SG11201909203WA - Tissue selective transgene expression - Google Patents

Tissue selective transgene expression

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Publication number
SG11201909203WA
SG11201909203WA SG11201909203WA SG11201909203WA SG 11201909203W A SG11201909203W A SG 11201909203WA SG 11201909203W A SG11201909203W A SG 11201909203WA SG 11201909203W A SG11201909203W A SG 11201909203WA
Authority
SG
Singapore
Prior art keywords
california
suite
international
south san
transgene
Prior art date
Application number
Inventor
Stephanie Tagliatela
Andrew Young
Szu-Ying Chen
Kartik Ramamoorthi
David Oberkofler
Original Assignee
Encoded Therapeutics Inc
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Publication date
Application filed by Encoded Therapeutics Inc filed Critical Encoded Therapeutics Inc
Publication of SG11201909203WA publication Critical patent/SG11201909203WA/en

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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0312—Animal model for Alzheimer's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

Abstract

1 N co 00 O O (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 11 October 2018 (11.10.2018) WIPO I PCT omit °nolo mol mom!! m Immo oimIE (10) International Publication Number WO 2018/187363 Al (51) International Patent Classification: C12N 15/63 (2006.01) C12Q 1/68 (2018.01) AO1K 67/027 (2006.01) (21) International Application Number: PCT/US2018/025940 (22) International Filing Date: 03 April 2018 (03.04.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/480,998 03 April 2017 (03.04.2017) US (71) Applicant: ENCODED THERAPEUTICS, INC. [US/US]; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). (72) Inventors: TAGLIATELA, Stephanie; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). YOUNG, Andrew; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). CHEN, Szu-Ying; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). RAMAMOORTHI, Kartik; 343 Oyster Point Blvd., Suite 100, South San Fran- cisco, California 94080 (US). OBERKOFLER, David; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). (74) Agent: YIN, Eva F. et al.; WILSON SONSINI GOODRICH & ROSATI, 650 Page Mill Road, Palo Alto, California 94304-1050 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, (54) Title: TISSUE SELECTIVE TRANSGENE EXPRESSION FIG. 4B S EQ D NO: 8 SEQ ID NO: 1 EFla (57) : Provided herein are compositions and methods for selective expression of a transgene. Compositions and methods for selective expression of a transgene comprise one or more human regulatory elements, which, when operably linked to a transgene, can facilitate selective expression of a transgene (e.g., cell-type selective expression) in a target cell as compared to at least one or more non-target cells. [Continued on next page] WO 2018/187363 Al MIDEDIMOMOIDEIREEMOMOHOHIIMIMMOVOIMIE GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art. 21(3)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) with sequence listing part of description (Rule 5.2(a))
SG11201909203W 2017-04-03 2018-04-03 Tissue selective transgene expression SG11201909203WA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762480998P 2017-04-03 2017-04-03
PCT/US2018/025940 WO2018187363A1 (en) 2017-04-03 2018-04-03 Tissue selective transgene expression

Publications (1)

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SG11201909203WA true SG11201909203WA (en) 2019-11-28

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Country Status (16)

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US (6) US10519465B2 (en)
EP (1) EP3607073A4 (en)
JP (1) JP7246359B2 (en)
KR (1) KR102604159B1 (en)
CN (2) CN110730823B (en)
AU (1) AU2018250161B2 (en)
BR (1) BR112019020777A2 (en)
CA (1) CA3058189A1 (en)
CL (2) CL2019002805A1 (en)
CO (1) CO2019011450A2 (en)
EA (1) EA201992358A1 (en)
IL (1) IL269767A (en)
MX (4) MX2019011772A (en)
SG (1) SG11201909203WA (en)
TW (3) TW202315946A (en)
WO (1) WO2018187363A1 (en)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2016054615A2 (en) 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2017060731A1 (en) 2015-10-09 2017-04-13 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
TW202315946A (en) 2017-04-03 2023-04-16 美商編碼製藥公司 Tissue selective transgene expression
CA3073515A1 (en) 2017-08-25 2019-02-28 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
JP7472036B2 (en) * 2018-04-09 2024-04-22 アレン インスティテュート Rescue of voltage-gated sodium channels in inhibitory neurons
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2020163102A1 (en) * 2019-02-05 2020-08-13 The Broad Institute, Inc. Interneuron-specific therapeutics for normalizing neuronal cell excitability and treating dravet syndrome
WO2020176426A1 (en) * 2019-02-25 2020-09-03 University Of Massachusetts Dna-binding domain transactivators and uses thereof
CA3134501A1 (en) * 2019-03-22 2020-10-01 Encoded Therapeutics, Inc. Multiplexing regulatory elements to identify cell-type specific regulatory elements
EP3976798A4 (en) * 2019-05-29 2023-07-12 Encoded Therapeutics, Inc. Compositions and methods for selective gene regulation
US20230287410A1 (en) 2020-05-11 2023-09-14 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases
US20230265453A1 (en) * 2020-07-08 2023-08-24 Baylor College Of Medicine Gene therapy for stxbp1 encephalopathy
KR20230037586A (en) * 2020-07-09 2023-03-16 가부시키가이샤 모달리스 Methods for treating Alzheimer's disease targeting the MAPT gene
WO2023073071A1 (en) * 2021-10-28 2023-05-04 UCB Biopharma SRL Nucleic acid constructs, viral vectors and viral particles

