAGGTACGGAGTAAGTCACCCAGAC

GCTGGGGCCGCCTGGCGGTTGCTCCTGCGATCCGTCTACAACTGCTCAGGTGAGGCATGCCGCGGGCA

CAATAGAAGCCCACTCGTCCGACGACCATCCCTTCAAATGAACACTAGTATCTGGTACAACAGGTCTG

ACGTATTCGAGGCATGGCGCCTCCTCCTCACTAGCGCACCGAGTCTGGCTACCTCCCCCGCGTTTAGG

TACGACCTTCTGGACTTGACCAGACAAGCTGTGCAGGAGCTCGTGTCACTTTACTACGAAGAGGCCAG

ATCTGCATATCTGTCTAAGGAACTGGCATCCTTGCTTAGAGCGGGTGGGGTTCTGGCTTACGAACTCC

TTCCAGCACTGGACGAGGTGCTCGCGAGTGACAGTAGGTTTCTCCTCGGGTCATGGCTGGAACAAGCA

AGGGCCGCGGCCGTTTCTGAAGCCGAGGCTGACTTTTACGAACAGAACTCTCGATACCAGTTGACGCT

GTGGGGACCCGAGGGGAATATCTTGGATTACGCGAACAAGCAGCTGGCAGGTCTGGTAGCCAACTACT

ATACGCCGAGGTGGAGGCTCTTCCTCGAGGCTCTTGTCGATTCCGTCGCCCAAGGGATACCCTTTCAG

CAGCACCAATTCGACAAGAACGTTTTTCAACTGGAACAGGCTTTCGTACTTTCCAAACAACGCTATCC

CAGCCAGCCTCGGGGTGACACAGTTGACCTGGCAAAAAAAATCTTCCTTAAATACTATCCAAGATGGG

TCGCCGGTAGTTGGGATTATAAAGATGATGATGACAAGTA

The ARSB protein sequence is:

(SEQ ID NO: 50)

MGPRGAASLPRGPGPRRLLLPVVLPLLLLLLLAPPGSGAGASRPPHLVFLL

ADDLGWNDVGFHGSRIRTPHLDALAAGGVLLDNYYTQPLCTPSRSQLLTGR

YQIRTGLQHQIIWPCQPSCVPLDEKLLPQLLKEAGYTTHMVGKWHLGMYRK

ECLPTRRGFDTYFGYLLGSEDYYSHERCTLIDALNVTRCALDFRDGEEVAT

GYKNMYSTNIFTKRAIALITNHPPEKPLFLYLALQSVHEPLQVPEEYLKPY

DFIQDKNRHHYAGMVSLMDEAVGNVTAALKSSGLWNNTVFIFSTDNGGQTL

AGGNNWPLRGRKWSLWEGGVRGVGFVASPLLKQKGVKNRELIHISDWLPTL

VKLARGHTNGTKPLDGFDVWKTISEGSPSPRIELLHNIDPNFVDSSPCPRN

SMAPAKDDSSLPEYSAFNTSVHAAIRHGNWKLLTGYPGCGYWFPPPSQYNV

SEIPSSDPPTKTLWLFDIDRDPEERHDLSREYPHIVTKLLSRLQFYHKHSV

PVYFPAQDPRCDPKATGVWGPWMDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for ARSB and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

ARSB-DNA

(SEQ ID NO: 51)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG AAATATTTATTAATTACAAGAAGAAAATAT

TAACCTTACAATGAGGAAATGTTTACCTTAACAAAGTTATCCCATAAGAAAGCAATTCAGCATCATGT

GCCTCTTAATAGGAAGCACCGAGAAACATACCACATCACTTTTTGTGGTATTCCTGCACAAATCCATA

ACCTCTATCTAATTGTGAAGAAACATCAAACTCCAATTGAGAGATTTTATAAAATAACTAGCTTGTAC

TGCACATTATGTGTTCAATACATAGATGGAAAATAGAATTAACTCTTCAATTTGCACATTTCCAGAGC

ACTATTAACATATGCTTTTATTTTAAAAACATAGGACGTAAGAATCTGTGCAAGTTTAGCTAGTTTCA

GGGGAAGTTGGTTAAAGGAGGAATTGTTCCTGCAGATTAGCACCCATCAATTATTTCTTCTTAATTTT

TATCAAGGAGAATAGGAAGGATTACGGGGGAGAAAGACACAAAACCACAGCGGAAATTCTCAGTCCTT

CAAGGTCTTCTACAGGGTGTTTAAATGAAAAGTTCAACCCTTGACATAATGAGTCAAACAGTTCTCTC

AAAAGTTATTCTTTGAATTTAATGCTAAAATTTTCACTTTTTTCTGCTAAATTTCATTGATTCCACTA

GAGATTAAAGTGCATACAACATAAACTAATCTACTTCATGCAAAGAAAAAAAGTGTATGTAAGAGGCA

AACATTTCATGTGTTAAAGGATTCTTGGGGAAAAAAAGGTAAAATCATGACCCTCATACAAATATAAA

AGTGAATACATGATTTTATTTAACTCATTAATAAGGAAATTGGTAAGGTGTTAAAACCAATTCAAAGG

ACAATCCAAAGAACAGATCAGGAATACTAAAATAAATATGCAAGCGGAGGTGAAACTGTTTTCCTTGG

TAGTGGTGGAGGGGAAGGATTGCTACTCCGCTGGATAAAGTTCATTTGTGTATATATAAATAAGAATT

ATTTTCCATTGTTATTTATCTATAACTTATAAAGTTGTAAACAACTTCCACGGAATCAGACTCAACCT

GGAAGGGTATGGTCTCTAGGCAATGCAAAAATTTTCCCCTACACCTGTTAACAACTATAATATCTCCA

GACAGAGTAGACAGAAAGTCTGGATGGCAACGGGAATCTACTGGTCATACGGCTAACTTCCTAATTCA

ATAAGCACGTGACTAAAGGATTTTTTCCTTCCACTCAGATATTTCAGGCTAACTAGATACTGTGTGCT

TCTTAGTGTCACTGCTTAGTGGGGGAGCCAGCTCTGAGTGGGGTCATATCCGGACAAGCGAATGAGCT

ATTTATTCAATGACCACGCAACACTCCAAATCCTCCCAGGGCAACTTGAAAGTAACCGCACCTTCCAA

AGGGCACCGTGCAATCAGACTGTGTGTTTGGCCTCCTGTTTGCTAGTGGGGAGGAAGCGGCTTCATGG

GTGTACACTACGCATAAATGAATGTGAAAGGCTATTTAGACCTCTGCCTTTTCACCGTCCTCCCACCT

GCCACAGGCTGGGCTCTTGTGCTAGAAATGACTTGCTAGCTAGACATCATGGTTCAGGATCTGAGTCA

GAGGTTTAACCATTTATAAGCTTTTTTCTTATGAAAAATTGGCACTAATTATAATGTCTAACTGTCAG

AGTTGTTGCAGGCTTTACAGGAGACGCGGGCTGTGAAGATGCTTTGTAAATTGTGAAGCGTTATTAAA

GAACACATCTTTTTTTTTTAGGAAACCACAGTGCAAATTTAATTGCCGGGGAAGATAACGGGCCTTGG

TGCCCTCCAAGCGTCAGCTGAGTTTCCAAGAAGCCGGGCAGCGGGCGCCCGCGGGTTCGTCTCTGGCT

CCTCCTCCGCCACAGCAGCCGGGGGCCCGGGTCGGAGGCGGCGGGGGCCGAGCGCCCGGCCTCGCAAG

CCCACGGCCCGCTGGGGGTGCCGTCCCGCGCCGGGGCGGAGCAGGCCCCGGCAGCCCAGTTCCT

ATGGGCCCCCGGGGCGCAGCGAGCCTCCCCAGAGGGCCAGG

ACCAAGGAGGCTCCTTTTGCCAGTCGTCTTGCCGCTTTTGCTCCTTTTGCTTCTCGCGCCACCCGGCA

GTGGGGCAGGAGCTTCTCGCCCACCTCACCTTGTCTTCCTGCTTGCTGACGACCTTGGATGGAATGAT

GTGGGTTTTCACGGTTCCCGGATTCGAACACCACACTTGGATGCTCTGGCTGCGGGGGGCGTACTGCT

CGATAATTATTACACACAACCGTTGTGCACACCGTCTCGGAGTCAGCTTCTGACGGGACGATATCAAA

TAAGGACTGGGCTGCAACATCAGATTATATGGCCATGTCAACCCAGTTGCGTGCCCCTTGACGAGAAA

CTCCTGCCTCAGCTGTTGAAGGAGGCTGGATACACGACACATATGGTCGGCAAGTGGCATCTGGGAAT

GTACAGGAAAGAGTGTCTCCCAACCCGGCGCGGATTCGATACGTATTTTGGGTACTTGTTGGGCTCAG

AAGATTACTATTCACACGAAAGGTGCACTCTTATCGACGCACTCAACGTTACGCGCTGCGCACTTGAT

TTCCGAGATGGAGAGGAAGTTGCAACTGGGTATAAGAATATGTATTCAACAAACATCTTTACTAAGCG

GGCAATAGCCCTCATTACTAATCATCCCCCCGAAAAACCGCTGTTTCTCTATCTCGCCCTGCAGTCAG

TCCATGAGCCGCTGCAGGTGCCTGAGGAGTACCTCAAACCATACGACTTCATACAAGATAAGAACCGA

CACCATTATGCGGGTATGGTCTCTCTGATGGACGAGGCTGTCGGGAATGTTACTGCAGCATTGAAATC

TTCCGGCCTTTGGAACAACACAGTCTTCATATTTTCTACGGACAATGGCGGGCAGACTCTTGCCGGTG

GCAATAACTGGCCTCTTAGGGGTCGCAAGTGGTCCCTCTGGGAAGGAGGCGTCCGCGGTGTAGGGTTT

GTAGCGAGCCCCCTCCTTAAACAAAAAGGAGTTAAAAACAGGGAACTTATTCACATTTCCGATTGGTT

GCCCACGCTCGTCAAGCTCGCGAGGGGTCATACAAACGGGACAAAACCGCTTGACGGGTTTGATGTAT

GGAAAACCATTTCTGAGGGGTCTCCTAGCCCCAGGATCGAGCTGTTGCATAACATAGACCCAAACTTT

GTAGACTCCTCACCCTGTCCCCGAAATTCAATGGCCCCTGCCAAAGATGATTCATCCCTCCCTGAGTA

CTCCGCTTTTAACACAAGCGTACATGCCGCTATTAGGCACGGCAATTGGAAGCTGCTGACCGGGTATC

CTGGTTGTGGTTATTGGTTCCCTCCCCCCTCACAATACAACGTGTCTGAAATACCGAGTAGTGATCCG

CCGACAAAAACCCTGTGGCTGTTCGATATTGATCGAGACCCTGAAGAACGCCACGATCTCTCCCGGGA

ATACCCCCACATTGTCACCAAGCTTCTTTCACGATTGCAATTTTACCACAAGCATTCAGTACCCGTTT

ACTTCCCCGCCCAGGACCCAAGGTGCGATCCTAAAGCAACGGGGGTTTGGGGACCATGGATGGACTAC

AAAGATGATGACGATAAATAG

The GUSB protein sequence is:

(SEQ ID NO: 52)

MARGSAVAWAALGPLLWGCALGLQGGMLYPQESPSRECKELDGLWSFRAD

FSDNRRRGFEEQWYRRPLWESGPTVDMPVPSSFNDISQDWRLRHFVGWVW

YEREVILPERWTQDLRTRVVLRIGSAHSYAIVWVNGVDTLEHEGGYLPFE

ADISNLVQVGPLPSRLRITIAINNTLTPTTLPPGTIQYLTDTSKYPKGYF

VQNTYFDFFNYAGLQRSVLLYTTPTTYIDDITVTTSVEQDSGLVNYQISV

KGSNLFKLEVRLLDAENKVVANGTGTQGQLKVPGVSLWWPYLMHERPAYL

YSLEVQLTAQTSLGPVSDFYTLPVGIRTVAVTKSQFLINGKPFYFHGVNK

HEDADIRGKGFDWPLLVKDFNLLRWLGANAFRTSHYPYAEEVMQMCDRYG

IVVIDECPGVGLALPQFFNNVSLHHHMQVMEEVVRRDKNHPAVVMWSVAN

EPASHLESAGYYLKMVIAHTKSLDPSRPVTFVSNSNYAADKGAPYVDVIC

LNSYYSWYHDYGHLELIQLQLATQFENWYKKYQKPIIQSEYGAETIAGFH

QDPPLMFTEEYQKSLLEQYHLGLDQKRRKYVVGELIWNFADFMTEQSPTR

VLGNKKGIFTRQRQPKSAAFLLRERYWKIANETRYPHSVAKSOCLENSLF

TDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for GUSB and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

GUSB-DNA

(SEQ ID NO: 53)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG TGCCCAGCTAAATATGGAGATGCGGTTTCT

CCATATTGGTCAGGCTGGTCTCGAACTCCCAACCTCAGGTGATCTGCCTGCTTCAGCCTCCCAAAGTG

CTGGGATGACAGGCGTGAGCCACCGCACCTGGCCAAGGCACTTGGTTTCTTAGATCACCCACTTGGCC

CTCTTCCAAGTTGTACTTTCCTTCTTTCCTTCCTGTTCTAAAGTTTTTTAATATTTTTTTTTTTCCTG

AAATGGCATCTCACTCTGTAGCCCAGGCTGGAGTGCGGTGGCGTGATCTCAGCTCACTGCAACCTCTG

CCTCCTGGGTTAAAGCATTTCTCCTGCCTCAGCCTCCCAAATAGTAGGGATTACAGGTGCCTGCCACC

ACGCCTGGCTAATTTTTGTATTTTTAGTAGAGATGGGGTTTCTCCACGTTGGCCAGGTTGGTCTCAAA

CTCCTGATATCAAGTGATCCTCCCGCCTTGGCCTCCCAAAGTGCTGGAGTTTGGAGTTACAGGCCTGA

ACCTCTGTGCCTGGCCTGCTTTTTTTTTTAGACTGAGTCTTGCTCTCTCGCCAGGCTGTAGTGCTGTG

GTGCGATCTCGGCTGACTGCAACCTCTGCCTCCCGCGTTCAATCGATTCTCCTGCCTCAGCCTCCCGA

GTAGCTGGGACTACGGGCGCATGCCACCATGCCCAGCTAATTTTTTAGTAGAGATGGGGTTTCATCAT

ATTGGCCAGGCTGGTCTCAAACCCCTGACCTCGTGATCCCCCCACCTCCGCCTCCCAAAGTGCTGGGA

TTACAGGTGTGAGCCACTACGCTCAGCACTGGCCTGCTTTTTAACAAGCTTTTACTACTGCTCTGCAG

CTTACCTCCCTCTGGCTTTCTGCCTTATGCCCCTCAGTGCAATTCTTTCTTCTGAGGAGGCAAGAATT

GAGGTTGTTGCTGATGTGTAGGGATTCACCACCCGGAACTCCACCAGTAACAGGTCGAGGATGCTAGT

TGAAAATGTTATGTAAGCTGCATGCTTTTTACAAATGGTAGTGGTTCTCATGTCCAGCAATGGCCACT

GCACCGTCCCTGTATGAAAGTCCCCTCCATAAATCTATGTCTCCTTTGCTGTCTCCAGATCTCCTCCT

CTACCTCTCCCACACGGTGCCTTCCCTACTGGAGTAAACTGGGGTCCCGCAGGGCACCCTTTCCCTGC

AATTTTTTTTTTTTTTTTGAGACAGAGTCTCGCTCTGTTGCCCAGGCTGGAGTGCAGTGGTGCGATCT

CGGCTTACTGCAGCCTTTGCCTCCCGGGTTCAAGCGATTCTCCTACCTCAGCTTCCTGAGTAGCTGGG

ATTACAGGCACGCACCATCACGCCCCGCTAATTCTTTTTTTTTTTTTTGTATGTTTACAAAATAGGGA

CGGGGTTTCACTATCGTTGGCCAGGCTGGTCTCGAACTCCTGGCCTCAAGTGATCTGCCCCCCTCGGG

TTCCCAAAGTGCACCTGGCCTCTCTCCCTGCATCTTTATGTGCCTGCAACACCAAGAGGGAGCTCTTG

GCCCCTGGGTTAAGGTCAGGGCTCCAGAAGCCCAGGGTCAAGGCTATGGCCGCATCCCAAGGACGTGC

ATCCCTGCAAGAGGCTGCATCAGACAGGCATGGAAGAGGCGCCGCTGGGGCCTCCTTGAATTCCTGCT

GGGAAAAGCAAGTGGAGGTGCTCCTTGAAGAAACAGGGGGATCCCACCGATCTCAGGGGTTCTGTTCT

GGCCTGCGGCCCTGGATCGTCCAGCCTGGGTCGGGGTGGGGAGCAGACCTCGCCCTTATCGGCTGGGG

CTGAGGGTGAGGGTCCCGTTTCCCCAAAGGCCTAGCCTGGGGTTCCAGCCACAAGCCCTACCGGGCAG

CGCCCGGCCCCGCCCCTCCAGGCCTGGCACTCGTCCTCAACCAAGATGGCGCGGATGGCTTCAGGCGC

ATCACGACACCGGCGCGTCACGCGACCCGCCCTACGGGCACCTCCCGCGCTTTTCTTAGCGCCG

ATGGCGAGAGGCAGTGCAGTAGCCTGGGCTGCTTTGGGACC

ATTGTTGTGGGGGTGCGCTCTCGGACTTCAGGGCGGTATGCTCTACCCTCAAGAGAGTCCCTCCAGGG

AATGTAAGGAGTTGGACGGCCTCTGGTCCTTCAGAGCCGATTTCAGTGACAATCGCCGCAGAGGGTTC

GAGGAGCAGTGGTACCGCCGACCGCTCTGGGAGTCAGGCCCAACCGTAGACATGCCAGTCCCATCTTC

ATTCAATGACATAAGCCAGGACTGGAGGCTGCGACACTTCGTCGGGTGGGTATGGTATGAGCGCGAAG

TGATCCTCCCTGAACGGTGGACTCAGGACTTGCGGACGAGGGTAGTACTTCGGATAGGTAGTGCTCAC

TCCTATGCTATTGTCTGGGTGAACGGAGTGGATACTCTGGAGCATGAGGGTGGCTATCTTCCTTTTGA

AGCTGACATCAGTAACCTGGTGCAAGTGGGGCCGCTGCCTTCCAGACTGCGAATCACCATCGCTATCA

ATAATACTCTGACCCCCACTACGTTGCCGCCAGGCACCATCCAGTATTTGACCGACACCTCTAAATAC

CCCAAGGGCTATTTTGTGCAAAACACTTACTTTGATTTCTTCAACTATGCCGGCCTGCAAAGATCTGT

GCTGCTTTACACCACTCCGACGACCTATATAGATGACATAACCGTGACGACGAGTGTCGAACAAGACT

CAGGTCTCGTCAACTACCAGATTAGTGTAAAAGGCAGTAATCTTTTTAAGCTCGAAGTGAGGCTTTTG

GATGCTGAAAATAAGGTGGTGGCAAATGGAACGGGCACACAAGGCCAACTTAAAGTGCCGGGGGTAAG

TCTGTGGTGGCCGTATCTTATGCATGAACGACCCGCTTATCTCTACAGTCTCGAAGTCCAATTGACGG

CACAAACAAGTCTTGGTCCTGTGAGTGATTTCTATACGTTGCCTGTGGGCATAAGGACTGTAGCTGTG

ACAAAGTCCCAATTTCTTATAAACGGTAAGCCCTTCTACTTTCATGGCGTCAACAAACATGAGGACGC

TGATATTCGCGGAAAAGGGTTTGATTGGCCTCTCCTGGTCAAGGACTTCAACCTTCTCAGATGGTTGG

GCGCGAACGCCTTTAGAACAAGCCACTATCCGTATGCAGAGGAGGTCATGCAAATGTGCGACAGATAT

GGGATTGTTGTAATCGATGAATGTCCCGGCGTGGGCTTGGCGCTCCCCCAATTTTTTAATAACGTGTC

TCTTCACCACCATATGCAAGTAATGGAGGAGGTAGTACGCCGCGACAAGAACCACCCCGCGGTAGTTA

TGTGGTCAGTAGCGAATGAACCAGCTTCTCATCTGGAAAGTGCCGGTTACTACCTCAAGATGGTGATA

GCCCATACAAAGTCTCTCGACCCTTCCCGCCCCGTGACATTCGTATCAAACAGCAATTATGCAGCCGA

TAAAGGCGCCCCATACGTAGACGTTATATGTCTTAATAGCTATTACTCCTGGTATCACGACTACGGTC

ATTTGGAACTCATTCAGCTGCAGCTCGCCACGCAATTCGAGAACTGGTACAAGAAATATCAGAAGCCA

ATTATACAGAGTGAATATGGTGCTGAAACAATCGCTGGATTCCATCAAGACCCACCATTGATGTTTAC

CGAGGAGTACCAGAAAAGTCTCCTCGAGCAATATCATCTTGGACTGGACCAAAAGCGACGAAAATACG

TAGTAGGGGAACTTATTTGGAATTTTGCCGATTTTATGACGGAACAGAGCCCTACACGAGTTTTGGGG

AACAAAAAGGGCATTTTCACCCGGCAACGGCAACCCAAGAGTGCCGCCTTCCTTCTTAGAGAAAGATA

CTGGAAGATTGCTAACGAAACACGCTATCCCCACTCTGTTGCTAAGAGCCAATGCTTGGAAAACTCCT

TGTTTACAGACTACAAGGATGATGACGATAAATA

The SLC19A3 protein sequence is:

	(SEQ ID NO: 54)
	MDCYRTSLSSSWIYPTVILCLFGEFSMMRPSEPF

	LIPYLSGPDKNLTSAEITNEIFPVWTYSYLVLLL

	PVFVLTDYVRYKPVIILQGISFIITWLLLLFGQG

	VKTMQVVEFFYGMVTAAEVAYYAYIYSVVSPEHY

	QRVSGYCRSVTLAAYTAGSVLAQLLVSLANMSYF

	YLNVISLASVSVAFLFSLFLPMPKKSMFFHAKPS

	REIKKSSSVNPVLEETHEGEAPGCEEQKPTSEIL

	STSGKLNKGQLNSLKPSNVTVDVFVQWFQDLKEC

	YSSKRLFYWSLWWAFATAGFNQVLNYVQILWDYK

	APSQDSSIYNGAVEAIATFGGAVAAFAVGYVKVN

	WDLLGELALVVFSVVNAGSLFLMHYTANIWACYA

	GYLIFKSSYMLLITIAVFQIAVNLNVERYALVFG

	INTFIALVIQTIMTVIVVDQRGLNLPVSIQFLVY

	GSYFAVIAGIFLMRSMYITYSTKSQKDVQSPAPS

	ENPDVSHPEEESNIIMSTKLDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for SLC19A3 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

SLC19A-DNA

(SEQ ID NO: 55)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCG CTATTACCATGCTATGCATGAATGAATGAATTCCTCAATAT

CTCTCTCTGATTTAGACTGTGGCTATGAGATATTAGAGAATCAGAGTAAACTTCTCAAGGTCCAATTT

GAACTCATTTTCTTCAAGAGGTGTGAAGGAAATCATGACACCTTCAGCCTTTAAGAAAATTTCACAAT

ATTCTTTGGGAGGACCCAGCAATTTGCAAGGTTGCACAATGAGCAGGGCACTCCTACACCTAAGGTTC

AGCTCAGGACTTTTCACAAATCATAGGAAGATGAAAATAGAAAACGCAGAGTCGTAGGACAGAGAACC

AAGACACCTCGGGGTGTTTTAATCCATTTCACTTCCACTGGTAGCCCTTAGAAGCCACGCAGTGTTTA

TCTTTAGGGAGGGTTTTTCAAGTCCACCAAGAGATGAACAGAAATAAAAATATTTTTTCCATTTTAAC

CAATAAGAACCTTGTAATGCTTTAAAAAAATTCTCTGTTTAATAAGAAGTTTCACTTTAATATATAAA

CTCTTTGCAGGCTAAGCGTAGTGGCTCATGCCTGTAATCCCAACACTTTGGGCGGGCAAGGCAGGAGG

ATCCTTTGAGCCCAGGAGTTCAAGACCAGCCTGGGCAAGATAGTGAGACTCTGTCTCTACCAAAAAAA

AAAAACAAAAAAATTAATGTGACGTGGTGGCACCCACCTGCAAGCCCAGCTACTTGGGAGGCTGAGGC

AGGAGGATCAGTGTGCCTGGGAGATTGAGGTTGCAGTGAGCTGGGGTTGCACCACTGCACTCCAGCCT

GGGAGATAAAGTGAGACTTTGTCAAAAAAAAAAAAAAAAGAAAGAAAGAAAGAGAAAAGAAAAGAAAA

GAGGCCAGGTGGCCGGGTGCTGTGGCTCACACCTGTAATCCTAGCACTTTGGGAGGCCGAGGCGGGTG

GATCACCTGAGGTCAGGAGTTTGAGACCAGCCTGGACAACATGGTGAAACCCCATCTCTACTAAAAAT

ACAAAAATTAGCTGGGTGTGGTGACGGGCGCCTGTAATCCCAGCTACTAGGGAGGCTGAGGCAGAAGA

ATTGCTGGAACCCGGGAGGTGGAGGTTGCAGTGAGCCAAGATCGTGCCATTGCACTCCAGCCCGGGGC

CAACAACAGCGAGGCTCCATCTCAAAAAAAAAAAGAAAGAAAGAAAGAAAGAAAAAAGACCAGGCGCG

GTGGCTCACGCCTGTAATCTCAGCACTTTGAGAGGCCGAGGCAGGCGGATCACGAGGCCAGGAGTTCG

AGATGAGCCTGGCCAATATGGTGAAACCAAACCCCGTCTCTACTAAAAACACAAAAATTAACTGGGCG

TTAATTTTTGGAGGCTGAGGCAGGAGAATCGCTTGAACCCGGGAGGCGGAGGGTTGTAGTGAGCCGAG

ATCGCGCCACTGCACTCCATCCTGGGCGACAGAGGGAGACTCTGTCTCAAAAAAAAAAAAAAGAAAGA

AAGAAAAGAGAAAAGAAAAGAAAAAGAAGAAGAAAAGAAAAAATTGTTTGTATCAGATTTTCAAAGTG

CCAGGAAACCATCCCACTCTACCTCCTTTAAAAAAAAAAAGTGCAGAGTGATTATAACCACTGGCTCT

GGAGCCAGGAGCCCCAACTAAAACCCAGGCTTCTCTATTTACTAGGCCTACTTCCTTCCGCCTCACTT

TCTTCATGTGTAGAGTGGGGGTGACAGCCAGCCAGCCCTCTCTCCTAAGCTTTTGGAGAGAAATAAAT

GGATTCATAAGAAAGGACATATTTGTAAATAAAGCAAACGATCGCTGTTGGATTCAGGCCCTCTTGCT

TCATTATGCTGAATGTCAGTGTATTACTTAAGTTGTTCCTTTCAGCAGAGTTGACTGTTTCTGCCCAG

AAAACCGGCCCTGGGCCAGGAAAATCTGGGCTTCCACACTGGGATGAAAGGTCTGGACGCCCGGATTC

GCATATGCAAAGCGTGGGGGCGTGGCCCCGGGCTCCGGGCCAGGCAGGCTCCGCCCCCTTCCCACC

ATGGACTGTTACCGGACCTCACTCTCTAGCAGTTGGATC

TACCCCACAGTCATCTTGTGCCTCTTTGGATTTTTCTCCATGATGCGGCCAAGCGAGCCGTTCCTTAT

ACCATACCTTAGCGGACCCGACAAAAACCTTACGAGCGCGGAAATTACGAACGAGATCTTCCCGGTAT

GGACCTACAGTTATCTTGTGCTTTTGTTGCCTGTCTTTGTCCTGACTGATTACGTTCGGTACAAGCCA

GTTATAATACTGCAGGGGATTTCCTTCATCATTACCTGGTTGTTGCTGCTCTTTGGACAGGGCGTAAA

AACTATGCAAGTAGTAGAGTTTTTCTACGGCATGGTTACCGCCGCAGAAGTGGCCTACTATGCCTACA

TCTACAGTGTGGTAAGCCCAGAGCATTACCAAAGAGTGAGTGGATATTGTAGATCAGTTACCCTTGCC

GCCTACACCGCGGGTTCCGTCTTGGCGCAGCTCCTGGTCTCACTTGCCAATATGTCCTACTTCTATCT

TAACGTTATTAGCCTCGCCTCCGTTAGCGTCGCCTTTTTGTTTTCCTTGTTTCTGCCAATGCCGAAAA

AAAGTATGTTTTTTCACGCAAAACCAAGTAGGGAGATCAAGAAGAGCAGCTCTGTTAATCCCGTTCTG

GAAGAGACACACGAGGGAGAGGCTCCAGGCTGCGAAGAACAGAAACCTACCAGCGAGATACTGTCTAC

ATCAGGAAAGTTGAACAAGGGACAGTTGAACTCACTCAAGCCAAGTAACGTCACAGTTGATGTGTTTG

TGCAATGGTTCCAGGATCTCAAAGAATGTTACAGTTCAAAAAGACTTTTTTACTGGAGCTTGTGGTGG

GCATTCGCAACCGCCGGTTTCAATCAAGTGCTTAACTATGTTCAAATCCTTTGGGATTACAAAGCCCC

AAGTCAGGACTCATCCATCTATAACGGCGCAGTCGAGGCGATTGCTACATTCGGAGGCGCTGTTGCAG

CGTTCGCCGTGGGCTACGTCAAGGTCAACTGGGATTTGCTGGGTGAGCTGGCTCTCGTGGTATTCTCC

GTCGTGAACGCAGGATCTCTTTTCTTGATGCACTACACTGCAAACATCTGGGCGTGCTACGCCGGGTA

CCTTATCTTTAAATCCAGTTACATGCTTCTCATCACTATCGCGGTTTTCCAGATTGCGGTAAATCTCA

ATGTCGAACGATACGCGCTCGTATTCGGCATAAATACTTTCATAGCGTTGGTTATTCAGACAATAATG

ACGGTGATCGTTGTAGATCAAAGAGGGCTGAACCTCCCCGTGTCCATTCAGTTCCTGGTGTATGGTAG

CTACTTCGCTGTTATAGCTGGAATATTCCTTATGCGCTCAATGTACATTACATACAGCACCAAATCCC

AGAAAGATGTGCAGTCTCCGGCTCCGTCTGAAAACCCCGACGTCAGTCATCCGGAGGAGGAGTCTAAC

ATCATAATGTCAACGAAACTTGATTATAAGGATGACGATGATAAATA

The PLP1 protein sequence is:

	(SEQ ID NO: 56)
	MGLLECCARCLVGAPFASLVATGLCFFGVALFCG

	CGHEALTGTEKLIETYFSKNYQDYEYLINVIHAE

	QYVIYGTASFFFLYGALLLAEGFYTTGAVRQIFG

	DYKTTICGKGLSATVTGGQKGRGSRGQHQAHSLE

	RVCHCLGKWLGHPDKFVGITYALTVVWLLVFACS

	AVPVYIYFNTWTTCQSIAFPSKTSASIGSLCADA

	RMYGVLPWNAFPGKVCGSNLLSICKTAEFQMTFH

	LFIAAFVGAAATLVSLLTFMIAATYNFAVLKLMG

	RGTKFDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for PLP1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

PLP1-DNA

(SEQ ID NO: 57)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG GGAGTTGGGAACCTTAAGGCACTGGGATGA

GTGAGACACGGTCACCTGGCTGGGTCCCTTTTCTGGCCCTGGGCATAAGAACAAGCATAAGAACAAGG

CGAAGACAGGGAATGTGGAGAGAGAAAAAGGAAACAGAGACAGACACATAGAGAGAAACAGAGACAGA

GACAGGTAACAGAGACAAAGAGATTGAGACTTGGGGAGAGGATAGAATGAATATTAAGGCAATGGAGA

TGGAAGAAAGGAGAAGAGAAATGTGTGTTAAGGGAATGTGGTTGATAAATTGGTATCTGTGGGTTTAT

TGGGAACACATATTTTCTTTCCAGTAGAGTTGCTGTCCCCAGAGCTGCTGCTGAGAACTCAGGGGAAG

CTTTGTGAGAGGGCTCAGTCTGAGTGATGTCCCCTGTGTGGAGAGCTGTCTCATCTATTCGAGCTGTG

AAGGATCATTTGCCTCATCCTCTAAATCTGAACTCCCAAAGTTTGGGCTCACTCCTGCCATCTGATGA

GCTGAGGAGACACATTTGGCGATTTCTTGCTCTTGGTCCTCCCCCTTCTCCAGGGCCATTCCGACAGT

GACCATCCAGAAGTTTATTGCTGGCTCAATCCTACAGTTGAGCTTCCAAAGCAGGCATAGTCTGTGGC

TGGCGAGCAGGGCTGGGGAGGGCGAGGGGCTCTAGGACCCTTTTCCATCAGTCACATGGCCTTAGTCT

CGTCTGCTCTGGAAAGCTACTATTATACCGTTTTGCAAGGGGCAGCATTTCCAGAGATCCTTTTTTCT

TGGGGCTGATACAAGAGCAAAGGATCTAGAGTTCTAGTACTCTAAGCAAGCCTCAAACGGTGCAGGAT

TTGGCCCCAGTGGGCCCACAGGGGCATCTGCCAAAGACTCGTCCATTTCCTAACAGCAGAGCCCAAGC

CAGTAACATGTCCAAAGTCACAGCCCAAAGAGAAAACTGTAAGACACAATCTTGCCTTTCTCCCACCC

CAGGAAGGTACTAGTCTCTGCTCAGTATCTCCCTCCTCTTCTCTCCTCTCCCAAGGCAAAATCACAGG

AAAAGTTCCAGGAGACCATACCTTCCTTCTCCGGAGTCTTCCAACAGGTGCCCCTCTTATTATATGCC

AGCCAGTTGTTTTAAAGGCCATCCATGGGAGGACACCGGCCTCTAGGTCAAGGGAGCTGTGAAGGGAG

ACAGGACTGACAAGGAGCCCAGACAGACACTGTTTAAGATTCACCTGTTCTAGAAAACCTTCCCAGGC

TGATCCCATCTAGCAGTGGGCATTTAAATACCTTCACAGTCTTTAGAAATAGCTTATTGCTAAGGCAG

GTTTTATATAGTATTTCCCTGTTTTTGCTGCTTAGCCATTTTTGTCTTACCGCTTCTTTCTCTGTGGC

AAGGACTATATTTCGTTTCTTCTCTCCACCCTCACTGCCACCTGCTGCTTCACTCTAACCCACTCCCT

GCCTCTTGTCTGCCTGGCTTAAGCTCTCTGATGAAGATATTACACTCTCAGGATAGAATACTGACTAC

CTGATTCCCAGATCGTGTTCTTGACTTGTCTCGAAGTCGATTATCCTTCTTGGAAATCACCCACATCT

TCAACGCTGGCCTTTTCTTTAACCTAGTCTGTGAACTTGCATAGGTCTTGCCCACTTCTGGGTCTTTA

TTATTGTAGCTATAAAATTATTACAATTCTAGCCTCCCCCAGCCTAATCACAATCTGTTCATCGAATT

GACAGCCTGCATTTTGTCTTCATGCAGTTGAAACTGAGAAGGATTTGGAGGAAATTATCTTAAAAGAC

AATTTTTTCTTCTCTATTGTGTTCGTAGAGCTGGGGGTGGGGGTGGGGGAGGAACGCTTATTTTCCAA

GGAATCGGGAGGGGAAAGGTGGAGAGGGCCAAGGGCAGCTAGGAGTGATGTGGGGAGCTGGAGCAGAT

TCGGAAGGACTTTGGGAGCTAATATCTAGGTTTTTGACTCTGAGCCCCTGTTGGGGCTCTCACTTCAT

GGCTTCTCACGCTTGTGCTGCATATCCCACACCAATTAGACCCAAGGATCAGTTGGAAGTTTCCAGGA

CATCTTCATTTTATTTCCACCCTCAATCCACATTTCCAGATGTCTCTGCAGCAAAGCGAAATTCCAGG

CAAGCCTTAGGGAAAAAAGGAAAAACAAAGAAAATGAAACAATTGGCAGTGAAAGGCAGAAAGAGAAG

ATGGAGCCCTTAGAGAAGGGAGTATCCCTGAGTAGGTGGGGAAAAGGGGAGGAGAAGGGAGGAGGAGA

GGAGGAGGAAAGCAGGCCTGTCCCTTTAAGGGGGTTGGCTGTCAATCAGAAAGC

TGGGTCTCCTTGAATGCTGTGCTCGATGTCTCGTAGGAGCGCCGTTCGCC

TCTCTCGTCGCCACCGGATTGTGTTTTTTCGGTGTTGCGCTCTTTTGCGGCTGTGGTCACGAAGCTCT

TACAGGTACGGAGAAACTCATTGAGACTTATTTCAGTAAGAATTATCAAGATTATGAGTATCTGATAA

ATGTAATTCATGCATTCCAGTATGTTATTTACGGAACGGCTTCTTTCTTTTTCCTGTATGGAGCCCTT

CTCCTGGCTGAGGGTTTCTATACAACAGGTGCAGTGCGCCAAATCTTTGGCGATTATAAAACTACTAT

ATGTGGAAAGGGGTTGTCCGCGACCGTTACAGGCGGCCAAAAGGGTCGGGGTAGCAGAGGTCAACACC

AGGCACACAGTCTGGAGCGAGTATGCCACTGTCTCGGAAAGTGGTTGGGTCACCCAGATAAATTTGTA

GGGATTACTTACGCTTTGACGGTTGTATGGTTGCTGGTTTTCGCGTGCTCAGCTGTACCCGTTTACAT

CTATTTTAATACCTGGACGACCTGTCAGTCTATAGCGTTCCCAAGTAAGACCTCTGCATCAATCGGTA

GTCTGTGTGCCGACGCGAGGATGTATGGCGTGCTGCCATGGAATGCTTTTCCAGGGAAGGTGTGTGGG

AGCAACCTCCTTAGTATATGTAAAACTGCCGAGTTTCAAATGACGTTTCACTTGTTCATAGCGGCGTT

CGTGGGAGCAGCAGCAACCCTGGTATCTCTCCTCACATTTATGATCGCTGCTACGTACAACTTTGCAG

TACTGAAACTTATGGGACGGGGCACTAAATTCGACTACAAGGACGACGACGATAAATA

The TPP1/CLN2 protein sequence is:

	(SEQ ID NO: 58)
	MGLQACLLGLFALILSGKCSYSPEPDQRRTLPPG

	WVSLGRADPEEELSLTFALRQQNVERLSELVQAV

	SDPSSPQYGKYLTLENVADLVRPSPLTLHTVQKW

	LLAAGAQKCHSVITQDFLTCWLSIRQAELLLPGA

	EFHHYVGGPTETHVVRSPHPYQLPQALAPHVDFV

	GGLHRFPPTSSLRQRPEPQVTGTVGLHLGVTPSV

	IRKRYNLTSQDVGSGTSNNSQACAQFLEQYFHDS

	DLAQFMRLFGGNFAHQASVARVVGQQGRGRAGIE

	ASLDVQYLMSAGANISTWVYSSPGRHEGQEPFLQ

	WLMLLSNESALPHVHTVSYGDDEDSLSSAYIQRV

	NTELMKAAARGLTLLFASGDSGAGCWSVSGRHQF

	RPTFPASSPYVTTVGGTSFQEPFLITNEIVDYIS

	GGGFSNVFPRPSYQEEAVTKFLSSSPHLPPSSYF

	NASGRAYPDVAALSDGYWVVSNRVPIPWVSGTSA

	STPVFGGILSLINEHRILSGRPPLGFLNPRLYQQ

	HGAGLFDVTRGCHESCLDEEVEGQGFCSGPGWDP

	VIGWGTPNFPALLKTLLNPDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for TPP1/CLN2 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