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7405A (en) * 1850-05-28 John weidman
CA2187626C (en) 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
NZ333334A (en) 1997-04-17 2001-06-29 Frank L Sorgi Delivery system for gene therapy to the brain
CA2205076A1 (en) 1997-05-14 1998-11-14 Jim Hu Episomal expression cassettes for gene therapy
ATE466952T1 (en) 1998-03-02 2010-05-15 Massachusetts Inst Technology POLY ZINC FINGER PROTEINS WITH IMPROVED LINKERS
US6303370B1 (en) 1998-03-24 2001-10-16 Mayo Foundation For Medical Education And Research Tissue-specific regulatory elements
CA2246005A1 (en) 1998-10-01 2000-04-01 Hsc Research And Development Limited Partnership Hybrid genes for gene therapy in erythroid cells
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6649371B1 (en) 1999-06-11 2003-11-18 Neurosearch A/S Potassium channel KCNQ5 and sequences encoding the same
CA2394229C (en) 1999-11-26 2010-04-13 Mcgill University Loci for idiopathic generalized epilepsy, mutations thereof and method using same to assess, diagnose, prognose or treat epilepsy
DE60023936T2 (en) 1999-12-06 2006-05-24 Sangamo Biosciences Inc., Richmond METHODS OF USING RANDOMIZED ZINCFINGER PROTEIN LIBRARIES FOR IDENTIFYING GENERAL FUNCTIONS
DK1287134T3 (en) 2000-06-07 2007-12-03 Ortho Mcneil Pharm Inc The human voltage-gated sodium channel 1A subunit and methods for its use
AUPR492201A0 (en) 2001-05-10 2001-06-07 Bionomics Limited Novel mutation
WO2003004660A1 (en) 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
US6998118B2 (en) 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
WO2003072751A2 (en) 2002-02-25 2003-09-04 Vanderbilt University Expression system for human brain-specific voltage-gated sodium channel, type 1
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
ES2334231T3 (en) 2003-01-28 2010-03-08 F. Hoffmann-La Roche Ag USE OF REGULATORY SEQUENCES FOR SPECIFIC TRANSITIONAL EXPRESSION IN CERTAIN NEURONAL CELLS.
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
US7094600B2 (en) 2003-06-26 2006-08-22 The Research Foundation Of State University Of New York Screen for sodium channel modulators
WO2005100577A1 (en) 2004-04-14 2005-10-27 Agency For Science, Technology And Research Method for gene delivery to neuronal cells
TWI293307B (en) 2004-09-30 2008-02-11 Ind Tech Res Inst A liver-specific chimeric regulatory sequence and use thereof
US20070161031A1 (en) 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
EP2021499A4 (en) 2005-12-16 2010-02-17 Univ Leland Stanford Junior Functional arrays for high throughput characterization of gene expression regulatory elements
US9192630B2 (en) 2006-01-20 2015-11-24 The Regents Of The University Of California Transplantation of neural cells
WO2008073303A2 (en) 2006-12-07 2008-06-19 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2245046B1 (en) 2008-01-22 2019-09-04 Chromocell Corporation Novel cell lines expressing nav and methods using them
US20110165129A1 (en) 2008-05-06 2011-07-07 Arnold Kriegstein Ameliorating Nervous Systems Disorders
EP2321417B1 (en) * 2008-08-20 2015-07-15 Brainco Biopharma, S.L. Stxbp1 as psychiatric biomarker in murine model system and its uses
WO2010037143A1 (en) 2008-09-29 2010-04-01 The University Of Montana Vectors and methods of treating brain seizures
US8815779B2 (en) 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
US20110135611A1 (en) 2009-12-03 2011-06-09 The J. David Gladstone Institutes Methods for treating apolipoprotein e4-associated disorders
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP3567106A1 (en) 2010-04-23 2019-11-13 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2566972B1 (en) 2010-05-03 2020-01-15 Sangamo Therapeutics, Inc. Compositions for linking zinc finger modules
PT2585596T (en) 2010-06-23 2021-03-23 Curna Inc Treatment of sodium channel, voltage-gated, alpha subunit (scna) related diseases by inhibition of natural antisense transcript to scna
CN103189507A (en) 2010-10-27 2013-07-03 学校法人自治医科大学 Adeno-associated virus virions for transferring genes into neural cells
EP2635686B1 (en) 2010-11-05 2018-03-28 The Regents of The University of California Neuronal specific targeting of caveolin expression to restore synaptic signaling and improve cognitive function in the neurodegenerative brain and motor function in spinal cord
EP2638163B1 (en) 2010-11-12 2017-05-17 The General Hospital Corporation Polycomb-associated non-coding rnas
EP2655621B1 (en) * 2010-12-20 2018-05-23 The General Hospital Corporation Polycomb-associated non-coding rnas
WO2012112578A2 (en) 2011-02-14 2012-08-23 The Children's Hospital Of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
KR102057540B1 (en) 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 Aav vector compositions and methods for gene transfer to cells, organs and tissues
AU2013201287B2 (en) 2012-03-06 2015-05-14 Duke University Synthetic regulation of gene expression
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
JP2016517278A (en) 2013-03-15 2016-06-16 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Vectors comprising stuffer / filler polynucleotide sequences and methods of use thereof
KR20160010526A (en) 2013-05-15 2016-01-27 리젠츠 오브 더 유니버시티 오브 미네소타 Adeno-associated virus mediated gene transfer to the central nervous system
EP3929279A1 (en) 2014-03-18 2021-12-29 Sangamo Therapeutics, Inc. Methods and compositions for regulation of zinc finger protein expression
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
US11020443B2 (en) 2015-04-23 2021-06-01 University Of Massachusetts Modulation of AAV vector transgene expression
CN104846015B (en) 2015-05-27 2018-03-27 深圳先进技术研究院 The composition of GABA serotonergic neurons in special heat nucleus accumbens septi and its application in schizophrenia difference behavior is improved
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3368065A4 (en) 2015-10-29 2019-03-20 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
TW202315946A (en) 2017-04-03 2023-04-16 美商編碼製藥公司 Tissue selective transgene expression

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