TPP1-DNA

(SEQ ID NO: 59)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG CTCCTTTGGGTTCCACTGACACCCCTGCCC

CTGCCCCTGCCCAGAACCAAGTGCCATTTCTCACTCTGGAGCCTTAATAAACTGCAATTTGTATCCAG

TCTCCAGCTTTGTTCTATAGGGATGACTGGAAGACACCTGGCAGAGTATTGTGGATACCTCAGGAGGC

ACAGAGGGGTGGTGGGGTGGGGATGGCACTGGGAGCTCAGGAGCCTAGCCTGAGGCTGATGATCACAT

CTCTCTGCAGCTATTCTTTTCCCTCTAATATGGGCAAAGAGGCCAGCAGGGCCTTGAGAGGGATCAGT

TGGGTGGGAGAGGGGATCTTGGGGATGAGCAGCTCTGGGCCCGAAGTTCTTAAAATAAGTTGGGGTGG

GTACAAGAGCAGTCAAGTGGTTTATTTGTTCTTCTTGGGGGCCACTAAAAATCCACATTGTAAAAAGA

GCTCAATTTGTTGAGCCTTTGTCATGTCAGGCTCTCTGCAGGTGGCTGCCTTGCTTAATCTCAGTGTA

AATTTTCACAGTCATACAAAGGGATTTGGGAACCACTGGTGTTGCCTGTTCTTTGGGGAGCCAGGCTG

TGAGGGAAAGGCAATTGGCCAGTGTTTGAGAAGTTCATGAGATTGAGGAAATTAGAAAAAAAAAAAAA

AGTTGTGGTCAAAGGGAAATGTTTCTGAGCATGATGCTAGTAAATACAGCTCTAGGATCCACGCTTAG

TGGGAAACTGGAGGGATCAATGGACAGAGAGAAGTCAGATTAATAGGAGTGAGGAAAAACAATGGCTT

GAAAAAACAGAAGGTTATAGTCTAAGAAAGCATTCTATAATTATAATTTCAAAGGTAGAGCAGTTTTG

AGAGATAGCAAGCCCAATGCAGCCATGGGACTGGTTTGCTTAAGTGAAGTGAAGGTAATGCTCACTGG

GTTTAATGAGATGAAGAAATGGAAGGTTAGGGTGTTGAATGGGTCTTTTACCTAGATATTTTGATCAT

CTAGGTCAAATGATCGCAGGAGTTGGGATGGACAGAAAGGTGAAGTGCCATAATCTTCAGTGTGGGAA

TATAATCTATAGGTTAATGGAAGCCAGTGGCCTGTAGGACAACAGCAGGAGTAGAGGGATCTATCTAA

AAAGTATTGAGGTCAGAACAAGGGATTTCACATGAGTATTGGATTAATGGTTTTGGAAGTTAAAATGA

CACTGCTGACACCATCTTCCATATGGATTTTTGGGGGGAAAAAAAGGAAGACTGGAAGGGAATTGAAT

CCTCAGGGTTTTAACTCTCATTGAAAATCCTCTTGTAATGACCCCAGTTGGAAGTAGTAAACTCAGAC

CTCCTTGTGGGCTTTGAGTTACCATTAGAAGAAGCAAAGGTCCGTTTCCATTTTATCCACCTGAGCCC

ACTAAACAAGATGCAAGATTCCTCCATGGCCCTTAGAACAAAGTTGATTTCCTTGAAAACCTTGGGAA

CCCATAATAAACAGGACCCCAGCTGATTGTAGATCTCCTGGGAAGCTACTTAAAACTGGAAACTGATT

TGAATAAAAGGATCCTGGTCTTGCAAAAGCTTTACTCTGTTCATAAATGTAGTCGTAAAATGAAGACA

AAATTGATAATATGGGGACATAAAGGATAAAAGAGGAAAAAAATTTTTTCACTGCAATCTTTCGAGGC

AGATCACTTTTATATAAGTCTCCATATTGTAAATGCAGAAACCTAGGCTCTGAGAGGTTATTTTGCTG

ATGTCATACAGCTAGGAAGAACCCCAGTCTGTGGGTCTCTACAGCCCACTCACTTGTGGTGATTCCCA

GGGGACCAGGCTCAGGACGCAGGTGGGGAGCCCTGGGCTTACTCAGTGCTTGACTGGCCAGAGGGGAG

AATCCGGGTGGCGGCCCCACCCTTGCCTAGCATTTGGGACCACCCATGCAAGGGAGGAGCCAGTACCG

TCACTAGTTACTAGGCAGAGGGGTAGTGGTGGTGGAATATAGAGCTCATGTGATCCGTCACATGAC

ATGGGCTTGCAAGCGTGCCTGTTGGGGCTCTTCGCATTG

ATTTTGAGTGGAAAGTGTTCCTACTCACCCGAGCCCGACCAGCGCCGCACACTCCCTCCTGGATGGGT

AAGCCTTGGCCGGGCCGATCCAGAAGAAGAGCTTAGTCTTACTTTTGCATTGCGACAACAGAATGTCG

AAAGGCTTTCCGAACTGGTACAAGCTGTGTCAGATCCGTCTTCACCTCAGTATGGTAAATACTTGACT

CTCGAAAACGTCGCAGATCTCGTACGCCCGTCTCCCCTTACTTTGCATACTGTTCAGAAGTGGCTGCT

TGCGGCTGGTGCTCAAAAGTGCCACTCCGTGATAACTCAAGATTTTCTGACCTGTTGGCTTAGCATCC

GGCAAGCAGAATTGCTCCTGCCCGGAGCGGAGTTCCACCACTATGTCGGTGGTCCTACAGAGACGCAC

GTGGTGAGATCACCTCACCCCTACCAGTTGCCACAAGCGTTGGCCCCTCATGTCGACTTTGTAGGAGG

ACTCCATAGGTTTCCCCCAACTTCAAGTTTGCGCCAGCGGCCAGAACCACAAGTCACTGGTACAGTTG

GACTGCATTTGGGAGTCACCCCCAGCGTCATACGCAAAAGATACAACCTTACTTCACAAGATGTCGGC

TCCGGGACGTCCAATAACTCTCAGGCATGCGCGCAGTTCCTGGAGCAGTACTTTCATGACTCCGATTT

GGCGCAATTTATGAGGCTTTTCGGAGGTAATTTTGCTCACCAAGCCTCTGTAGCAAGGGTCGTTGGTC

AACAAGGACGGGGGCGGGCTGGCATCGAAGCAAGTCTTGATGTACAATACCTGATGTCAGCGGGAGCT

AATATTTCTACGTGGGTTTATAGCTCCCCCGGCCGCCATGAGGGTCAGGAACCGTTTCTTCAATGGCT

GATGTTGTTGAGCAATGAGAGCGCGCTTCCTCATGTCCACACGGTCTCCTACGGAGATGACGAAGATA

GCCTGTCATCCGCATACATACAACGGGTCAACACAGAACTCATGAAAGCCGCGGCCAGGGGCCTCACC

TTGTTGTTTGCTTCTGGAGATAGCGGAGCCGGCTGCTGGTCTGTGTCTGGGCGACATCAATTCAGGCC

AACATTCCCGGCGTCCAGTCCCTATGTCACTACCGTGGGGGGCACCTCCTTTCAAGAGCCGTTCCTCA

TAACCAACGAAATTGTCGACTACATTAGTGGAGGCGGCTTCTCAAATGTGTTCCCCCGGCCGTCATAC

CAGGAGGAGGCAGTTACAAAATTCCTCTCTAGTAGCCCGCATTTGCCTCCATCCAGCTACTTCAATGC

AAGTGGGCGCGCGTATCCCGATGTAGCAGCCCTTTCAGACGGCTATTGGGTAGTCAGTAATCGCGTCC

CCATACCCTGGGTTTCTGGCACTTCCGCCTCAACGCCTGTGTTCGGGGGCATATTGTCACTGATAAAC

GAACACAGGATATTGTCCGGCAGGCCGCCCTTGGGATTTTTGAACCCTAGATTGTATCAACAACACGG

GGCGGGTTTGTTCGACGTAACCCGAGGCTGCCATGAGTCTTGCTTGGACGAAGAGGTAGAAGGTCAAG

GGTTCTGTAGTGGGCCAGGCTGGGACCCTGTAACTGGATGGGGCACCCCGAACTTTCCCGCTCTGCTT

AAAACGCTTCTCAACCCTGACTATAAGGATGACGACGACAAGTA

The ACY2/ASPA protein sequence is:

	(SEQ ID NO: 60)
	MTSCHIAEEHIQKVAIFGGTHGNELTGVFLVKHW

	LENGAEIQRTGLEVKPFITNPRAVKKCTRYIDCD

	LNRIFDLENLGKKMSEDLPYEVRRAQEINHLFGP

	KDSEDSYDIIFDLHNTTSNMGCTLILEDSRNNFL

	IQMFHYIKTSLAPLPCYVYLIEHPSLKYATTRSI

	AKYPVGIEVGPQPQGVLRADILDQMRKMIKHALD

	FIHHFNEGKEFPPCAIEVYKIIEKVDYPRDENGE

	IAAIIHPNLQDQDWKPLHPGDPMFLTLDGKTIPL

	GGDCTVYPVFVNEAAYYEKKEAFAKTTKLTLNAK

	SIRCCLHDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for ACY2/ASPA and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

ACY2/ASPA-DNA

(SEQ ID NO: 61)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG TTTTTTGGGTCATCCCAAAAATCAAGTACA

CTTTGGGAAACAGGGAGTTAAAAATCTTTCGGAAGTTAACTTGGTATAAGAGTATGAAAAGCCTCTGT

TGCTCTACTTCTGAGGAAATGCTCTCCTTTTTCCTAATAAAGCTTTCCACAGAAGACAAACTCTGTAC

ACAAAAAACAAGGTAAGTATGGGAAATAATTGCTGCAATTCATTAACTCTCAAAAAAGTTATTCATTT

TGACATGTAAGAGAAACTTTATTTATGAATGTGAAATGTGCTTCAAAGTGCTGATCTGTTAAAATTCC

ATAAAACAAGCTTCAAAATGCAAGTCATTTGTGCCAGCTACGAGTAAATAACTGAAGCAGCATTTCCA

GAGAGACTAGGGCAAAGGACAGAACAAACTACAAATCAGCAGAAAAGAAACCAAGCATAGTAGAGTGT

TAATAATTCACCATGGTTACTGCTCTTAAAACCAAAGCAACTAAACAACTGTCATCTGTTTACTTTTA

CGTGTGTTTTCATCATCTCTACACCGTCTGAAGTGTTCAGTGTACATGGATGTGGACAGACAGTCAAA

GCCTACAGGAGCTTTTTTTTTCTATTAGAACAGTCATTTTAATGCATAGATTTGATGTGGTTATTTTG

CTTTCTCTATCTCTGCATTATAAGCTCTTAAAGGAAGAACATTCAGTGGGTTTATTAAATATTAGGAA

GCATTCATATTTTATTTTTCCACCATGTATTTGGTTTGTATGCATGTTTGTTTCCTGAGCACACTGTA

ATGGCTGTTTATGTGAATTCTGAAAGAGCAAAACTAGAACTGTGAAGAAGACCAAAAATAACAGAGAG

GCACCCTATTGTCCTGTGAAAAACACTAGACTTGGGATGAGAAACATGGGTCTCAGGCCTAGGTTCTC

CTACATCCCTGCAGAACCTTAACTGAGTTACTTAACCTCTCTTTCGTTTACCCCTGCAGTGGGGATGT

CAACACCTAACCCACCCTATCGTATAAAATTAATGTATGAATAAACTGAGTGAAAGGACATTATAAAT

TAAACTCTGCAATTGTAATAAATTACTCTTATTGTACCCTCTATCAAACAACCTGGCGTTACTAGTAC

ATGGTATTCCCAGTACTTGAACATATGTGACTTTCTGGGTGACTGAGCATGAAATCAAATCAGATTTG

GCTCAGAGTCATTTCTCCAAAGGGATGTAATCATCTTAGTATTCTGGAAAATTTCCCCTAATAAGACC

CCTTTTTGAGTAACACAGTCCAGTGGAGAGTCCCCGCTGACAGTGAATAAGGCTGTGTCTATGTAAGT

TCACATGATGTGGTTAGCACATGCAGTGTGTCCCAAGACATTTTCAGGCATCACAAAAACTTGAAAAG

TACTAACAGTTGTCTTTCCTTTGGTATTTAGGCAAAAAATGAGGAAGATTCATAAGTAAGTTAACACA

GAATTACCAAAAACCTCATGAGAAAGAAATGAAGGGATGTTTCAGGGTAGCTTTTCGAGTCCCAAGTG

ATCATTGATTTGGCCCTTCTGTTTCTGATTGTGAAAAAGCATTCTGAAAGAATGGGCGCCGGTTCAAG

AGGAGGTCTCTGAGGGTCAGCCTTTGGCTGTTTATAGAGGTGAGATTCAAATGCCCTCAGATCATGAA

AAGTGCTTGAACTCATTTCAGAGAAGAAATGTTTTGAAATGCCCGTGGAAATACTGAGTGGGCCACTT

TTTTATTTTGTGAAATTAGAAACGTATAAACTATTTTTATTATTACAGATAATTGTGAAAGCCTCACT

GGATGACATCCTAGCTGAGGAAAAGAATGGAATTTTTGTATTAGTATGAAATGCTAATTTATTACTGT

ATTTAGATCTACTGACCATCTGAGGGAGTTAGAAGTTAAAGTAAACAGCTGTATCTCTAGTTGATCTT

TGCATATTTTAATCCAAATATGGCAAAGGGCAGGGCTAAAGAAGGGAGTGTCCATAAACGGGGCTC

ATGACAAGTTGTCACATAGCTGAGGAACACATCCAGAAG

GTTGCTATTTTTGGCGGCACTCACGGTAACGAACTGACAGGGGTCTTCCTCGTCAAGCACTGGCTTGA

GAATGGGGCTGAAATTCAGCGAACAGGACTTGAAGTAAAACCGTTTATAACGAACCCACGCGCTGTAA

AAAAGTGCACGCGATATATCGATTGCGACCTTAACCGGATATTTGATCTGGAGAATCTTGGGAAGAAA

ATGTCAGAGGACCTCCCTTATGAGGTGAGACGAGCGCAGGAGATTAACCACCTCTTTGGTCCCAAGGA

TTCCGAAGACTCCTACGATATAATTTTCGATCTCCACAACACGACCTCTAACATGGGTTGTACCTTGA

TCCTCGAGGATAGTCGCAACAATTTCTTGATTCAAATGTTCCACTACATAAAAACATCTCTCGCTCCC

CTTCCTTGCTACGTATACCTCATTGAGCATCCAAGTCTTAAGTACGCAACCACCCGGAGCATAGCGAA

GTATCCAGTGGGAATTGAAGTCGGGCCTCAACCCCAGGGAGTTTTGCGGGCGGACATCCTCGACCAGA

TGAGGAAAATGATTAAGCACGCGCTGGACTTCATACACCATTTTAATGAAGGGAAAGAGTTCCCGCCA

TGTGCCATAGAGGTTTATAAGATTATTGAGAAGGTAGATTACCCTAGGGACGAGAATGGGGAAATCGC

TGCCATCATACACCCCAATCTCCAGGACCAAGATTGGAAGCCACTTCATCCCGGCGACCCAATGTTCT

TGACGCTGGACGGCAAAACGATTCCCCTTGGGGGTGATTGCACGGTATATCCAGTGTTTGTGAACGAG

GCTGCTTATTACGAAAAGAAAGAAGCATTCGCCAAAACCACGAAATTGACACTGAATGCAAAATCTAT

CAGATGCTGTCTCCACGACTACAAGGATGACGATGATAAATA

The MANBA protein sequence is:

	(SEQ ID NO: 62)
	MRLHLLLLLALCGAGTTAAELSYSLRGNWSICNG

	NGSLELPGAVPGCVHSALFQQGLIQDSYYRFNDL

	NYRWVSLDNWTYSKEFKIPFEISKWQKVNLILEG

	VDTVSKILFNEVTIGETDNMFNRYSFDITNVVRD

	VNSIELRFQSAVLYAAQQSKAHTRYQVPPDCPPL

	VQKGECHVNFVRKEQCSFSWDWGPSFPTQGIWKD

	VRIEAYNICHLNYFTFSPIYDKSAQEWNLEIEST

	FDVVSSKPVGGQVIVAIPKLQTQQTYSIELQPGK

	RIVELFVNISKNITVETWWPHGHGNQTGYNMTVL

	FELDGGLNIEKSAKVYFRTVELIEEPIKGSPGLS

	FYFKINGFPIFLKGSNWIPADSFQDRVTSELLRL

	LLQSVVDANMNTLRVWGGGIYEQDEFYELCDELG

	IMVWQDFMFACALYPTDQGFLDSVTAEVAYQIKR

	LKSHPSIIIWSGNNENEEALMMNWYHISFTDRPI

	YIKDYVTLYVKNIRELVLAGDKSRPFITSSPTNG

	AETVAEAWVSQNPNSNYFGDVHFYDYISDCWNWK

	VFPKARFASEYGYQSWPSFSTLEKVSSTEDWSFN

	SKFSLHRQHHEGGNKQMLYQAGLHFKLPQSTDPL

	RTFKDTIYLTQVMQAQCVKTETEFYRRSRSEIVD

	QQGHTMGALYWQLNDIWQAPSWASLEYGGKWKML

	HYFAQNFFAPLLPVGFENENTFYIYGVSDLHSDY

	SMTLSVRVHTWSSLEPVCSRVTERFVMKGGEAVC

	LYEEPVSELLRRCGNCTRESCVVSFYLSADHELL

	SPTNYHFLSSPKEAVGLCKAQITAIISQQGDIFV

	FDLETSAVAPFVWLDVGSIPGRFSDNGFLMTEKT

	RTILFYPWEPTSKNELEQSFHVTSLTDIYDYKDD

	DDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for MANBA and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

MANBA-DNA

(SEQ ID NO: 63)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG GATTATTCTTTGATGTTCTCTGTTGTCAAA

GTGAAGTAACCTAAATGATGTTATGCTTGCATCCTGGAATGAAAGGAGGAATGAGAATCTTCTATTAT

TGTTTTTCTAAAAATTATATTTCTTAATAAACAGAATATATCCATATGTATCATCAAGGATGAATTTT

CCTCTGATTTGGCTTAATGAAATCTCTGGGAATTATGAAAAAATTCCTTTGCCGCTCTTGTTGCCATA

AACCTTGTGTTATCAGTGTTGCACAGACCATGGCTAGCCATTTAATGCCGTGAATTATGGCATATAAT

TTGAATTGAGAGCTTTGTTTCTATCAAAGGTAAAAATGCTTCTGGAGTGAGTGAGGAAGGCTTTGAAG

CAGTGAGAGCGTTTCCCCTATAGCATCATTGGGCTCTGACAGTAGGACCTTGAAGAGGAGGGTTCTAA

TCCTGTCTGCCACTATGTGACAGGCAAGTCACCTGATCTTTTTTTTTTTTTTTTTTTTAGAGTCTTTC

TCTATAGCCCAGGCTGGAGTGCAGTAGTGCCATCTCGGCTTACTGCAACCTCTGCTTCCTGGGTTCAA

GCGGTTCTCCTGCCTCAGTTTCCCGAGCACCTGCGACTACAGGCACGTGCCACCACGCGCCCAGCTAA

TTTTTGTATTTTTAATAGAGACAGGGTTTCACCATGTTGGCCAGGCTGGCCTCGAACTCCTGACCTCA

AGTGATCCACCCGCCTCGGCCTCCCAGAAGTGCTGGGATTACAGACGTGAGCCACCGCGCCTGGCCCC

GATCTCTTTGAACTCAGTTCCCTCATCTGTAAAGCCAGCGGCTTGGGTTGGATAATCCATACTGTCCT

TTAAGCCAAGATATTCAGGGCTTGTAAATAATGCTGACTTTGCAAAGTAAGATATTGCCCGGTGTCTG

CAGAATCAGGCAATTCCAAGGCCATTGTAAGGCGAAACAAAGCTCAGATTTGATGAGAGAAAATTTCT

TTTCAAGGACTTCTTCCGAAGCATAAATATACGGCAGATAGCGTCAACACAGGAGAAAGGCAGTCTAT

TTTATGCAATGTAAAATTCCGTCTTAACTTCTAGGATTACTGTTACATCCTATTGCAAAAATGGGACT

CAGGTAGACCATTTGACTACAAGACCCGAAATAAGTCAGTGGTATTTCTTGGGTACTCTGGCATTGTT

AGGGACGATCACATATGTTTACTCATTTTATACTTATAACACCTTGAAAACTTCAAGAAACGGAGATG

GAGATTTTGCGGCTTGCCCAGGTCCCACAGCTAGTGAGTGCAGAGGATTTTAAACTCAGCTGCGCGCC

TAGTGCAGGTTCCAGAACCTCAGAAACCTCCGTGGGGCTTTATGAAACATAAGGACGCAGGCCGCTAG

TAACAATAGCTAACGTGAATGCCTTCTCTGGGCCAGGCTCCATGCTGCATGCTATAGTCGCATTTTCT

TGGTTAGCCCTCAGAACAACCTTCTGATGGAGATACTGCTAGTACTTGTTTACTCTTTGAAGGGCGGG

GTAGGCTCTGAGACCCGCGAGTTGAGCTGCAGCGGGGTGAGTGTTACGCTACCAGGTAGACGAGGGTC

TGTGTAAATTTGGCGGAAGGGAAGTGGTGGGTTGTCTCCCAGTTTGGGTTCTCTGAAGGAGTGAGGGG

ACTCTCCAGCTATGGTCTCCACGTACAGTCGGTCTCTGATCTCCCGAGGCTTTGTGCCACTGCGCGCC

CAGGGATCTTTCTGTCCCAATTACCGTGCAAGATTCTTGCCTCCAGTTGTATGTACTGAGGCGGCGGG

AAGAAGCGCCGAGGGAAGGCTCAGACCGCAATGCCCCCCAGCCTGTAGCTATTCTCGGGGGCTGCAGC

TCCCGCACCCGCGACGCCCGGCTTTCAGCGGGTGCAGGCGCAGCCTCCTCGTTCCCCTCCCGCCCGCG

GCTGCGCCCGGAGAGCGCTCAGCTGACCTAGGGGCGGGGCCGGGGCGGGGACCTAAAGCCTCGGCTAC

TTCCGAGGGGG ATGAGGCTTCACCTGCTGCTCCTTC

TCGCGTTGTGTGGCGCGGGTACAACCGCCGCGGAATTGAGTTATTCCTTGAGGGGGAATTGGTCCATC

TGCAATGGCAACGGCAGCCTGGAACTTCCGGGCGCTGTACCGGGTTGTGTGCATTCTGCTCTTTTCCA

ACAAGGGCTCATTCAAGATTCATACTACAGATTTAACGACCTTAACTATAGGTGGGTGAGCCTTGATA

ACTGGACATATTCCAAGGAGTTCAAAATCCCTTTCGAAATATCAAAATGGCAAAAGGTTAATCTCATC

CTTGAAGGCGTTGACACCGTTTCTAAAATACTGTTCAACGAGGTGACTATCGGAGAAACCGACAATAT

GTTCAATCGATATTCTTTCGACATTACTAACGTGGTTCGAGACGTAAACAGCATTGAATTGAGGTTTC

AGTCAGCAGTGCTCTATGCCGCCCAGCAAAGCAAAGCCCATACCCGGTATCAGGTACCTCCAGACTGC

CCTCCTCTCGTCCAGAAAGGTGAATGCCATGTCAACTTCGTGAGGAAAGAGCAGTGCAGTTTCTCTTG

GGACTGGGGTCCATCCTTTCCGACCCAGGGCATTTGGAAAGATGTTCGAATTGAGGCCTACAATATTT

GCCATCTCAATTACTTTACCTTCTCCCCGATATACGACAAGTCCGCCCAGGAGTGGAATCTCGAGATA

GAATCCACGTTTGACGTAGTTAGCTCTAAGCCTGTTGGCGGCCAAGTGATCGTAGCCATACCAAAGCT

TCAAACACAACAGACGTACTCAATCGAGCTCCAACCAGGAAAGAGAATAGTGGAACTTTTCGTTAATA

TTAGCAAAAATATAACCGTTGAGACCTGGTGGCCTCACGGCCATGGAAATCAAACGGGTTACAACATG

ACTGTTTTGTTCGAGCTTGACGGGGGCCTGAATATAGAGAAAAGCGCCAAAGTTTACTTCCGGACCGT

AGAACTTATCGAAGAACCGATTAAAGGTTCCCCGGGCCTGAGCTTTTATTTCAAAATAAACGGCTTTC

CTATCTTTCTTAAAGGCTCCAATTGGATACCAGCAGATTCCTTTCAGGATCGAGTAACATCTGAATTG

TTGCGACTGTTGCTGCAATCAGTAGTAGATGCCAATATGAATACGCTGAGAGTCTGGGGAGGCGGGAT

TTACGAACAAGACGAGTTTTACGAACTCTGTGATGAGCTTGGGATTATGGTGTGGCAAGATTTCATGT

TTGCATGCGCACTTTATCCCACTGACCAGGGTTTCTTGGACTCCGTCACTGCGGAGGTAGCATATCAA

ATTAAAAGGTTGAAGAGTCACCCCTCAATTATAATATGGTCCGGAAACAATGAAAATGAAGAGGCTTT

GATGATGAATTGGTATCACATTTCCTTTACCGATCGGCCAATTTATATAAAAGATTACGTGACGCTTT

ACGTGAAGAACATACGGGAGCTTGTGTTGGCTGGAGACAAGTCCAGACCATTCATCACCAGTTCACCT

ACGAATGGGGCGGAAACCGTAGCCGAGGCATGGGTATCACAAAATCCTAATTCCAATTACTTCGGCGA

CGTGCATTTTTATGACTACATAAGTGATTGTTGGAACTGGAAGGTTTTCCCGAAGGCAAGGTTTGCGT

CCGAATATGGCTATCAGAGTTGGCCAAGTTTTTCCACTCTCGAGAAAGTTAGTAGCACTGAAGACTGG

AGCTTCAACTCCAAATTCAGTTTGCATCGGCAGCACCACGAAGGCGGAAATAAGCAAATGCTTTATCA

GGCTGGACTCCACTTTAAACTGCCCCAGTCCACGGACCCGCTCCGAACGTTTAAAGACACTATATACC

TGACGCAAGTCATGCAAGCACAATGTGTTAAGACGGAAACCGAATTTTACAGACGATCTAGGAGTGAG

ATTGTGGATCAACAGGGCCACACGATGGGAGCTCTGTACTGGCAGCTTAATGACATTTGGCAGGCGCC

CTCTTGGGCTAGTTTGGAGTATGGAGGTAAGTGGAAGATGCTTCATTATTTTGCTCAAAACTTCTTCG

CGCCCCTCCTTCCCGTCGGTTTTGAAAACGAAAACACATTTTATATCTATGGGGTCAGCGATCTGCAC

TCCGACTACTCAATGACGCTTAGCGTTCGAGTGCATACCTGGTCAAGTCTCGAGCCCGTCTGCTCTAG

AGTCACGGAACGCTTTGTCATGAAGGGTGGGGAGGCCGTATGCTTGTACGAAGAACCTGTATCCGAGC

TTTTGCGGAGGTGTGGTAATTGTACTCGAGAAAGCTGTGTAGTGAGCTTCTATCTTTCTGCTGACCAC

GAGTTGCTTAGTCCTACCAATTATCATTTTCTGAGTAGCCCCAAGGAAGCGGTTGGCCTGTGCAAGGC

GCAGATTACGGCGATTATTAGCCAACAAGGGGATATCTTTGTGTTCGACCTCGAGACCTCCGCCGTTG

CCCCCTTCGTGTGGCTTGATGTGGGCAGTATCCCGGGAAGATTCTCCGACAACGGATTCCTGATGACA

GAAAAAACTCGCACAATACTTTTTTATCCATGGGAACCGACTTCCAAGAACGAACTTGAGCAATCATT

TCATGTGACGAGCTTGACGGATATATACGATTATAAGGACGATGACGATAAGTA

The LIPA protein sequence is:

	(SEQ ID NO: 64)
	MKMRFLGLVVCLVLWTLHSEGSGGKLTAVDPETN

	MNVSEIISYWGFPSEEYLVETEDGYILCLNRIPH

	GRKNHSDKGPKPVVFLQHGLLADSSNWVTNLANS

	SLGFILADAGFDVWMGNSRGNTWSRKHKTLSVSQ

	DEFWAFSYDEMAKYDLPASINFILNKTGQEQVYY

	VGHSQGTTIGFIAFSQIPELAKRIKMFFALGPVA

	SVAFCTSPMAKLGRLPDHLIKDLFGDKEFLPQSA

	FLKWLGTHVCTHVILKELCGNLCFLLCGFNERNL

	NMSRVDVYTTHSPAGTSVQNMLHWSQAVKFQKFQ

	AFDWGSSAKNYFHYNQSYPPTYNVKDMLVPTAVW

	SGGHDWLADVYDVNILLTQITNLVFHESIPEWEH

	LDFIWGLDAPWRLYNKIINLMRKYQDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for LIPA and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

LIPA-DNA

(SEQ ID NO: 65)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG TGTATCTAAGAAGCAACTAACTTGCTTTTG

ATTTTACCAGCTAATAGGTGGAAGGGACTTGCCTTGTCTCAGGTGAGACTTTGGACTATGGACTTTTG

AGTTAATGCTGAAATGAGTTAAGACTTTGGGGGACTACTGGGAAGGCATGATTGGTTTTGAAATGTGA

GGACATGAGATTTGGGAGTGGCCAAGGGCAGAATGATATGGTTTGGCTGTGTCCCCACCCAAATCTCA

TCTTGAATTATAGCTCCTGTAATTCCCACATGTTGTGGGAGGGACCCGGTGGGAGATAACTGAATCAT

GGGGGCAGTCTAGCCCATACTGTTCTTGTGGTGGTAAATAAGTCTCACAAGGTCTGATGGTTTTATAA

GAGGAAAATCCTTTTACTTGGTTCTTTCTCTCACATGTGCCGCCATGTAAGACAAGCCTTTTGCCTTC

TGCCATGATTGTGAGGCCTCCCCAGTCCTGTGAAACTGTGAGTCAGTTAAACCTTTTTCCTTCATAAA

TTACCCAGTCTTGGGTATGTCTTTATTAGCAGCATGAGAACAGACTAATACACCTTCTAATTAGTCTC

CCTGCTTCTGTAGTTCAGAATGATCTTTCTAAACTAGATTATGCTGCCCTTCTATTGAAAATATTACA

CTGGCTTCTCATCTTACTCAGAAGACACGGTAAGATCTTGACAAGTGGCTCCCAAGGCACCAGACTTC

TAGGACTTCATGTTCTATTACTTTCTACTATGTACATTCTGTTTTAGCCTCAAAGACCTCACCACTCT

GGCTAGGACCCACCAGGAATATTCCTACCACAGGGCCTTTGTACCTGCTATTTTCACCCAGAATGTGC

TTTCTCCAGTATCTAAGGCTGTTAACTACCTCCTTGCCTGTGCTCAGGTGTCACTTCATAAGAGTCCT

TTCCTGATTACCATATACATATCATCCTCCTTATCCCTGTTTTTTTTTTTTTTTGGGGGATGGAGTCT

CACTCTGTTGTCCATGCTGGAGTGCAGTGGCATGATCTCCGCTCACTGCAACCTCCACCTACCTGGTT

CAAGCCATTTTCCTGCCTCAGCCTCCCAAGGAGCTGGGATTAGAGGCACACACCACCACACCCGCTAA

TTTTGTATTTTTAGTAGAGATGGGGTTTCACCAAGTTGGCCAGGCTGGTCCTGAACTCCTGTCCTCAG

GTGATCCGCCCTCCTCAGCTTCCCAAACTGCTGGGATTACAGGCGTGAGCCACTGTGCCCAGCCCCTT

GTTGTTTGTTGTTGTTGTTGTTTGTTTGTTTTTGTTGTTATTTATAGCACTTATCACCACCTCATGTA

TTATATAGTTATTCATGATGTGTCTCTTCCCATCAGAATATATACAAGAGAACAGGAACCTTGTTTGG

TTGGTTTCTATACCTCCACTGTCTTACTAATGCCTGGCATTTAGTAGGTGCTCAATAAAATCTTTGTT

TTAGAAAATGATTAATAAATAATATGTACATATTGAATATTGTTTCCTCTTTTAACAAATCTTTAATA

ATAATAAAAAATTTAAAGCAGCAAGATTGCTTTGCATCAGGCACAATGATAAGCGCTTTAAATGGAGC

CCATGATTCACTTTCATTTATAGAGCCCACATGCTCTCTGTCAGGCGCAGAAGGTGCCAGCCCTGCAG

GAGCTTCGGGGGCGGAGCAGAAGGCCGCCTAAAACAGCCTTTGCTAAGAGAGCATGCGTAGGCGACGC

GCTGGTAGAGCTGTGGACCTGCCAGCCTGCGAGGCGGAGGACGGGCTCCATCTCTTAGAAACGCCTAC

GGCGCATGCTCTATGGGGTCAACTGGGGGGCTGGCAAGCGGCAGCGCTGGTCTGGGGCGGAGTCTCCG

AGGCACTTCCCGGTGGCTGGCTGCTCTGATTGGCTGAACAAATAGTCCGAGGGTGGTGGGCATCCGCC

CTCCCGACAAGGCAGACCAGGCCCCCTGCAGGTCCCCTATCCGCACCCCGGCCCCTGAGAGCTGG

ATGAAGATGCGCTTCCTTGGCCTCGTAGTTTGTCTGGTT

CTGTGGACACTGCACAGTGAGGGTAGCGGCGGTAAATTGACGGCTGTAGATCCCGAAACTAATATGAA

TGTTAGTGAAATAATCTCTTACTGGGGATTTCCATCCGAGGAGTATCTTGTAGAAACAGAAGATGGCT

ATATACTTTGTCTCAACCGGATACCACACGGACGGAAAAACCACAGCGATAAGGGGCCAAAGCCGGTG

GTTTTCCTTCAACACGGTCTTCTGGCAGATTCTAGTAATTGGGTTACCAACCTTGCAAACTCTTCACT

TGGATTCATTCTTGCGGATGCTGGATTTGACGTATGGATGGGGAACTCCAGAGGGAATACGTGGTCCA

GAAAACACAAGACATTGTCTGTATCTCAAGATGAGTTCTGGGCATTCAGTTACGACGAAATGGCGAAG

TATGATCTCCCAGCATCCATAAATTTCATCCTGAACAAGACTGGTCAAGAACAAGTATATTACGTCGG

ACACAGTCAAGGCACTACTATTGGCTTTATCGCTTTCTCCCAAATCCCGGAATTGGCGAAAAGAATCA

AGATGTTCTTCGCATTGGGTCCGGTGGCGTCTGTCGCTTTTTGCACCAGCCCCATGGCGAAACTTGGG

AGGTTGCCGGATCATCTCATAAAGGACCTCTTTGGAGATAAAGAGTTTCTTCCTCAAAGCGCATTTCT

TAAGTGGTTGGGAACACATGTCTGCACTCACGTGATCTTGAAAGAACTTTGTGGAAACCTGTGTTTCC

TCCTGTGCGGGTTCAATGAACGAAATCTTAATATGTCCAGGGTGGACGTCTATACCACCCACAGCCCG

GCAGGTACGTCAGTCCAGAATATGTTGCATTGGAGCCAAGCGGTAAAGTTCCAAAAATTCCAGGCATT

TGACTGGGGTTCTTCAGCTAAGAATTACTTCCACTATAATCAGAGCTACCCGCCCACTTATAATGTCA

AGGATATGTTGGTTCCAACTGCAGTTTGGAGCGGCGGGCACGATTGGCTGGCGGATGTGTATGATGTG

AATATCCTTCTCACACAAATTACTAATCTCGTGTTCCACGAGAGCATACCAGAGTGGGAACATTTGGA

TTTTATATGGGGACTGGATGCTCCATGGCGGCTCTACAATAAAATCATCAATCTGATGCGGAAATACC

AAGACTACAAAGACGACGATGATAAGTA

The CTNS protein sequence is:

	(SEQ ID NO: 66)
	MIRNWLTIFILFPLKLVEKCESSVSLTVPPVVKL

	ENGSSTNVSLTLRPPLNATLVITFEITFRSKNIT

	ILELPDEVVVPPGVTNSSFQVTSQNVGQLTVYLH

	GNHSNQTGPRIRFLVIRSSAISIINQVIGWIYFV

	AWSISFYPQVIMNWRRKSVIGLSFDFVALNLTGF

	VAYSVFNIGLLWVPYIKEQFLLKYPNGVNPVNSN

	DVFFSLHAVVLTLIIIVQCCLYERGGQRVSWPAI

	GFLVLAWLFAFVTMIVAAVGVTTWLQFLFCFSYI

	KLAVTLVKYFPQAYMNFYYKSTEGWSIGNVLLDF

	TGGSFSLLQMFLQSYNNDQWTLIFGDPTKFGLGV

	FSIVFDVVFFIQHFCLYRKRPGLQAARTGSGSRL

	RQDWAPSLQPKALPQTTSVSASSLKGDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for CTNS and the 5′ and 3′ UTR sequences are listed below using the same

CTNS-DNA

(SEQ ID NO: 67)
GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGAGGAACTTGGTTAGGTACCTTCTGAGGCGGAAAG

AACCAGCGTTACATAACTTACGGTAAATGGCCCGCCTGGCTGACCGCCCAACGACCCCCGCCCATTGA

CGTCAATAATGACGTATGTTCCCATAGTAACGCCAATAGGGACTTTCCATTGACGTCAATGGGTGGAG

TATTTACGGTAAACTGCCCACTTGGCAGTACATCAAGTGTATCATATGCCAAGTACGCCCCCTATTGA

CGTCAATGACGGTAAATGGCCCGCCTGGCATTATGCCCAGTACATGACCTTATGGGACTTTCCTACTT

GGCAGTACATCTACGTATTAGTCATCGCTATTACCATG AAACTGAGGCACGAAAATCACTTGAACCCG

GGAGGCAGAGGTTTCAGTGAGCAGAGATTGCACCACTGCACTCCAGCCTGGGTGACAGAGCGAGACCC

TGTCTCAAAAAAAAAAAAAAAAAAAAAAGAAGTGCTCTATTTCAGGAGAAACTGGCACTTTCTGAGCC

TACTCTCCCCTAATGCCAGCTCTCCTGCTCACCCCACCAGGGTCAGAGCCAACTTTGCCTCCAATTCA

TAGTCCTTTAAGTAAGAATCCTTTTAATATGCCCTAATGTCCCAACCAAACTAATCTTGAAAGCTTCT

ATGTAGATACAAAGTGCTCCTGAAATCCCTATCCTCAGAAATGCTTCTGAGCCAAATGGGCTCTGAAC

CCTAAACAACCGTGTCCATGTATGTGGCAAGAGCTTGTGAAAAACAAAGCTGGGCCAGGCGCAGTGAC

TCACAACTGTAATCCTAGCACTTTGGGAGGCTGAAGTGGGCAGATCACTTGAGGTCAGGAGTTCAAGA

CCAGTCTGGCGAACATGGCGAAACCCTGTCTCTACTAAAAATACAAAAAGTAGCCGGGCGCGGTGGCT

CACACCTGTAGTCCCAGCTACTCGGGAGGCTGAAGCAGGAGAATCACTTGAATCCAGTTGGCGGAGGT

TGCAGTGAGCCCAGATCACGCCACTGTACTCCAGCCTGGGCAACAGAGCGAGACTTGGTAAGAAAGAG

AAAGAAAGGAAAGAATGAAGGAAGGAAGGAAGGAAGGAAGGAAGGAAGGAAGGAAGGAAGGAAGGAAG

GGAAGGAAGGGAAGGAGTCTCGCTCTGTCACCCAGGCTGGAGTGCAACGGAGCGATCTCGACTCACTG

CAAGCTCCGCCTCCCGGGTTCGCGCCATTCTCCTGCCTCAGCCTCCCGAGTAGCTGGGACTACAGGCG

CCCGCCACCACGCCCCGCTAATTTTTTGTATTTTTAGTACAGACGGGGTTTCACCGTGTTAGCCAGGA

TGGTCTCGATCTCCTGACCTCGTGATCCGCCCGCCTCGGCCTCCCAAAGCGCTGGGATTACAGGCGTG

AGCCACCGCGCCCGGCTGACCAAAGGTTTCTTGGTCCGCATTCTGCTTCTGTGGAATGAGCCAGGAGC

CAGTTAGGCCTGATTTGACATCTGATTTCCGGAGGAAAACCCAGACTCTGCCCTGGGCAACAAACTGA

ATCCTGAACTTGAGGTCACAGGGCAGGTGTGAGGAGCGGAGAGCAGCAAGAGTGAAAGGGAGGCCTGT

GGTCATTCCATACACACAAGAGATCAGTTCCTCCAAGGTCAGGGGACAGAGAGCACAGGGATCCAGCG

CCAAGCGCAAGGCCCCCAGAAGAAGCCAGAGAGTCGGGGAGGGGGCGGGGGGGAATCGGTCCCAGCAG

GTGGGAAGGATTCTGGGACCAGACCTAAGGGATCATGAGCACAGCTGCTGCAGGCAGACGGGCCCCTG

GAGAAGCTGGGGACAAGCTGGAATAGAGACTTCATTGCGGGAAGGGCTGTCAGGGAGGCCTCCTGGGG

TGGAAAAGGGTGGTCAGGAGGCTCCTGGAGGCGGCGCGGCCCCGGGGGTCCAACTCACCTGGGGCCCG

GCCACCGCGCTCTCGACCGCCGCCTCTGCCCGCGCAGCACGGGCACAGCTCGCCAGCACTGCGAACCC

GGATGGGTCGTCGGGCGCGGCCCTCAGCAGAGCTGCCTTCACAGATGTGGTGCCCAGGTCAATGCCGA

GGGTGATCGGCCGCGCAGCCATTATCTCCCTGACCCGCGCAGCTCCAGTCTGCAGCCAGCGGCCCCAC

AAGTCCGCGCTCTTCGCCCAGGGGGGCGGGGCAGGGGCGGGGAGTCGCCTGCCAATCTTTCAGCCACA

CCCAACATGGAGGCTTCTCGTCTTCCCACTGGCCGGGGAAGGCGAGCTTCCACGCAACCTCTCGGCGG

GCCCCGGCTATAGGCGGAGAGGCGGCGGAAGGCGGGACCTAAAGGGGGCCCCGCCCCACGGGCTC

ATGATCCGAAACTGGCTGACCATATTCATCCTTTTTCCC

CTCAAACTTGTCGAAAAATGCGAGTCTAGCGTCAGCCTCACAGTCCCTCCGGTCGTGAAACTCGAAAA

TGGCTCCAGCACCAATGTGAGTCTGACGCTTAGGCCCCCCTTGAACGCTACCCTCGTTATTACGTTTG

AGATTACTTTTAGGTCTAAAAATATCACTATACTGGAACTGCCTGATGAAGTGGTTGTGCCTCCTGGC

GTCACCAATTCAAGTTTCCAGGTTACCTCACAAAACGTAGGGCAACTTACAGTCTACTTGCATGGAAA

CCATAGCAATCAGACTGGGCCGCGCATCCGCTTCCTCGTTATTCGAAGTTCAGCGATATCAATTATCA

ATCAAGTGATAGGATGGATATACTTTGTCGCCTGGAGTATTAGCTTTTATCCCCAGGTAATTATGAAT

TGGAGAAGAAAATCTGTTATAGGGTTGTCATTTGACTTCGTGGCACTCAATCTTACGGGCTTTGTGGC

ATATTCCGTATTTAACATAGGACTCCTTTGGGTGCCGTATATCAAAGAGCAATTTCTTCTCAAGTACC

CCAATGGGGTAAATCCAGTGAACTCTAATGACGTCTTTTTCTCTTTGCATGCGGTCGTGCTTACACTG

ATCATAATCGTACAGTGCTGCCTTTATGAGCGGGGAGGCCAGAGGGTATCATGGCCTGCAATCGGTTT

TTTGGTCCTTGCCTGGCTTTTCGCTTTCGTTACTATGATCGTTGCGGCAGTGGGCGTAACAACATGGC

TTCAATTTCTCTTTTGTTTTTCTTATATAAAGCTGGCAGTCACTCTGGTCAAGTATTTTCCCCAAGCA

TACATGAACTTTTATTACAAGTCTACTGAGGGGTGGTCCATTGGGAACGTCCTCCTTGATTTTACAGG

TGGCTCCTTCAGTTTGTTGCAAATGTTTCTCCAAAGCTATAATAATGACCAGTGGACATTGATATTTG

GCGACCCGACTAAATTCGGGCTCGGTGTATTCTCTATCGTTTTCGACGTTGTATTCTTTATACAGCAC

TTCTGTCTCTATAGAAAACGCCCTGGTCTTCAGGCTGCCAGAACCGGTAGCGGTTCTCGGCTGAGACA

AGATTGGGCGCCATCTTTGCAACCCAAGGCGTTGCCACAAACAACTTCTGTAAGCGCATCCTCTTTGA

AGGGAGACTATAAAGATGATGACGATAAGTA

The GNS protein sequence is:

	(SEQ ID NO: 68)
	MRLLPLAPGRLRRGSPRHLPSCSPALLLLVLGGC

	LGVFGVAAGTRRPNVVLLLTDDQDEVLGGMTPLK

	KTKALIGEMGMTFSSAYVPSALCCPSRASILTGK

	YPHNHHVVNNTLEGNCSSKSWQKIQEPNTFPAIL

	RSMCGYQTFFAGKYLNEYGAPDAGGLEHVPLGWS

	YWYALEKNSKYYNYTLSINGKARKHGENYSVDYL

	TDVLANVSLDFLDYKSNFEPFFMMIATPAPHSPW

	TAAPQYQKAFQNVFAPRNKNFNIHGTNKHWLIRQ

	AKTPMTNSSIQFLDNAFRKRWQTLLSVDDLVEKL

	VKRLEFTGELNNTYIFYTSDNGYHTGQFSLPIDK

	RQLYEFDIKVPLLVRGPGIKPNQTSKMLVANIDL

	GPTILDIAGYDLNKTQMDGMSLLPILRGASNLTW

	RSDVLVEYQGEGRNVTDPTCPSLSPGVSQCFPDC

	VCEDAYNNTYACVRTMSALWNLQYCEFDDQEVFV

	EVYNLTADPDQITNIAKTIDPELLGKMNYRLMML

	QSCSGPTCRTPGVFDPGYRFDPRLMFSNRGSVRT

	RRFSKHLLDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for GNS and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

GNS-DNA

	(SEQ ID NO: 69)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	TCTTGCCTAGGCTAGATTGCAGTGGTGCAGTCATA

	GCTCACTGCAGCCTCGAACTTCTGGGCACAAACAA

	TCCTCCCACCTCAGCCTCTTAAGTAGCTGGGACTA

	CGGGCATGCACTACCATGCCCAGCTAATTTTTATA

	TTTTTTGTAGAGCCAGGGTCTTGCTGTGTTGCCCA

	GACTGGTCTCAAACTCCTGGCCTCAAGCAGTCCTC

	CTGCCTCTGCCTTCTAAAGTGCTGGGATAACAGGT

	GTGAGCCACTGCACCGGGCCTAAAGTCTAAATTCC

	TATGTGGCCCTGCATCACTGACTCCTGCCTGCCTC

	TCTGACCTCATCTTACCTCACTCTCCCTTTTCCTC

	CCTAAGCTGCTTCCATCACCACACTGGCTTCTCTG

	TTTCCTCCCAGGTGCCTCTCTCCTTCCTCTGGCAG

	CCCTTGTACTTGCTATATACCCAGCCTGAAACATC

	TTCTGAATTTTTGCATGACTGACTCCTCATCGCAT

	GGCTCTCCGTACTGAAGTTACCTCCTGAGAGGCCT

	TTTCTGACCACCCATCTAAAGTAGTTCCCCTTTCT

	TCCCACCAGCTGTAATCACATCATCCTGGTTTATT

	CTCTTCATAGCACTTACCACCACCTGAAATGACTG

	ATTGTTTACCTGTTAGTTGTTTTAAGTCCCCAAGA

	GTGCAGAGATCATGAGGGCGAGGACCATCTATCCT

	CTGTCCACCACTGCCTAGATTAATGCCTGGCACGT

	GTTGTGTTTAATAAATGTTATTACACAGTCTTACT

	GTAAATCAGATATTCTTTCATAATTTTACATATTT

	ATCAGACAGTTTGACAATTAAGTCCCATACAGTCA

	ACTCTGGTCCTTTTCTCCACTTACTCTTTGAATCC

	TCATTTCACCTTCAAATGTCCTCACAGTAACTCAG

	GTGTTTTATCAAACCAATGTCTCATGAAGCCCAGT

	ATTCTTTTCCTGAGGATGGTACCCAGTGATGCTTT

	GAGGAGAGTATGGCTGACGACCTTGCTTCGGAAGG

	CTGGCGAAGATCTTCTGCTTCTCTAGCGTCTCTTG

	GCAGCCTCTTCTGGATCTACTCATGAAATTATCAG

	AGCTTTTTTTGGATAAGTTAGAATCCTCATCCTTT

	TGAGTTTATGGATTCCGTAGGTTTTGTATTTATAT

	AAAACAATGGGGAAAAGTCATTTATACATTATTGA

	AAATGAGGCTCCTGTTAACCTACTTATGTATTTAG

	ATTAGCAATCTTTAAGGAATTATTGCACAACAAAA

	TATACTTAAAACTGCATTCAATATGGTCTTAGGTT

	TCAAAGGTATCTCAGAATCATCGTGAACTGACCCA

	TATTACAACTAAAATGACCTCTTCAATGGTGCTTT

	TTGCTGTCTGGAATCTGAAGAATATTAGTGTTCCT

	TTTATTCGTATTTAAATGACTGTCTTCATACTTTT

	GTCCTCATTCTAGAAGGAAGTTAATTGTTCTCTGT

	ACTTGTAAGGATCTTAGATTTTGAACAAAAGATGA

	AAAGCAGGACATAAAATAGTTTCTGAGTAGCCTTT

	TGCCTGACAGTGTTCTGTGTACACCTGCTTGCCAC

	CTGGTTCAAGTCCTTATCCTGAGGTGCTTGTGGGT

	CTATGCAGACAACAGTAGTTGGTAGTTGAACAGTG

	TGTGTCTGGCCTCCACCACTGAGTGTGTGAGGTTT

	GAATCAGTCTCGGAGTCCTGGGGCCTTGTCTGGCT

	TTCAGAATCCAACTGTTGTCTTACTTCTGTTCTAT

	AGGGCGGAAGGCTCCCTTTATATCATTTCCCACAT

	CCTACTTCTAAAGATTAATTCATGGAGATAGGTCT

	GCCCTATCTGCCTTATCTGGGCGCTGATGACTAAT

	AAGGGCACAAAGATGTTATTGCCATCAACTGAACT

	TTCCCTTCCTGCAAGCATTTTGACTTCCTGCACTT

	TTATACAGGATGGAGCTGGCCGGAACCAAACTTGT

	CTCTAGAATCATACAAGACTCTCTTTGGCTGTTGC

	TTAGAGCTGACCATTTGGGAATAAAAGATTGAGTA

	AGATGTAACAAAACAGGAATTCTGGGCTGTGTGTC

	TCAATTTATTGGAAGAAATTGTTTAATGGATAATT

	ACTTACTTTTAC





	ATGAGGCTCCTGCCGCTCGCCCCGGGACGGCTCAG

	AAGAGGGTCTCCAAGGCACCTCCCAAGTTGTTCCC

	CCGCGCTTCTCTTGCTTGTGCTGGGAGGATGTCTC

	GGAGTTTTTGGCGTTGCGGCGGGAACACGACGGCC

	AAACGTTGTCCTCCTCTTGACGGATGATCAAGACG

	AGGTGCTGGGGGGTATGACACCGTTGAAGAAGACC

	AAAGCACTGATAGGTGAGATGGGTATGACTTTCAG

	CAGCGCTTATGTTCCGTCTGCCCTTTGCTGCCCTA

	GCCGCGCCAGTATCCTTACGGGGAAGTACCCACAT

	AATCACCATGTAGTTAACAACACCTTGGAAGGTAA

	TTGCTCTTCAAAGTCCTGGCAGAAAATTCAGGAAC

	CCAATACCTTCCCGGCCATACTTCGATCTATGTGT

	GGATATCAAACATTTTTCGCCGGGAAGTATCTCAA

	TGAATACGGTGCTCCCGATGCGGGCGGACTCGAAC

	ACGTTCCCCTGGGTTGGAGCTATTGGTACGCACTC

	GAGAAAAATTCAAAGTACTATAATTACACCTTGTC

	TATAAACGGAAAAGCCCGGAAACATGGTGAGAACT

	ATTCTGTCGACTATCTGACGGACGTGCTGGCGAAT

	GTAAGCCTCGATTTTCTTGATTACAAGAGCAATTT

	CGAACCTTTCTTTATGATGATCGCAACGCCTGCTC

	CCCATAGTCCATGGACTGCGGCACCACAATATCAA

	AAAGCGTTTCAGAATGTCTTTGCCCCACGAAACAA

	AAACTTTAATATACACGGCACCAATAAGCATTGGC

	TGATAAGACAAGCGAAGACACCCATGACCAATTCA

	AGCATACAGTTCCTGGACAACGCATTCCGCAAGAG

	GTGGCAAACGCTGCTCTCAGTTGACGATTTGGTTG

	AGAAATTGGTAAAAAGACTTGAATTTACTGGCGAA

	CTGAACAATACGTATATCTTCTATACATCTGATAA

	CGGATACCACACTGGTCAGTTTAGTCTCCCCATAG

	ATAAACGGCAGCTCTACGAGTTCGATATCAAAGTA

	CCCTTGTTGGTAAGGGGACCGGGAATAAAACCTAA

	CCAGACGTCCAAAATGCTGGTGGCAAACATCGACC

	TCGGCCCAACGATTCTGGACATCGCTGGGTATGAC

	CTTAACAAGACACAAATGGATGGCATGTCCCTGCT

	GCCAATCCTCAGAGGGGCCAGCAATCTTACATGGC

	GCTCAGATGTCCTGGTAGAATATCAAGGTGAGGGA

	CGGAATGTAACAGACCCTACCTGTCCCAGCTTGAG

	CCCTGGTGTATCTCAGTGTTTTCCTGATTGTGTTT

	GTGAAGACGCATACAATAACACATACGCTTGTGTA

	AGGACGATGAGTGCACTTTGGAACTTGCAGTATTG

	CGAGTTTGACGATCAGGAGGTGTTTGTTGAGGTGT

	ATAACTTGACGGCAGATCCGGACCAAATTACCAAT

	ATAGCTAAAACCATTGATCCGGAACTCCTCGGCAA

	GATGAATTATAGACTTATGATGCTGCAGTCATGCA

	GTGGTCCCACATGCCGAACGCCAGGAGTGTTCGAC

	CCTGGGTACCGGTTCGATCCCCGCCTGATGTTCTC

	CAATAGGGGTTCAGTTCGGACGAGGAGATTCTCAA

	AGCACCTCCTTGACTATAAAGATGACGACGACAAG

	TA

The HGSNAT protein sequence is:

(SEQ ID NO: 70)

MSGAGRALAALLLAASVLSAALLAPGGSSGRDAQAAPPRDLDKKRHAELK

MDQALLLIHNELLWTNLTVYWKSECCYHCLFQVLVNVPQSPKAGKPSAAA

ASVSTQHGSILQLNDTLEEKEVCRLEYRFGEFGNYSLLVKNIHNGVSEIA

CDLAVNEDPVDSNLPVSIAFLIGLAVIIVISFLRLLLSLDDFNNWISKAI

SSRETDRLINSELGSPSRTDPLDGDVQPATWRLSALPPRLRSVDTFRGIA

LILMVFVNYGGGKYWYFKHASWNGLTVADLVFPWFVFIMGSSIFLSMTSI

LQRGCSKFRLLGKIAWRSFLLICIGIIIVNPNYCLGPLSWDKVRIPGVLQ

RLGVTYFVVAVLELLFAKPVPEHCASERSCLSLRDITSSWPQWLLILVLE

GLWLGLTFLLPVPGCPTGYLGPGGIGDFGKYPNCTGGAAGYIDRLLLGDD

HLYQHPSSAVLYHTEVAYDPEGILGTINSIVMAFLGVQAGKILLYYKART

KDILIRFTAWCCILGLISVALTKVSENEGFIPVNKNLWSLSYVTTLSSFA

FFILLVLYPVVDVKGLWTGTPFFYPGMNSILVYVGHEVFENYFPFQWKLK

DNQSHKEHLTQNIVATALWVLIAYILYRKKIFWKIDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for HGSNAT and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

HGSNAT-DNA

	(SEQ ID NO: 71)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	CAAAAGGGTAAAGTTGTTTACCATTCGGACTTTTG

	GCCCCCTTCTCCGTGTACAAATTGGTAAAAAGCCT

	CAGGATTTTTGAGCTGTCCTTATCCCTCCCCTTGT

	TTCTTTTCTTTTCTTTTTTTTTTTGAGACAGGGTC

	TCACTCTGTCGCCCAGGCTGGAGTGCAGTGATGTG

	GTCTTGGCTCACTGCAACCTCCGTTCAAACGATTC

	TCCTGCCTCAACCTCCTAAGTAGCTGGGAGTACAG

	GCAGGCACCACCACTGCTGGCTAATTTTTGTATTT

	TTAGTAGAGACAGGGTTTCACCATGTTGGCCAGGC

	TGGTCTTGAACTCCTGACCTGCCAGGCTGGAGTGC

	AGTGGCATAATCTTGGCTCACTGCAAACTCTGCCT

	CCCGGATTCAAGTGATTCTCCTGCCTCAGCCTCCC

	AAGTAGCTGGGACTACAGGTACTTGCCATGACGCC

	CGGCTAATTTTTTGTATTTTTAGTAGTGACAGGGT

	TTCACCATGTTGGCCAGGATGGTCTCGATTTCTTG

	ACCTCGTGATCCACCTGCCTTGGCCTCCCAAAGTG

	CTGGGATTACAGGCGTGAGCCACTGTGCCCGGCCA

	TATGTGTTTAATAACCCAGTTTGTCTCTTCTTGCC

	TTCAGGACATCAAACGGTCATGCTACTGGAAGCTC

	GGACCATGGCCCCTTCTGCTGGGCACCCTTAGATA

	GGCCTCTGAGGGAGATCTGATTGCCATTTTCCCGA

	AACAGTGCCCCCTATCAGCAGGAAGCAGTTAAGAT

	CTGTCTTTGTCCCTATTCTTATCCTAGTGGCAGTT

	AGATAGACTTCTTTAGAGCGAGGAATGAGCCAGAT

	GGGAAGGGTTCCCTGGTAAAACTCCAACTGGCCTG

	TGCACTGCTCTGGGGTGGAGCCACAGAAGTTCGCT

	CCTCAGTTTGCAGCTGGGAGGAGCCTGGCCCTCCT

	CTTCCTCGGGAGAACCTGGGATTTAATCTGTGAGG

	TGGGAAGCCTACAACTAGGAGAACTCTGGCTCTGC

	TGAGTCCCTGTTCCCCTTTTTCTTCCTTTACACCC

	AATAAAACCCTGCCTTACCCTTCAAATTGTCTGCG

	AGCCTAATCTTTCCTGGCGTGTGACAAGGACCTCC

	ACCGTCTTTAGCTGAACTAAGGAAAAGTCCCGCAA

	CATTTTCATGTGTGCCTTTGTATTTTTATTACATA

	TATGGATGTCCCTAAACAACATACATTATTGTTTT

	GCTTTATATATTAAAAAGGCATTCTGCAATTTGCA

	TTTTCCCCAGCCTTGTGTTTTGAGATTTATCCATG

	TGGATGCGGGAAGCTGTAGAGCACTTCCCCTGCTG

	TGGGGCAGTTTGTTTTCTCGCTGGCGGACATTCAG

	CTTGTTGACAAGGTTTTGCAATTACAAGCACCTCT

	GCTATAGGTATTACTGTACCTGTCCCTTATACCTG

	CGATAGTGTCTCCCGGCATACAGCCAAGAATGGGT

	GTGCACACTCCTCGACCTGCTCCCTTCCACTTCGT

	CAGCCTGGCCGGGGGCTCTTTCAAGAGGTGGGGCC

	GCTTGGTGGCTTCTAAGTCGTGCCTCTCCCTCTGG

	GTAATAAACGCCTTGGGGGCAGGGACTTTGCAGTC

	TTGCTTATTGAGGCGTTTTCCAAAACAGGGACCGA

	GGGCTCTAACACCCGTGCGGAGGGTGGCCGGCGGG

	GGCGGGAGTTTGGCAGAATGCGGCTGGAGAAGGTG

	GGGCGAGGGAAGAAGGCTCCTGGCCTGCAGGGGCC

	TCTCTTCCGGCTGTTTTTCCTCCCGAAGACAAACA

	CTCCCAACGCAGGGGCGCAGAGCCTCGGCTTCTTT

	CCTGGCCAGCTCCCTCAAGGCAGGGCCTAGCATGG

	CCCGGCCCAGCCCCTAGGTTCCCCCGCCCGCGGCT

	CCCGGGCGCCTCCGCCTCCCGCGGTGACTCAGGCG

	GCGGTGACGGCAGCGGAGGGGGCGATGACGGGCGC

	GCGGGCGTCCGCGGCGGAGCAGCGCAGGGCGGGGC

	GCAG





	ATGAGCGGCGCGGGCAGAGCTTTGGCGGCCCTTCT

	TCTGGCGGCATCTGTGCTTAGTGCAGCACTCTTGG

	CTCCTGGGGGAAGTAGTGGTCGAGACGCTCAGGCC

	GCGCCACCGAGAGATCTGGATAAGAAACGGCACGC

	AGAGCTCAAGATGGACCAGGCACTTCTGCTCATAC

	ACAACGAATTGCTTTGGACCAATTTGACGGTATAT

	TGGAAGAGTGAGTGTTGTTACCATTGTCTCTTCCA

	GGTTCTTGTGAACGTGCCACAGAGTCCTAAAGCTG

	GGAAGCCTAGTGCTGCGGCGGCATCTGTTTCTACA

	CAGCATGGTAGCATACTGCAACTTAACGACACTCT

	GGAGGAAAAAGAGGTATGTCGATTGGAATATAGAT

	TTGGCGAGTTTGGCAATTATTCATTGCTGGTTAAA

	AATATCCACAACGGTGTTAGCGAGATTGCATGCGA

	TCTCGCTGTTAATGAAGATCCGGTCGATTCTAACC

	TGCCGGTCTCAATAGCTTTTCTGATAGGGCTGGCA

	GTGATTATCGTAATCTCATTCTTGAGGTTGCTCCT

	CTCACTCGACGATTTTAACAACTGGATCAGCAAGG

	CAATTAGTTCCAGAGAAACTGACAGGCTCATTAAC

	AGCGAACTTGGTTCACCCAGTCGCACAGACCCCCT

	TGACGGCGATGTGCAACCTGCGACATGGAGATTGT

	CCGCCCTCCCTCCGCGACTGCGATCAGTGGATACC

	TTCAGAGGAATTGCGCTCATTCTTATGGTTTTCGT

	AAATTACGGAGGGGGTAAATACTGGTATTTTAAAC

	ATGCCAGTTGGAATGGACTCACCGTTGCAGATCTC

	GTGTTCCCGTGGTTCGTCTTCATTATGGGCTCATC

	CATTTTTCTCAGCATGACCTCCATCCTCCAGCGAG

	GATGCTCCAAATTTCGCCTCCTCGGAAAGATAGCC

	TGGCGAAGTTTCCTGTTGATTTGTATTGGGATTAT

	TATCGTAAACCCCAACTACTGTCTGGGGCCTTTGT

	CATGGGATAAAGTAAGGATACCTGGAGTTCTCCAA

	AGACTCGGCGTGACTTATTTCGTCGTTGCAGTTCT

	CGAACTCCTCTTTGCCAAGCCGGTCCCCGAGCACT

	GCGCATCAGAACGCTCATGCCTCAGCCTTCGCGAC

	ATAACAAGCAGTTGGCCTCAGTGGCTGCTTATCCT

	TGTCCTGGAAGGCCTTTGGCTTGGTCTTACCTTTT

	TGCTTCCCGTACCAGGATGCCCAACTGGCTACCTC

	GGGCCCGGAGGGATCGGCGACTTTGGAAAGTATCC

	CAATTGTACCGGAGGAGCCGCGGGCTATATTGATC

	GACTTCTCTTGGGGGATGACCATCTTTACCAACAC

	CCGTCTAGTGCAGTACTTTACCATACCGAGGTAGC

	CTATGACCCGGAGGGAATCCTTGGTACCATTAACT

	CAATAGTCATGGCGTTCCTTGGTGTACAGGCTGGA

	AAAATTCTCCTTTACTACAAAGCTCGAACGAAGGA

	TATTCTTATAAGATTTACAGCGTGGTGCTGCATCC

	TTGGACTGATTTCTGTGGCCCTGACGAAGGTATCC

	GAGAACGAGGGATTCATCCCCGTTAATAAGAATCT

	GTGGAGCCTTTCCTATGTCACCACGCTCAGCTCTT

	TTGCTTTCTTCATTTTGCTTGTTCTCTATCCAGTA

	GTGGATGTAAAGGGCCTGTGGACAGGGACTCCGTT

	CTTCTACCCGGGGATGAACAGCATACTTGTTTATG

	TGGGACATGAAGTATTTGAAAACTATTTCCCCTTT

	CAATGGAAACTTAAAGATAATCAAAGTCACAAAGA

	GCACTTGACACAAAACATTGTAGCGACAGCGTTGT

	GGGTCCTCATTGCATATATCCTCTACAGAAAGAAG

	ATATTTTGGAAGATAGACTATAAAGATGATGATGA

	TAAGTA

The NeuroD1 protein sequence is:

(SEQ ID NO: 72)

MTKSYSESGLMGEPQPQGPPSWTDECLSSQDEEHEADKKEDDLETMNAEE

DSLRNGGEEEDEDEDLEEEEEEEEEDDDQKPKRRGPKKKKMTKARLERFK

LRRMKANARERNRMHGLNAALDNLRKVVPCYSKTQKLSKIETLRLAKNYI

WALSEILRSGKSPDLVSFVQTLCKGLSQPTTNLVAGCLQLNPRTFLPEQN

QDMPPHLPTASASFPVHPYSYQSPGLPSPPYGTMDSSHVFHVKPPPHAYS

AALEPFFESPLTDCTSPSFDGPLSPPLSINGNFSFKHEPSAEFEKNYAFT

MHYPAATLAGAQSHGSIFSGTAAPRCEIPIDNIMSFDSHSHHERVMSAQL

NAIFHDDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for NeuroD1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

NeuroD1-DNA

	(SEQ ID NO: 73)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	TGTCGCGAACTGGAGGTTTCTGCTTTCTGGGGGTG

	AGGACAAGGATTTAGCAGAGGGACCTCTAGGCTAC

	CCCTGACCTCGAAGCTAGCTGGAGTCCTTTCTCCC

	ACTCCGTCCCCAGTTCTCCTCCGGAACTCCCCAAA

	GTTGGAAAAATGAGAGCCTAGGCACGAGTTGCATA

	TGCCTGCGGCATAGTGTGCTGCCAGTCGGGGCAGC

	TGCCGGGTGAGGGGCAGTACTTAACCTGGCCCTGC

	CGCAAACGTCGGGCTGTGTACTATCTTCTAAGGGC

	TAGTACTCCGACGCCCTCGAGTTTTAGCCCAACAC

	TGAAGATACAGACAGTACAGGGGTAAATCCTGGGC

	CCAGGCTCCTGGGTGAATTATTTTATCCTCCCCTA

	TGCGCTTTGTCAGCCAAGTGAATGTAGAAAAGCAA

	GCAAAACTCCAGAACCCCACAACGATTCACGCCAC

	GCTTCGGTGGCACAACTCTTTTCTTCCCCTTTTAG

	ATTAGTACTGTGCAGGGAAGGCTGCACCTGCTACC

	TTCAGACACCGGGGATCTGGCTAGGACCCTCTTCC

	TCTACCCCTACTCTCTCCTCCCGTCCCCACCCTCT

	CCCAGATGCACCCCAATCCAGCCTCTCAACCCTGT

	CGCTGTTTGCAAACTCGAGGGATGTTTAGTGCAAT

	TGCCTTTAAATAAGCAATTCACGTAAGAGGTATTT

	ATTGCAGAGGGTTTCAGGTCTGGGAGGGGGTAAGG

	AGTTAGTTAATGAAGGATTAGAGGCAAAGCTGGGA

	AAGAACGAGTAGACAGGCCAGTTTCTTTCTTTCTT

	TTTAAAATCTATTATTCTGGAAAACCCTTGTTCGA

	AAAGAGTAAAGAATTGCTATTTGGGCAACAATATG

	GCTCCTTCAGAACTGGTCGAGAATTTCTGTGGTCT

	TTGCTTGACCTTCAAGTTGGGAGTTTCAAATCAGA

	GGCTTTGAAGTCTGGAGGAGTTCGCAGCCATTAAT

	CCTTCCGGGGTTGTGCTAAAGGCGTTTCTAGAAAG

	AGATCGGAAAGACCCGTTCAGCACAGGCCCATTGC

	ATCTTCTCTTTGAAAACGCTTGTCTGAATCAAGTA

	TCTTATACAGACCCGAGACTGAGGGGAGCAAGAGC

	GCAGGTCCGGCTGTGGCGAGGTGTCCACCACCGTA

	CTTGTGGTCAATGCCTGGACTAGAGAGCCGAGGCC

	GTCCAGGGCGGGCTGGCCAGGGAACCAGAAGCGTC

	CCTGGACTGGCTGAGTGAGGGTGCCCTTTCCAGCA

	ACCCAGTGGCCATGGTTAAAAGGCAGAATACAACC

	GTCAAAGAAGTCCCCAGCTCCCACATAAGAGAGCG

	CAAAGGCCAATGGAACCAAGGCCTCCTCGCGAGAA

	CTAGCTCGCTTTGAGGACAAGATCCTGGGGATGGA

	GGTGGGGACTGACGTAGTGAGAGGGTCTGGAGGAA

	CAGTCGTAGCTGAAGGTCAGGACATGAAGAATTGC

	ACGTATGGTAGGGATAGAATGGCAGGTACAACTTT

	TCTGTACTGAAGGAACTGGGAGAGGACGATCCGGT

	TAGGGAGGTTGGGGAACTAATCTCAACGCTGCGTT

	TACAGATGAAGCCGCTTTTATATGGCGTATATGTT

	TGCTTAGAGGGGCCGACGGAGATTAGGAGAAGCCA

	TCCTTTGGCGCCAATGATCAAAGCGTCTGCCAAGG

	AGAAGAAGCCAAGGGATGGGCCTTTCAGAGAGGGC

	AAGGAGTCATGCTGCTCTGGATGCCAGTGTCAGGA

	CAAGAAATCGAAAGGAGCGAGGACTCTTCACTGCG

	TGCCTCAGTCTCCCCGCTTCTGCCTCTTTCACCTC

	TGTCCTACTTCCGGCGCGAAAGCAGGCCACTCGCT

	CTGATCTAGACCTAGTTAACATTAGCTTTTCCCTT

	CCTTCCCTCAAATCCCCTCCCCCCTCTATCCCCGT

	CCCTTCTGCCGCCTGAAAGGGTTAATCTCTCCTGC

	GGGTAAAAACAGGTCCGCGGAGTCTCTAACTGGCG

	ACAGATGGGCCACTTTCTTCTGGCCACAAAGGGGC

	CGGAATGGAGCGCTCCGCGGCATACAAATGGGCAG

	GTCACGTGGTTCCAGGCTCTTGGCTGGACCGGGAA

	GACCATATGGCGCATGCCGGGGAGGAAGGAGGAGG

	GGCGGGGGTAGGGGTGGAGGGTGAGGGGAGCGGTT

	GTCGGAGGAGGGCGGGAGACGAGCAAGGCGTGGGG

	AGAAGTGGGGAGGAGGGGAGAACGGGGAGCGCACA

	GCCTGGACGCGTGCGCAGGCGTCAGGCGCATAGAC

	CTGCTAGCCCCTCAGCTAGCGGCCCCGCCCGCGCT

	TAGCATCACTAACTGGGCTATATAACCTGAGCGCC

	CGCGCGGCCACGA



	ATGACGAAAAGCTACAGCGAGTCAGGGTTGATGGG

	GGAGCCTCAACCCCAAGGGCCCCCATCTTGGACGG

	ATGAATGCCTGTCATCACAAGACGAGGAACACGAG

	GCAGACAAGAAGGAGGACGACCTGGAAACGATGAA

	CGCTGAGGAAGACAGCCTCAGGAACGGAGGCGAAG

	AAGAAGATGAAGACGAAGACCTGGAGGAAGAGGAG

	GAAGAAGAGGAAGAGGATGACGACCAGAAGCCCAA

	ACGGCGCGGGCCCAAAAAGAAGAAAATGACTAAAG

	CCAGGTTGGAGCGGTTCAAACTCAGGAGGATGAAG

	GCTAATGCAAGAGAGAGGAATCGCATGCATGGACT

	CAACGCCGCTCTCGATAACCTCCGGAAGGTCGTGC

	CATGTTATTCTAAAACACAAAAACTGTCCAAAATC

	GAGACCCTGAGGTTGGCAAAGAACTACATATGGGC

	GCTCAGCGAGATTCTTAGATCTGGTAAGTCTCCGG

	ACCTCGTTTCCTTCGTCCAAACCCTGTGTAAAGGT

	CTGTCACAGCCAACCACCAACCTTGTAGCTGGGTG

	CTTGCAGTTGAACCCCCGCACTTTCCTTCCTGAAC

	AAAATCAGGATATGCCTCCGCACCTCCCTACCGCC

	AGTGCATCTTTTCCGGTACATCCCTACTCATACCA

	AAGCCCGGGTCTGCCTAGTCCGCCGTATGGTACAA

	TGGATTCCTCCCACGTGTTCCACGTAAAACCCCCA

	CCTCATGCCTATTCCGCTGCCTTGGAGCCTTTTTT

	TGAGTCTCCGTTGACAGACTGCACCAGTCCTTCTT

	TTGATGGGCCCCTGTCACCTCCTCTTAGCATCAAT

	GGAAACTTCTCTTTCAAACACGAGCCAAGTGCAGA

	GTTCGAGAAAAATTATGCTTTTACTATGCACTACC

	CCGCAGCTACCCTGGCAGGTGCGCAAAGTCATGGA

	TCTATCTTTTCAGGAACGGCTGCGCCGAGGTGTGA

	GATACCCATTGATAATATCATGAGTTTCGATAGCC

	ACAGCCATCACGAGCGCGTGATGAGCGCTCAACTG

	AATGCAATTTTTCATGACGACTATAAAGATGACGA

	CGATAAGTA

The SLC17A5 protein sequence is:

(SEQ ID NO: 74)

MRSPVRDLARNDGEESTDRTPLLPGAPRAEAAPVCCSARYNLAILAFFGF

FIVYALRVNLSVALVDMVDSNTTLEDNRTSKACPEHSAPIKVHHNQTGKK

YQWDAETQGWILGSFFYGYIITQIPGGYVASKIGGKMLLGFGILGTAVLT

LFTPIAADLGVGPLIVLRALEGLGEGVTFPAMHAMWSSWAPPLERSKLLS

ISYAGAQLGTVISLPLSGIICYYMNWTYVFYFFGTIGIFWFLLWIWLVSD

TPQKHKRISHYEKEYILSSLRNQLSSQKSVPWVPILKSLPLWAIVVAHFS

YNWTFYTLLTLLPTYMKEILRFNVQENGFLSSLPYLGSWLCMILSGQAAD

NLRAKWNFSTLCVRRIFSLIGMIGPAVFLVAAGFIGCDYSLAVAFLTIST

TLGGFCSSGFSINHLDIAPSYAGILLGITNTFATIPGMVGPVIAKSLTPD

NTVGEWQTVFYIAAAINVFGAIFFTLFAKGEVQNWALNDHHGHRHDYKDD

DDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for SLC17A5 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

SLC17A5-DNA

	(SEQ ID NO: 75)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	GTCAAACTAAGCAAAGGGTATGGAGAACTCACTGT

	ACTATCTTTGCAACGTTTATGTAAACCTAAAATTT

	CTAAAATAAAAAATATTAAAAAAAAGAATTCCTCT

	GGTTTGAAGTCTTTCAAGTCTGTTGCAACTATATT

	CCCTGGCCCAAGTTATCTATAACCTTGGTCATCTG

	GGAGCTTGTTAGCAATACAGAATCTCAGGCACATC

	CCAGATGTACTGGGCTTCTCAGAGAAACAGCCCCA

	ATTGGGTAGTTTGGGTAATAAAGTTTTGGCTTTCA

	CAATATAGGCTGGCCAACTCGACCCAAAACAGATG

	ATGGTGCACATGCCTGGAGACCCAGGAGGACCTGT

	GGTGCAGATGAAGTCTGAGGACACTCTGCTGGAGA

	AGTCTCCCTTGCTTGGGCAAGAGAAGTGGGGGAGT

	CGGATTTTTTATTCTAGATAGGCCTTCAACTGATT

	GAAGGAAGCCCACCCAGATTATGGAAGGCAGCCAT

	CTTTACCCAGAGTTCAATGATTTTAGTGTTAACCT

	CATCCAAAGACAACCAGCAAGTTAACACATAAAAT

	TAACCATCACACCAGATCTGCTGAATCTGAATCTA

	CATTTTAAAAGGATTTGCCAGGTGACTCTGCATAA

	TCCAGTTTGAAAATTGCATTTTTCTACAACCCTTA

	GAGAGTGTAAAAATCTACATGGTAATTCAGTAACT

	CAGAAACAAGGCTTGATTTGCTGCTTTAACTTGAG

	AACTTTTCTAGCATTTACTGATCTGGAGGTAGGGA

	TGCCTACAGAAATCCTCGCCCTCCCTTCTAGGTAA

	AGGACCCTCCCCTTTGTTCTCACGTGAACCTCACT

	AAGCTTCTAACCTGGTTTACTGAATTAGTGGCTTG

	TGGGTCTTCCCTGTCTACACCAAACTCTCCGAGAG

	CATCTACATCTTATTTTCTACCTCCACAGAGACAG

	CCTTTATTGGCGGATCCAGATCTGCCCTGCCAAGG

	CAAGCTGGGTTTTCCTAGTTTCACTTTCAGAGTCC

	GCAAATGCTAGCACAATCTATCCAAATTCTATCGT

	GGAGATAAAGCATGCACACATACATCAGAGGATTC

	CATAGGTCGAACCATATGAAACTACCTATGTTGAT

	TACTTTTACCTATAAAACCAGCAACATCATAGGAC

	TCAACAAATTTCCATTGCTGTTTTTCCGCCTAAAA

	GCTATTTTAGGACCAGGAGCAGTGGCTCACGCCTG

	TAATCCCAGCACTTTGGGAGGCCGAGGAGGATTGC

	TTGAGCCCAGGAGTTCGAAACCAGCTTGGGCAATA

	TAATAAGACCAAGCCTCTACAAAAATAAAAAGTAG

	AAACAAAATTAGCCAGATGTGGTGGCATGCTCCTG

	TAGTCCCAGCTACTTGGGGGAGCTGAAGAGAATCG

	CTTGAGTCCAGGAGTTGGAGGTTGCAGTGAGCTGC

	GATTGCGCCACTGCATTCCAGCTTGGGCAACAGAG

	CAAGACCCTGCCTGGGAGAAAAAAAAAAAAAAAAA

	AAAGCTATTTTAGGGACAATATGCCGCCACAGTCA

	CGAGGTAAATATGCCGCCACAGTCACGAGGTAGAT

	CGTACAAAGGTTGCTAACAGTATGGCAACTGACCG

	CTAACAAGCGAATGTCAAGTTGTGAAATGCACACC

	ACCCACTTCCCTAGTTCGGTCGAAGCCCTCCCCTT

	AATTATGTGCAATTCAAGTCCCCACTGCCCGCCCG

	CAAGCCCCCACTCATCCTCGCTGCGGGCAGGGTGG

	CCCCTGCACTTTACAAGGGGGTGCAGGAGCGGGAG

	ACGGTCGTCCGAACACCGGCTCCCCGGCATGCGTA

	GACCGGCGGGCGGAGCGGGCTCACTTTGCGCCAAT

	CCTACGAGAACTCCCAGAACTCCGCTTCCCTAGTC

	CAACCCAAGCCAGAGTTGCCCACACCTAAGATGGC

	GGCGGGGGCGGAGTCGGCGCGGCCGCCTCTGGGCG

	GGACCGCGGGGACTAGACGTGGCCGCGGGGCGGTGT

	CAT





	ATGCGGAGCCCCGTGAGAGACCTGGCCAGAAACGA

	TGGGGAAGAGTCTACAGACCGGACCCCGTTGCTTC

	CGGGTGCTCCCAGGGCAGAGGCCGCCCCTGTCTGT

	TGTAGCGCACGCTACAACCTCGCCATACTTGCTTT

	TTTCGGGTTCTTCATAGTTTACGCCCTGCGGGTGA

	ACCTTAGCGTAGCTCTTGTAGATATGGTCGACTCT

	AACACTACTCTGGAAGACAACAGAACCTCTAAAGC

	GTGTCCAGAGCACTCAGCCCCTATTAAGGTACACC

	ATAACCAAACCGGAAAAAAGTATCAGTGGGACGCG

	GAAACCCAGGGGTGGATACTCGGCTCATTTTTCTA

	CGGTTATATTATCACCCAGATTCCTGGAGGCTATG

	TAGCCTCCAAGATCGGTGGGAAAATGCTCTTGGGA

	TTCGGCATTTTGGGTACTGCGGTACTTACCCTGTT

	CACTCCTATAGCAGCCGATCTTGGGGTTGGCCCAT

	TGATCGTTCTCAGAGCCCTCGAAGGTCTGGGAGAG

	GGCGTTACATTTCCAGCAATGCACGCGATGTGGTC

	AAGTTGGGCGCCACCCTTGGAGCGAAGCAAACTTC

	TCTCCATTTCATATGCAGGGGCCCAGCTGGGAACT

	GTAATAAGTCTGCCGCTGAGCGGTATAATCTGCTA

	CTATATGAATTGGACATACGTCTTTTATTTTTTCG

	GAACAATAGGGATTTTCTGGTTTCTCTTGTGGATA

	TGGCTGGTTAGCGATACCCCGCAGAAGCACAAACG

	CATAAGTCACTATGAAAAAGAGTACATCTTGTCTT

	CACTTCGCAATCAACTGTCTTCTCAAAAGAGCGTG

	CCTTGGGTACCGATATTGAAGAGCCTGCCTCTCTG

	GGCAATCGTAGTAGCGCACTTTTCATATAATTGGA

	CCTTTTACACACTGTTGACTCTTCTCCCAACGTAT

	ATGAAGGAAATTCTCCGATTTAACGTGCAAGAAAA

	TGGATTCTTGTCATCACTTCCTTACTTGGGTTCAT

	GGCTCTGCATGATTTTGTCTGGTCAGGCGGCCGAT

	AATCTGCGCGCCAAATGGAACTTTTCTACCTTGTG

	TGTAAGACGAATCTTTTCCTTGATCGGCATGATAG

	GCCCCGCTGTGTTTTTGGTAGCAGCCGGCTTCATT

	GGGTGCGATTACTCACTGGCTGTCGCGTTTCTCAC

	AATTTCTACCACACTTGGCGGTTTTTGTAGCTCTG

	GCTTCAGCATAAACCATCTCGACATAGCACCTTCC

	TACGCGGGTATACTGTTGGGTATCACAAATACCTT

	TGCCACAATCCCAGGTATGGTCGGCCCAGTTATAG

	CCAAATCTCTGACTCCTGACAATACTGTAGGAGAA

	TGGCAAACAGTATTCTATATCGCCGCTGCTATCAA

	TGTTTTCGGGGCAATTTTCTTCACGTTGTTCGCGA

	AGGGGGAAGTCCAAAACTGGGCTCTTAATGACCAT

	CATGGGCACAGGCACGACTACAAGGATGATGATGA

	TAAGTA

The CLN6 protein sequence is:

(SEQ ID NO: 76)

MEATRRRQHLGATGGPGAQLGASFLQARHGSVSADEAARTAPFHLDLWF

YFTLQNWVLDFGRPIAMLVFPLEWFPLNKPSVGDYFHMAYNVITPFLLL

KLIERSPRTLPRSITYVSIIIFIMGASIHLVGDSVNHRLLFSGYQHHLS

VRENPIIKNLKPETLIDSFELLYYYDEYLGHCMWYIPFFLILFMYFSGC

FTASKAESLIPGPALLLVAPSGLYYWYLVTEGQIFILFIFTFFAMLALV

LHQKRKRLFLDSNGLFLFSSFALTLLLVALWVAWLWNDPVLRKKYPGVI

YVPEPWAFYTLHVSSRHDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for CLN6 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

CLN6-DNA

	(SEQ ID NO: 77)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	AAAGGCAGATGTGTTTGTATTTAAGAGATTGGAGG

	ATTAAATGAGCTAGAGAGGCTCCCTCCACACGGCC

	TCATCCTCTGATGGCTGGAAGCCCACCTCTGAGTG

	TTATTAAATCGATGATGACATCATGGGCACTGCAT

	GCCCCTCCCTCTGGAGCCCAGCAGGCTTGCAAAGC

	CTGCAGAGGATAAGAAATTGGGGTCAGAGCGGGAA

	GCCTCAATCCTTCTTCTCTGGTGTATACCGAGATC

	GATGTGTAGATCAGATTCACCAGAATTAAATCTGG

	GGCCCCGTCTGAGTGCTGTCCTTGGCTGACCCTTG

	GGCACAGTACTTCAGCTCAGGGCCTAGGTTTTTCT

	CTGCTACAAAACAGAGACAATCACCTGTTCTCCTT

	ACGACCTGGTGTGTGTGGATCAAATGAAACAAGGA

	AAGCTTTAAAGCAAATGTGTTCTTCAGGGGACCAG

	GGGCGGAGGGGGGGGATTGAGGTGTGGCCTGTGGG

	CTGCTAGAGCCCAGGATGGGGCTGATGGAAAATGA

	CTCCCAGGCAGGCAGTCCTAGCCTCCCACAGCCAA

	GAAAGGACATGCAGGTTCTGACCAAAGGGGAAAGA

	AATAAATCCAATAAACATCTCCCAAGAAGAAAGAC

	AGGAAAGAAAATAGAATGGAAATAGGCATAGCTCA

	GACCCCTCATGGTCTGGGAAGCAGACCAAGGATAC

	TGGGCAGGGGAATGGCACATTGCATTTGCATCTGA

	GGCACATCCCTTCCAAGAGGCACATCCCTTCTGCA

	GCGGCTTGAGGGCTGGGTGAGAGGAGACAGGACTG

	GAGACCTGCACATGTCGTGTGCGCCTGGTGTAGCA

	CGGGGGTGGTGGGGAGAATGGGAGTTGGTTGCAGT

	TGTTCTAGTTAGCGGCCTGGGCTGCTGACAGTGAC

	CGTGGGGAGGAGAGAGTTTGTGACATTGACAGTGG

	TGTGGAAGGTAACCTTTCTGTGGAGGGAAACCTCA

	GGTCTCGAGACGGGCTATTGTAAATAACTTAGTCC

	ACCAGTGCTGGGCTCTCTGCTAAGAGTTTCATATA

	GTCATCACAACTCCAATGTAGTAGATGCAATAGGC

	ATCTTCTTGTTACAGATGAGTAAACTGAAGCTGTG

	AGGGGTTGAGCCATTTGCCCGCATCACAGCTAGTT

	CAAAGTGGTACCACTAATGAAGTCCTGGCAATCTG

	CCCTGCAGGCTGTTCTCAGGTTTGACCTTTGGTTC

	TGTTTTTCACTTGAGGATGCTGTCCCCTTAGGTTC

	TGTCTGTCTTGTATGCTCCGGTGACTCACGAGCCT

	GTCCACTGGGTTCCCAGGTCTCGCCTCAGCTTCAG

	ACCACCGGTCCAACAGCCTTCCTGACCCACCTCAA

	GCTTAATGCGTTCAAAGATGAATCTTTATTTACAC

	GAAATCTGCTCTAGCCCACTTCCCGGGGGGTGCCT

	CCACTATCCACCCAGGGGCAGAATCCGGCCACTGC

	CTGAACTCTTCTCCCCTCACCCCCCACATCCAGAG

	TCCCAGTCACCATACCTCTCACACGACTCTAGATG

	TCACCAACTCGCTCCTTCCCACTGTCACCGCCAGC

	CCCTCACTAGAACATCTGCAACCCACGTGCTGCAC

	CACGCGGGTGCTACACGTGCAGGTCTCCAGTCCTG

	CCTCCTCTCCTCCACCCGTAAGAACAAGGGAAATC

	CCACAGAACCCTCTGCCCCGGGGAGCATTTAAATC

	AGCTTAGTCTATCTCCAGGTCGCTACCCCGCCCCA

	GCCCAACACCCAGGCCTCAGTCACCCTAGACAGAG

	ATTCCCGTACCCAGGCAGGCAAGGGACCCTAACAC

	ATCCCAGCCCGCTGGTGGTCACGCGCGCAGCCCGG

	ACAGGCCAGCAGCCCCCGCCCCGCGAGGACGCCAC

	ACTCGCAGCGCCCGGCCCTCCCCTAGCGGAGCCCG

	CCCCGTCTCCCTCCGCCCCGCCTCCCTCCGCTCCG

	CTCCGCTCCGCTCCGCCCCGCCCCGGGTCCGCAGC

	CCCG





	ATGGAAGCCACTAGAAGAAGACAACACTTGGGAGC

	GACAGGTGGACCCGGTGCGCAGTTGGGAGCCTCCT

	TTCTTCAAGCTAGGCATGGTAGCGTAAGTGCTGAC

	GAAGCCGCACGCACTGCACCTTTCCACTTGGATTT

	GTGGTTTTATTTCACCCTCCAGAATTGGGTTCTGG

	ATTTCGGGCGACCCATCGCAATGCTGGTGTTCCCG

	CTTGAGTGGTTCCCCCTTAATAAACCCAGCGTGGG

	AGACTATTTTCACATGGCATACAACGTGATAACCC

	CTTTCTTGCTCCTTAAACTCATAGAGCGCAGCCCC

	AGAACGCTTCCCAGAAGCATAACGTATGTGTCCAT

	TATTATCTTCATAATGGGTGCCTCAATACACCTTG

	TCGGAGATAGCGTAAATCACCGACTCCTCTTTTCA

	GGATACCAACACCACCTGAGTGTTAGGGAAAACCC

	GATTATTAAGAACCTCAAACCTGAGACCCTTATTG

	ATTCTTTCGAGCTGCTGTACTATTACGACGAATAC

	TTGGGTCATTGTATGTGGTATATTCCATTCTTTCT

	CATTCTCTTCATGTATTTTTCTGGGTGCTTTACGG

	CAAGCAAGGCGGAGAGCCTTATCCCAGGCCCCGCG

	CTCCTTCTTGTTGCCCCAAGCGGTCTCTATTATTG

	GTATCTGGTAACCGAGGGGCAAATATTTATACTTT

	TTATTTTCACCTTCTTTGCCATGCTCGCCTTGGTT

	TTGCACCAGAAGAGGAAGAGATTGTTCCTCGATAG

	CAACGGACTTTTTCTGTTCTCTTCCTTCGCGCTTA

	CCCTTCTCTTGGTAGCCTTGTGGGTCGCCTGGCTG

	TGGAATGACCCAGTTTTGCGGAAAAAGTATCCCGG

	TGTGATCTACGTGCCTGAACCTTGGGCATTCTACA

	CTCTTCATGTGTCCTCTAGACACGATTATAAGGAT

	GATGATGACAAATA

The CLN8 protein sequence is:

(SEQ ID NO: 78)

MNPASDGGTSESIFDLDYASWGIRSTLMVAGFVFYLGVFVVCHQLSSSLN

ATYRSLVAREKVFWDLAATRAVFGVQSTAAGLWALLGDPVLHADKARGQQ

NWCWFHITTATGFFCFENVAVHLSNLIFRTFDLFLVIHHLFAFLGFLGCL

VNLQAGHYLAMTTLLLEMSTPFTCVSWMLLKAGWSESLFWKLNQWLMIHM

FHCRMVLTYHMWWVCFWHWDGLVSSLYLPHLTLFLVGLALLTLIINPYWT

HKKTQQLLNPVDWNFAQPEAKSRPEGNGQLLRKKRPDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for CLN8 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

CLN8-DNA

	(SEQ ID NO: 79)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	TGAAATCGTATGGATCAAAGCTGTCCTCTTGGCCT

	GAGCCGGTTGCTGAGTGCGGGCCACAAGATCAGAT

	GAGCCAGTTTATCCATCTGGATGGTCCCAGCTAAT

	TCAGCAAGTGCAAGGTCTGCAAAATATCTCAAGCA

	CTGTTGTTAGGTTTTTCAATAGTGATGTTATCCCC

	AGGAGCAATTTAGGGAGGGTCAGAATCTTGTAATC

	TCCAGCTGCATGAATTCTAAACCATAATTTCTAAT

	CTTGTGGCTAATTTGTTAGTCCTACAAAGGCAGTC

	TAGTCTCCAGACAAGAAAAGGATTTGTTTCGGGAA

	ACCATAAACTAAGTTTCTTCCAAAGTTAGTTTGGC

	CTACACCCAGGAATGAACAAGGACAGTTTGGAGGT

	TAAAAGTAGGATGGAGTTGGTTAGGTCAGATCTCT

	CACTGTCTCAGTTATAATTTTGCAATGGTGGTTTC

	ACTGGTTTTAAGATGGAGTTTATTTTATTTTATTT

	TTATTTTTTTGAGACAGAGTTTCATTCTTGTTGCC

	CAGGCTAGAGTGCCATGGCGTGATCTCAGCTCACT

	GCAACCTCTGCCTCCTGGGTTCAAGCAATTCTGCT

	TCAGCCTCCTGAGTAGCTGAGATTACAGGTGCCCA

	CCACCACACCCGGCTAATTTTTTTCTCCCAAACTG

	CTGGGATTACAGGCTTGAGCCACCGCGCCCGCCAA

	GATGGGTTGTTGATTTTAAAACGATGTCACCCTGG

	CTCTCCTAGGTTCCTGTTCCCCTAACATCATGAAG

	GCTGCTGGCAGTTCTCTGGCCTCCAGGGAGGCCTC

	CACACACATTTACCCATGTTCTTAGACAATCCTAG

	GGACTCAAGGCTTCAACAAACCTAAATGAGCCAGC

	AGAAAAATAAACTTGAACATGAATGTCAACGTTTT

	CCCACATGTACTAAAGGGTTATAATAATTTTGAAA

	TGTTTATTTCAAAGAGCGTTGGTAATTTAAACGTC

	CTATTTAATCCCCAAAACAGTCCTGAGGGGGAGAT

	AAGGCAGTTATCTTCATAGTACAGGAAAGGAAAAA

	AGCGAGGGTCCAAGGCCGACTATACCCTCAGCTCC

	ATTAGCCCCCGAGGCCTCCCTGACAGGCGGGGCGG

	ACAATCCCAGTGCAGATGCTCTGTATCGATCGCAT

	GCTATCGGTTCTTTCAAGGAACGTGTATTGATCAT

	CAATTAAGTGGTGAGTACTCCTCTAGATGTCGATT

	CTAAAGAGGGAACAAAACACATTAGACAGAAGAAA

	CATAAGTAACTCGAATAACAACCTTAGAGAGAAGA

	AACATAAGTAACTCGAATAACAACCTTAGCAAACT

	GCGGAAACTCCTACAGACAAAAACTCAGGTGTGGG

	CGCAGAAGGGCCGGGGATGCGCTCGGTCAAGACTT

	GAAGGTCCGGGGCTTGGGCCCTGTGTCCTCACCGA

	GCCCCGGGGAGGCTCCGGCTCCGTCCACACCCGGA

	GCGCGACTCAGCCGCGGCCGGGGCTCTTCTGGAGA

	ACGGCGCGGTCAGGCTCTGCCGCAGCTTTCAGGTC

	CCCTCGGCCCCGTCCGGGTGCAGCGCAACCAGGCT

	GCCCGCGGAGCCCCACCGCCCCCATGCCGCCGCCC

	GCCATGGTTCAGCCCGGCCCCGCCCCTCCCGCCGG

	CCGTGGTTCAGCGCGTCCACCCCGCCGCCCCACAG

	CGCCCGCCGCGCCGCGCCCCGCCCCCCGCCGCGCC

	CCGCCCCCCGCCGCGCCCCGCCCCCCGCCGCGCCC

	CGCCCCCCGCCGCGCCCCGCCCCCCGCCGCGCCCC

	GCCCCCCGCCGCGCCCCGCCCCCCGCCGCGCCCCG

	CCCCCCGCCGCGCCCCGCCCCCCGCCGCGCCCCGC

	CCCCCGCCGCGCCCCGCCCCCCGCCGCGCCCCGCC

	CCCCGCCGCGCCCCGCCCCCCGCGCCGCGCCCCGC

	CCCCCGCCGCGCCCCTCCCGCGCCCGCCGCCCCGC

	CCCTCCCGCGCCCGCCGCCGTCGTAGCGCCTAGGC

	GGGGCCTCTCGTAGCGACGCCCCCAGGGGGGCAGA

	ACGCGTGCGCACGCGCGTGCGAACGCGCGTGCGCG

	GGCGGTGTTTGAGGCCGGC



	ATGAATCCCGCGAGTGATGGCGGCACTTCAGAGAG

	TATCTTTGACTTGGACTATGCATCATGGGGGATAC

	GCTCAACTCTTATGGTTGCAGGGTTCGTATTTTAT

	CTGGGGGTTTTTGTTGTCTGTCACCAACTTTCCAG

	CAGTCTCAACGCCACTTATAGATCCCTTGTGGCTA

	GGGAGAAAGTCTTTTGGGACTTGGCCGCAACCCGA

	GCCGTTTTTGGTGTCCAGTCCACGGCGGCTGGACT

	GTGGGCCCTCCTTGGTGATCCTGTTCTGCACGCTG

	ATAAAGCCAGAGGCCAACAGAACTGGTGTTGGTTC

	CATATAACTACGGCGACTGGCTTCTTTTGTTTTGA

	AAACGTAGCCGTACACCTGTCCAATTTGATCTTCA

	GAACATTCGACCTCTTTCTGGTAATCCACCATCTT

	TTCGCCTTTCTCGGCTTTCTTGGATGTCTTGTCAA

	TCTCCAAGCCGGTCACTACCTCGCAATGACTACTT

	TGTTGCTCGAAATGTCAACCCCATTTACTTGCGTA

	TCATGGATGCTGCTGAAAGCGGGATGGAGTGAATC

	ACTTTTTTGGAAGTTGAATCAGTGGTTGATGATTC

	ATATGTTTCACTGTCGAATGGTACTGACATATCAT

	ATGTGGTGGGTCTGTTTCTGGCACTGGGACGGGCT

	GGTATCTTCACTTTATCTCCCTCACCTGACCCTTT

	TCCTCGTAGGCTTGGCGCTGCTCACACTGATCATT

	AATCCTTACTGGACCCACAAGAAAACGCAGCAACT

	GCTGAACCCTGTGGATTGGAACTTCGCACAGCCCG

	AGGCCAAGTCACGGCCTGAAGGCAACGGTCAACTG

	TTGAGGAAGAAGAGACCGGACTATAAGGATGATGA

	CGACAAATA

The GM2A protein sequence is:

(SEQ ID NO: 80)

MQSLMQAPLLIALGLLLAAPAQAHLKKPSQLSSFSWDNCDEGKDPAVIRS

LTLEPDPIIVPGNVTLSVMGSTSVPLSSPLKVDLVLEKEVAGLWIKIPCT

DYIGSCTFEHFCDVLDMLIPTGEPCPEPLRTYGLPCHCPFKEGTYSLPKS

EFVVPDLELPSWLTTGNYRIESVLSSSGKRLGCIKIAASLKGIDYKDDDD

K

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for GM2A and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

GM2A-DNA

	(SEQ ID NO: 81)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	CCCAGATACAGAATCTTCTTGGGTCCAAATACCCC

	CTAGAGGTTTCCCATTGGCCACTTGGTGCTCACCT

	CATGTAAATGAAGTGGTGGCCCGCAATCAGTCTGA

	CTGGTTAATCAGAGGCCGAAGTGAAGTTACAAAGG

	TCATGCTCCTGTGCAAACATCTGATTGGTTGTGGA

	AAGCAACCAATCAGGCTGAAGTGAAGTTACAAAGT

	TGCACTTCTATGCAATTTTAACATATAAACTGAAA

	TGTAGCATAGAAATCGAAATGTACTTTCAATTTCC

	CATCTGCTGCACAGAAAAGGTGGGGGGTTGTAAAG

	AGAGTAGCCTCTGGTCCTCTTGTTACTTAGGTGTG

	GAAAGTTAGGGTTTTCCTTTCGATTTAGTTCTAGG

	AAGTCAGCGTGAAATGGCCTTAGGTTCCCTGCCTG

	CAGACTCTATTCTCCTACCTCAGTTGGGCTAGGTG

	AAAGGGAAGAGTTGATCAGAATGGGAGGATCATTA

	TGTAAAGTATGGAGCTTAGTTAATAATAGTGTACC

	AATGTCATGCCCTCAGATTTAAAAAAAATTTTTTA

	ATTTTTTATTCTTTTGCCACCTCCTTGTCGCTGGC

	AGTGCCCCCCAGTTATGCACAAGCACAAGCTCTGG

	GTCTGCAGTCTGAACCCCCGGCCAGGCCACGGGTG

	TCTTATCAACAATAGAGGAAACGAACAGAACTTTG

	TAGTATTTTTGCAACTTTTCTGTAAACCAAAATAA

	AAAGTTTACTTTAAAAAAGATGATCAGGTGAGTTT

	TAAATTCTACACATGCATTCATGTACTCAACAGAT

	TGTCTATTATGCATCAGGCACTGGGTATAAATGAG

	TGCCTGCATCATGATGCCACCCCTTGTGTAGTTTA

	CAATCCTGTGGTAAATTGGCGGTATTGGTAACAAC

	ATCCCTGTTTTTTCTAAAAGCAAATCTCCAACTAG

	TATATTAACCTGACAAAATCTTGGCTGATGGGGTG

	TCTAAAAAGAAGTCTTGCTCTATTCCTTCCTAGCT

	GTGCGACCTTGGCAGAGCTAACCTCACTGTGTATC

	AGTTTCCTCAGTGCTAAAATTGGGGAAGAAAAAAA

	AAAAGCATTCCCTTGTGCGTCCTTAGCATGCTTTC

	AAGCAGTTACTAAATGTTAGTTTTTATAACGGTTA

	TAATTATTAAAAAGATACGGATTCGTGAAGTCAAG

	AGATCAAGACCGTCCTGGACAACACGGTGAAACCC

	CGTTTCTACTAAAAACACAAAAATTAGCTGGGCGT

	GTGGCGCGTGCCTGTAGTCCCAGCTAGTCGAGAGG

	CTGAGGCGGAAGAATCGCTTGAACCTGGGAGGCAG

	AGGTTGCAGTGAGCCGAGATCGCGCCACTGCACTC

	CAGCTCGGGCGACAGAGCGAGAGTCCGTCTCAAAG

	CAAAAAAAAAAAAAAAAAAGACACACATATGGGGA

	AGCGTAACATGGCAGAGAGTCTGACATTTTTGTCC

	GGAAGGACTTGGGGATGAAAACACTGAATCCGCGG

	TTTCCCAAAGGTCCTTCCAAAGGTGACGGCCCCGG

	CACGAGTTCACAAACCACAGCTCCGCCCCATCCCC

	TGCCCGGCCCCGCCCTTCCCAGGAGCTCTGCTCCG

	CCCCGCCCCCGACTCCGCTCGGCCCCTCCTCGCGC

	CACGCCCCCGCCCCTCCCGCGCGCGCAGCTCCGCC

	CCGCCCTCGTGATCCCGCCCCAACCTCGTGCTAAG

	CTCCCTTCCGTTCCAGCCGCCTTCAGCCCCGCCGC

	GCTCAGTTCCGCCCCTTTCTCTTCCAACTCCGCCT

	ACGCCCCAGAGAGGGAAGGCCTTGACACCTACGCT

	AGAGAGACAGGCTCCAATGAAAAGAGCGAGGGGGC

	GGAGCCGGGAGGAATCGGTCCAATTCTCGGCCGGG

	AAGTCTCTGGCCGGGACGGGGCAGGGCGAACCTGC

	CAGTGACTGGACTCAGCTTCTTTGCGTAACCAATA

	CTGGAAGGCATTTAAAGGCACCTCTGCCGCCACAG

	ACCT





	ATGCAGTCTCTCATGCAAGCTCCTCTCTTGATCGC

	CCTTGGTCTGCTTTTGGCTGCCCCAGCACAAGCTC

	ACCTGAAAAAACCTTCCCAACTTAGCTCCTTTTCC

	TGGGATAATTGCGATGAAGGAAAGGACCCGGCTGT

	AATCCGAAGTTTGACACTCGAACCTGACCCCATCA

	TCGTCCCGGGCAATGTCACGCTTTCAGTAATGGGT

	AGCACGAGCGTTCCGCTCTCTAGCCCATTGAAAGT

	AGACCTCGTGTTGGAAAAAGAAGTAGCCGGGTTGT

	GGATAAAAATTCCATGCACGGATTATATTGGCTCA

	TGTACCTTTGAACACTTTTGCGATGTTTTGGACAT

	GTTGATCCCCACAGGCGAGCCATGTCCAGAGCCCC

	TTAGGACGTATGGGCTTCCGTGTCACTGCCCATTC

	AAGGAAGGTACATATAGCCTGCCGAAGTCAGAATT

	TGTCGTCCCTGATCTCGAATTGCCTAGTTGGCTGA

	CAACCGGCAACTATCGAATTGAATCTGTACTTTCT

	TCAAGCGGTAAACGGCTCGGCTGTATCAAAATAGC

	CGCCTCCCTGAAGGGGATAGACTACAAGGACGACG

	ACGATAAATA

The HYAL1 protein sequence is:

(SEQ ID NO: 82)

MAAHLLPICALFLTLLDMAQGFRGPLLPNRPFTTVWNANTQWCLERHGVD

VDVSVFDVVANPGQTFRGPDMTIFYSSQLGTYPYYTPTGEPVFGGLPQNA

SLIAHLARTFQDILAAIPAPDFSGLAVIDWEAWRPRWAFNWDTKDIYRQR

SRALVQAQHPDWPAPQVEAVAQDQFQGAARAWMAGTLQLGRALRPRGLWG

FYGFPDCYNYDFLSPNYTGQCPSGIRAQNDQLGWLWGQSRALYPSIYMPA

VLEGTGKSQMYVQHRVAEAFRVAVAAGDPNLPVLPYVQIFYDTTNHFLPL

DELEHSLGESAAQGAAGVVLWVSWENTRTKESCQAIKEYMDTTLGPFILN

VTSGALLCSQALCSGHGRCVRRTSHPKALLLLNPASFSIQLTPGGGPLSL

RGALSLEDQAQMAVEFKCRCYPGWQAPWCERKSMWDYKDDDDK

The proprietary viral enhancer sequences, native promoter sequences, coding sequence for HYAL1 and the 5′ and 3′ UTR sequences are listed below using the same nomenclature.

HYAL1-DNA

	(SEQ ID NO: 83)
	GCGCAGCACCATGGCCTGAAATAACCTCTGAAAGA

	GGAACTTGGTTAGGTACCTTCTGAGGCGGAAAGAA

	CCAGCGTTACATAACTTACGGTAAATGGCCCGCCT

	GGCTGACCGCCCAACGACCCCCGCCCATTGACGTC

	AATAATGACGTATGTTCCCATAGTAACGCCAATAG

	GGACTTTCCATTGACGTCAATGGGTGGAGTATTTA

	CGGTAAACTGCCCACTTGGCAGTACATCAAGTGTA

	TCATATGCCAAGTACGCCCCCTATTGACGTCAATG

	ACGGTAAATGGCCCGCCTGGCATTATGCCCAGTAC

	ATGACCTTATGGGACTTTCCTACTTGGCAGTACAT

	CTACGTATTAGTCATCGCTATTACCATG

	GGACTTGCTGGTGACACAGCCTCAGGAAGTCCTGA

	GGACATGTGCCCAGGGCGGTTGGGGCACAGCTTGG

	TTTTATAGATTTTAGGGAGACATGAGACATTAATC

	AATATGTAAGAAGTACATTAGTTCCAGAAAGAAAG

	GTGGAGACTGCTCAAATCAAGGCTCCCAGGCTCAA

	AGCACTGGGGGCTTCCAGGTCACAGATAGGTGAGA

	GACAGATGGTTGCATTCTTTTGAGTTTCTGGTAAG

	TCTTTCCAAAGGAGGCAATCAGAATATGCATCTAT

	CTCTGTGAGCAAAAGGATGACTTGAATAGAATGGG

	AGGCAGATTTGTCCTGAGCAGTTCCCAGCTTGAAG

	AGGCCCAAGATACTTTCCTTTCACATTTACCCCAT

	TTTCTTTTTCAAAATCTTTTGGAGAAAGCATTTTG

	CAAGAAAATGAGTATCTGGTCTCAGGTTTCATCTG

	ATCTCTCATTGCTAGATAAGTAGGTCCGGAAAGCT

	CATTTTTAGCAGGTTGTAAAGTCTCATGCAGTGTG

	AAGAGAAAATAGGGAGAAGGAAGGAAGAGAAAAAA

	AAAACAGCAAAAGAACAATCCCAGCCCTGGCGGGG

	TGGCTCATGCCTGTAATCCCAACAATTTGGGAGGC

	TGAGGCGGGTGGATCACCTGCGGTTGGGAGTTCGA

	GAACAGCCTGACCAACATGGAGAAACTCTGTCTCT

	ACTGAAAATACAAAAAACTAGCCAGGCATGGTGGC

	TCATGCCTGTAATCCCAGCTACTCAGGAGGCTGAG

	ACAGGAGAATCACTTGAACCCAGGAGGCGGAGGTT

	GCAGCAACCTGACATTGCGCCATTGCACTCCAGCC

	TGGGCAACAAGAGTGAAACTCCATCTCAAAAAAAA

	AAAAAAAAAAAATCCTGGGAAAATATAGGCCACAT

	TACTCTGAAGTCCATACATTGGTAGGCAGGTATGA

	AAGTGGCTTATGTATGTACATAAACAGGTTACTGT

	TACTTTCTTCTGAAGTGTAAGTTGTCTGACTTTAG

	TTGACACGCTTTTAAGAAACCACAGCTAGGGCCGG

	GCGCAGTGACTAACGCCTGTAATCCCAGCACTTCG

	AAGAGGCCGAGGCGGCCGGATCACAAGGTCAGGGT

	TCGAGACCAGCCTGGTCAATATGGTGAAACCCTGT

	CTCTACTAAAAATACAAAAATTAGCCAGGCATGGT

	AGCAAGCGCCTGTAGTCCCAGCTACTCGGGAGGCT

	GAGGCAGGAGAATTGCTTGAACCTGGGAGGCATAA

	GTTGCAGTGAGCTGAGATCGCGCCACTGCACTCCA

	GCCTGGGTGACAGAGCGAGACTCCATCTCAAAAAA

	AAAAAAAAAAGAAACCACAGTGGCCGATTGCAGTG

	GCTCCCCCTCCCCCTCCCCCTCCCCTCCCCCTCCC

	CCTCCCCCTCTCCCTCCACGGTCTCCCTCTGATGC

	CGAGCGGAAGCTGGACTGTACTGCTGCCATCTCGG

	CTCACTGCAACCTCCCTGCCTGATTCTCCTGCCTC

	AGCCTGCCGAGTGCCTGCGATTGCAGGCACGCGCC

	GCCACGCCTGACTGGTTTTCGTATTTTTTGGGTGG

	AGACGGGGTTTCGCTGTGTTGGCCGGGCTGGTCTC

	CAGCTCCTAACCGTGAGTGATCTGCCAGCCTCGGC

	CTCCCGAGGTGCTGGGATTGCAGACGGAGTCTCTC

	AATGGTGCCCAGGCTGGAGTGCAGTGGCGTGATCT

	CGGCTCGCTACAACATCCACCTCCCAGCAGCCTGC

	CTTGGCCTCCCAAAGTGCCGAGATTGCAGCCTCTG

	CCCGGCCGCCACCCCGTCTGGGAAGTGAGGAGCGT

	CTCTGCCTGGCCGCCCATCGTCTGGGATGTGAGGA

	GCCCCTCTGCCTGGCTGCCCAGTCTGGAAAGTGAG

	GAGCGTCTCTGCCCAGCCGCCATCCCATCTAGGAA

	GTGAGCAGCGCCTCTTCCCGGCCGCCATCCCATCT

	AGGAAGTGCGGAGCCTCTCTGCCCGGCCACCCATC

	GTCT





	ATGGCAGCGCATCTCCTCCCGATATGTGCACTTTT

	TCTGACTCTTCTTGATATGGCACAAGGATTTCGGG

	GTCCGCTCTTGCCTAATCGACCTTTTACAACAGTA

	TGGAATGCTAACACACAGTGGTGCCTTGAAAGGCA

	CGGTGTCGACGTTGACGTCTCCGTCTTCGACGTAG

	TAGCCAATCCAGGCCAAACCTTCAGGGGGCCAGAT

	CATGACTATCTTCTATAGTTCTCAACTGGGGACTA

	TCCCTACTACACCCCCACGGGTGAACCTGTATTCG

	GCGGGTTGCCACAGAATGCTAGCCTTATCGCGCAC

	CTGGCCAGGACGTTTCAAGACATACTCGCCGCCAT

	TCCAGCACCCGACTTCTCTGGTCTGGCTGTGATCG

	ATTGGGAGGCCTGGCGACCTAGGTGGGCTTTCAAC

	TGGGATACCAAGGACATCTATAGGCAGCGGAGCCG

	AGCATTGGTGCAAGCCCAGCATCCGGATTGGCCAG

	CGCCTCAGGTGGAAGCGGTAGCGCAGGATCAATTC

	CAGGGCGCCGCGAGAGCTTGGATGGCCGGGACATT

	GCAACTTGGGAGAGCCTTGAGGCCTCGCGGCCTGT

	GGGGTTTTTATGGATTCCCAGATTGCTACAACTAT

	GATTTTCTCTCTCCAAATTACACAGGCCAATGTCC

	AAGCGGCATACGCGCACAGAACGATCAGCTTGGCT

	GGCTCTGGGGACAATCTCGAGCCTTGTACCCTAGC

	ATCTATATGCCAGCTGTCTTGGAGGGCACCGGAAA

	ATCTCAGATGTACGTGCAACATAGGGTTGCAGAGG

	CCTTCCGGGTTGCCGTCGCAGCCGGAGATCCGAAT

	CTGCCTGTTCTTCCCTATGTACAGATCTTTTATGA

	TACCACAAATCACTTTTTGCCGTTGGACGAGCTTG

	AACATAGTTTGGGTGAAAGTGCGGCCCAAGGTGCG

	GCGGGAGTAGTTCTTTGGGTTTCCTGGGAAAATAC

	ACGGACTAAAGAGTCATGTCAGGCCATAAAGGAGT

	ACATGGATACCACACTGGGCCCTTTCATTTTGAAT

	GTTACGAGTGGCGCGTTGCTTTGTAGTCAGGCACT

	GTGCAGCGGACACGGCAGGTGTGTGCGGCGGACGT

	CACATCCAAAGGCCCTGCTGCTGCTGAACCCGGCG

	TCATTTTCAATTCAACTTACTCCCGGTGGGGGGCC

	CCTCTCACTTAGGGGCGCGCTCTCCCTGGAAGACC

	AGGCCCAGATGGCAGTAGAGTTTAAGTGCAGATGT

	TACCCAGGGTGGCAGGCGCCTTGGTGCGAGCGCAA

	ATCCATGTGGGATTACAAAGATGATGATGATAAGT

	A

In certain embodiment, any of the GOI described herein are codon optimized for manufacturing, optimal expression in a host cell, such as in a human cell. Further Codon optimization of GOI can be done based on codon optimization table publically available, such as the Codon and Codon Pair Usage Tables (CoCoPUTs) maintained at the George Washington University (GWU). The CoCoPUTs is derived from all available GenBank and RefSeq data source.
In certain embodiment, the GOI's are codon optimized for expression in neuronal tissues or specific cell types in neuronal tissues, such as neurons, depending on the target cell type the GOI's are to be expressed. This can be done using TissueCoCoPUTs, which are human tissue-specific codon and codon pair usage tables derived from human genomic codon usage information and tissue-specific transcriptomic data. The TissueCoCoPUTs maintained by the GWU website represent 52 human tissues. Transcriptomic data are derived from the Genotype Tissue Expression (GTEx) Portal.

3. Delivery Methods

In certain embodiments, the viral vectors (such as AAV) of the invention are delivered to neuronal target cells by intravenous injection or direct intraparenchymal injection or direct intracranial injection. Such delivery means effectively bypass the BBB, and are common means for the administration of gene therapeutics to the brain. In certain embodiments, the neuronal target cells intended to receive gene therapy through intraparenchymal injection are clustered in a focal location, and are this particularly suitable for focal delivery of the viral vector.
In certain embodiments, the viral vector (such as AAV) of the invention is delivered locally or compartmentally, such as direct intraocular injection, or injection in the cochlea.
In certain embodiments, the viral vectors of the invention are delivered to neuronal target cells by intravenous injection.
In certain embodiments, the neuronal target cells intended to receive gene therapy through intravenous injection are globally located in the CNS, or in multiple CNS regions. Such global delivery across the BBB is achieved by using selected AAV serotypes (such as AAV9) that have been shown to deliver genes across the BBB after intravenous injection.
In certain embodiments, the viral vector (such as AAV) of the invention is delivered to achieve widespread delivery for diseases of extensive pathology including glioblastoma multiforme (GBM), lysosomal storage diseases (LSDs), Alzheimer's disease, Parkinson's disease and Canavan's disease. In certain embodiments, the viral vectors are delivered intracerebroventricularly, intrathecally, intranasally, or systemicaly (e.g., intravenous and intra-arterial injections).
The systemic delivery route for gene delivery vehicles is very promising, and has the potential to be a conduit to the entire CNS (every cell in the brain is a maximum distance of 40 μm from an endothelial cell), provided that the issue of BBB blockage and the filtration systems of the spleen and liver are adequately dealt with.
In certain embodiments, the viral vectors (such as AAV) of the invention are delivered to neuronal target cells by intravascular, intrathecal, and/or cerebrospinal fluid (CSF) administration. Many AAV capsids, such as AAV9, AAV2.5, rAAVrh.39, rAAVrh.43, rAAV7, rAAVrh.8 and rAAVrh.10, have remarkable CNS tropism after vascular (Foust et al., Nat Biotechnol. 27:59-65, 2009; Gray et al., Gene Ther. 20:450-459, 2013; Zhang et al., Mol Ther. 19:1440-1448, 2011; Bevan et al., Mol Ther. 19:1971-1980, 2011; Yang et al., Mol Ther. 22:1299-1309, 2014; Duque et al., Mol Ther. 17:1187-1196, 2009), and CSF administration (Samaranch et al., Hum Gene Ther. 24:526-532, 2013; Gray, supra; Samaranch et al., Hum Gene Ther. 23:382-389, 2012).
In certain embodiments, the viral vectors (such as AAV) of the invention are delivered to neuronal target cells by direct intracranial injection. A large range of AAV capsids, including pseudotypes rAAV2/1, rAAV2/2, and rAAV2/5, hu.32, hu.37, pi.2, hu.11, rh.8, hu.48R3, bb2, cy5, rh20, rh39, rh43, AAV7, AAV8, and AAV9, have been demonstrated for their CNS gene transfer properties by direct intracranial injection (Burger et al., Mol Ther. 10:302-317, 2004; Cearley et al., Mol Ther. 16:1710-1718, 2008; Cearley and Wolfe, Mol Ther. 13:528-537, 2006; Lawlor et al., Mol Ther. 17:1692-1702, 2009). Vectors based on distinct AAV serotypes or pseudotypes can be chosen for specific applications in the nervous system, depending on their preferential or global tropism within the different regions of the brain and target neuronal tissues.
In certain embodiments, the viral vector is AAV9. The vast majority (>90%) of cells in the CNS transduced by intravenously injected AAV9 using strong promoters like CBA are astrocytes and neurons. The distribution of vector is widespread with transduced cells detected in all areas of the brain from the olfactory bulb to cerebellum.
In certain embodiments, delivery of the viral vectors of the invention across the BBB is further enhanced by a variety of techniques including: intra-nasal administration and/or intracranial NSC transplantation that bypasses the BBB; transiently permeating the BBB using osmotic manipulation; magnetic resonance imaging guided focused ultrasound (MRIgFUS) to selectively open the BBB and allow therapeutics to pass into the parenchyma; convection enhanced delivery (Debinski and Tatter, Curr Opin Mol Ther. 12:647-653, 2010); introduction into a fluid space, e.g., vasculature, brain ventricles or intrathecal space in the spinal cord, such that cells lining the space produce the deficient protein (Chen et al., Nat Med. 15:1215-1218, 2009); injection into regions of the brain that serve as a nexus of interconnections with retrograde transport of vectors, such as the thalamus, e.g., for AAV (McCurdy et al., Sci Transl Med. 6:231ra48, 2014); and/or by taking advantage of the “bystander effect” whereby proteins released from the producer cells can be taken up by the deficient cells (McCurdy; Chen, Supra).
In certain embodiments, the viral vector (such as AAV) of the invention comprises a targeting peptide that bind selectively to the brain endothelial cells or a brain endothelial cell receptor (such as the low-density lipoprotein (LDL) and other related receptors that can be targeted by Apolipoprotein E-derived peptides) to target the viral vector to the brain vasculature.

4. Viral Production

Another aspect of the invention provides a method of producing the rAAV vector of the invention, comprising introducing the rAAV vector of the invention into a packaging cell line that constitutively or inducibly provides rep/cap proteins in trans.
In certain embodiments, the packaging cell line is a HEK293 cell line, a HeLa cell, or an A549 cell.
In general, the subject rAAV vector can be produced using any of many art-recognized approach. In certain embodiments, the rAAV vector is produced based on the helper-virus-free transient transfection method with all cis and trans components (e.g., vector plasmid and packaging plasmids, along with helper genes isolated from adenovirus) in host cells such as 293 cells. In certain embodiments, the rAAV vector is produced using recombinant herpes simplex virus (rHSV)-based AAV production system, which utilizes rHSV vectors to bring the AAV vector and the Rep and Cap genes into the cells. In certain embodiments, the rAAV vector is produced based on baculovirus system which requires simultaneous infection of insect cells with several baculovirus vectors to deliver the rAAV vector cassette and the Rep and Cap genes. In certain embodiments, the rAAV vector is produced based on the AAV producer cell lines derived from HeLa or A549, which stably harbored AAV Rep/cap genes. The AAV vector cassette can either be stably integrated in the host genome or be introduced by an adenovirus that contained the cassette.

Claims

1. A recombinant adeno-associated viral (rAAV) vector, comprising:

(1) a polynucleotide comprising a gene of interest (GOI), under the transcriptional control of the native/endogenous promoter of said GOI;

(2) a heterologous enhancer that enhances transcription of the GOI from the native/endogenous promoter; and,

(3) a pair of inverted terminal repeat (ITR) flanking the polynucleotide.

2. The rAAV vector of claim 1, wherein GOI is a neuronal gene, or is a gene expressed in neuronal tissues (CNS and/or PNS).

3. The rAAV vector of claim 1 or 2, wherein the GOI is defective (e.g., loss-of-function) in a neuronal disease or condition.

4. The rAAV vector of claim 3, wherein the neuronal disease or condition is a CNS disease or condition.

5. The rAAV vector of claim 4, wherein the CNS disease or condition is: Ceroid-Lipofuscinosis, Fucosidosis, Giant Axonal Neuropathy, Globoid cell leukodystrophy, Mucolipidiosis Type IV, Neuronal Ceroid Lipofusinoses, Niemann-Pick Disease, Sandhoff Disease, Sanfilippo syndrome, Tay-Sachs Disease, Hunter Syndrome, Sialidosis, Multiple Sulfatase Deficiency, Childhood Epilepsy, CMT Peripheral Neuropathy, Neuronal Ceroid Lipofusinoses, Aspartylglycosaminuria, Mucopolysaccharidoses, Parkinsons Disease/Symptoms, GM1-Gangliosidosis, Charcot-Marie-Tooth Type 1A, Retts Syndrome, Dannon Disease, Mucopolysaccharidoses (due to GNS, HGSNAT, NAGLU, or ARSB), Sly Syndrome, Biotin basal ganglia disease, Pelizaeus-Merzbacher disease, Neuronal Ceroid Lipofusinoses, Canavan Disease, Beta-Mannosidosis, Wolman Disease, Cystinosis, Alzheimer's Disease/Symptom, Salla Disease, Jansky-Bielschowsky disease, Neuronal ceroid lipofuscinoses, or GM2 gangliosidosis.

6. The rAAV vector of any one of claims 1-5, wherein the GOI is: CLN3, FUCA1, GAN, GALC, MCOLN1, PPT1, SMPD1, HEXB, SGSH, HEXA, IDS, NEU1, SUMF1, GAT1/SLCA1, FIG. 4 , CLNS, AGA, IDUA, GDNF, GLB1, PMP22/MFN2, MECP2, LAMP2, NAGLU, ARSB, GUSB, SLC19A3, PLP1, TPP1/CLN2, ACY2/ASPA, MANBA, LIPA, CTNS, GNS, HGSNAT, NEUROD1, SLC17A5, CLN6, CLN8, or GM2A.

7. The rAAV vector of any one of claims 1-6, wherein the rAAV has a tropism for neuronal cells, or for a cell in neuronal tissue.

8. The rAAV vector of claim 7, wherein the rAAV is AAV9, AAV2.5, rAAVrh.39, rAAVrh.43, rAAV7, rAAVrh.8 or rAAVrh.10, preferably AAV9.

9. The rAAV vector of claim 7, wherein the rAAV comprises AAV9, AAV2.5, rAAVrh.39, rAAVrh.43, rAAV7, rAAVrh.8 and rAAVrh.10 capsids, preferably AAV9 capsids.

10. The rAAV vector of any one of claims 1-9, wherein the polynucleotide further comprises a 5′-UTR coding region, and a 3′-UTR coding region.

11. The rAAV vector of any one of claims 1-10, wherein the heterologous enhancer is a viral enhancer.

12. The rAAV vector of claim 11, wherein the viral enhancer is CMV enhancer.

13. The rAAV vector of claim 11, wherein the viral enhancer is SV-40 enhancer.

14. The rAAV vector of claim 11, wherein the heterologous enhancer comprises a polynucleotide of SEQ ID NO: 1.

15. A pharmaceutical composition comprising the rAAV vector of any one of claims 1-14.

16. The pharmaceutical composition of claim 15, which is formulated for intravenous administration (e.g., to the blood) or intrathecal administration (e.g., to spinal fluid).

17. A cell infected with the rAAV vector of any one of claims 1-14.

18. A recombinant AAV virus comprising the rAAV vector of any one of claims 1-14, wherein the serotype or pseudotype of the recombinant AAV virus is for preferential infection of a neuronal cell or a cell in neuronal tissue.

19. A method of treating a (genetic) disease or disorder in a subject, the method comprising administering the recombinant AAV virus of claim 18 into the subject.

20. The method of claim 19, wherein the genetic disease or disorder is a neuronal disease or disorder, and/or wherein the recombinant AAV virus preferentially infects a neuronal cell or a cell in neuronal tissue of the subject, thereby treating the neuronal disease or disorder.

21. The method of claim 19 or 20, wherein the rAAV virus is administered to the subject intravenously or intrathecally.

22. A method of producing the rAAV vector of any one of claims 1-14, comprising introducing the rAAV vector of any one of claims 1-14 into a packaging cell line that constitutively or inducibly provides rep/cap proteins in trans.

23. The method of claim 22, wherein said rep/cap proteins comprise one or more AAV9 capsid proteins.