IL276368A - Adeno-associated virus compositions to restore PAH gene function and methods for using them - Google Patents

Adeno-associated virus compositions to restore PAH gene function and methods for using them

Info

Publication number
IL276368A
IL276368A IL276368A IL27636820A IL276368A IL 276368 A IL276368 A IL 276368A IL 276368 A IL276368 A IL 276368A IL 27636820 A IL27636820 A IL 27636820A IL 276368 A IL276368 A IL 276368A
Authority
IL
Israel
Prior art keywords
adeno
methods
associated virus
gene function
pah gene
Prior art date
Application number
IL276368A
Other languages
English (en)
Hebrew (he)
Original Assignee
Homology Medicines Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Homology Medicines Inc filed Critical Homology Medicines Inc
Publication of IL276368A publication Critical patent/IL276368A/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10051—Methods of production or purification of viral material
    • C12N2710/10052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
IL276368A 2018-02-01 2020-07-29 Adeno-associated virus compositions to restore PAH gene function and methods for using them IL276368A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862625149P 2018-02-01 2018-02-01
US201862672377P 2018-05-16 2018-05-16
PCT/US2019/016354 WO2019152843A1 (en) 2018-02-01 2019-02-01 Adeno-associated virus compositions for restoring pah gene function and methods of use thereof

Publications (1)

Publication Number Publication Date
IL276368A true IL276368A (en) 2020-09-30

Family

ID=67478926

Family Applications (1)

Application Number Title Priority Date Filing Date
IL276368A IL276368A (en) 2018-02-01 2020-07-29 Adeno-associated virus compositions to restore PAH gene function and methods for using them

Country Status (14)

Country Link
US (3) US20190256867A1 (https=)
EP (1) EP3746101A4 (https=)
JP (1) JP2021512871A (https=)
KR (1) KR20200127170A (https=)
CN (1) CN111902164A (https=)
AU (1) AU2019215152A1 (https=)
BR (1) BR112020015798A2 (https=)
CA (1) CA3090226A1 (https=)
CL (1) CL2020002008A1 (https=)
CO (1) CO2020010793A2 (https=)
IL (1) IL276368A (https=)
MX (1) MX2020008133A (https=)
PE (1) PE20212076A1 (https=)
WO (1) WO2019152843A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
IL294713A (en) * 2020-01-16 2022-09-01 Takeda Pharmaceuticals Co Adeno associated virus based gene therapy for phenylketonuria
WO2021207077A1 (en) * 2020-04-06 2021-10-14 Homology Medicines, Inc. Adeno-associated virus compositions for ids gene transfer and methods of use thereof
TW202208632A (zh) * 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
JP2023544165A (ja) 2020-10-01 2023-10-20 ジェンザイム・コーポレーション 肝臓に向けられた遺伝子補充療法によってｐｋｕを処置するためのヒトｐａｈ発現カセット

Family Cites Families (172)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL71505A0 (en) 1983-04-14 1984-07-31 Hughes Howard Med Inst Phenylalanine hydroxylase cdna clones,their production and their use in the diagnosis of classical phenylketonuria
IL74761A0 (en) 1984-04-13 1985-06-30 Hughes Howard Med Inst Human phenylalanine hydroxylase cdna clones,their production and their use in diagnosing classical phenylketonuria
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
DE4339922C1 (de) 1993-09-03 1994-10-06 Max Planck Gesellschaft Vektor für Leber-Gentherapie
US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
DE4407859C1 (de) 1994-03-04 1995-03-02 Max Planck Gesellschaft Vektor für die leberspezifische Gentherapie
CA2187626C (en) 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
AU3718595A (en) 1994-09-16 1996-03-29 Uab Research Foundation, The Transduction of genes into human hematopoietic stem cells using recombinant adeno-associated viral vectors
CA2200835A1 (en) 1994-09-23 1996-03-28 Frederick M. Boyce Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5650309A (en) 1995-05-16 1997-07-22 The Regents Of The University Of California Viral vectors
US7001764B2 (en) 1995-06-27 2006-02-21 Cell Genesys, Inc. Compositions comprising tissue specific adenoviral vectors
DE19525900C1 (de) 1995-07-15 1996-12-12 Max Planck Gesellschaft Leberspezifischer Adenovirus-Expressionsvektor
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5780447A (en) 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
US6521225B1 (en) 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6281009B1 (en) 1996-09-11 2001-08-28 The General Hospital Corporation Use of a non-mammalian DNA virus to express an exogenous gene in a mammalian cell
DE19644500C2 (de) 1996-10-25 1998-11-05 Deutsches Krebsforsch AAV-DNA mit Helfervirus-Sequenzen
FR2755699B1 (fr) 1996-11-08 1998-12-18 Rhone Poulenc Rorer Sa Nouvelles constructions et vecteurs pour l'expression ciblee et inductible des genes
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
AU5588298A (en) 1996-12-02 1998-06-29 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
CA2269661A1 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Recombinase-activatable aav packaging cassettes for use in the production of aav vectors
CA2275892A1 (en) 1996-12-23 1998-07-02 Gene-Cell, Inc. Nucleic acid constructs and uses thereof for direct nucleic acid incorporation into cells
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
AU744445B2 (en) 1997-04-24 2002-02-21 University Of Washington Targeted gene modification by parvoviral vectors
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
GB9715064D0 (en) 1997-07-17 1997-09-24 Ppl Therapeutics Scotland Ltd Protein expression
WO1999018227A1 (en) 1997-10-08 1999-04-15 Advanced Research And Technology Institute Chimeric parvovirus-based recombinant vector system that specifically targets the erythroid lineage
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
WO1999064569A1 (en) 1998-06-10 1999-12-16 Advanced Research And Technology Institute Methods and compositions for generating recombinant adeno-associated virus vectors
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
US6919209B1 (en) 1998-12-04 2005-07-19 City Of Hope Method of genetically modifying very primitive quiescent human hematopoietic stem cells
US6200758B1 (en) 1999-02-19 2001-03-13 New York State Office Of Mental Health Phenylalanine hydroxylase gene variants, and amino acid and pterin homeostasis, in the definition, detection, treatment and prevention of psychotic, mood and personality disorders
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6610906B1 (en) 1999-06-09 2003-08-26 The Regents Of The University Of Michigan Nucleotide sequences for gene regulation and methods of use thereof
ES2284545T3 (es) 1999-11-16 2007-11-16 Genzyme Corporation Vectores y transgenes con elementos reguladores para la administracion de genes del higado.
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
AU2002306539A1 (en) 2001-02-16 2002-09-04 The Board Of Trustees Of The Leland Stanford Junior University Minimal plasmid vectors that provide for persistent and high level gene expression and methods for using the same
DE10120265A1 (de) 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren
ATE437221T1 (de) 2001-05-14 2009-08-15 Gbp Ip Llc Lentivirale vektoren kodierend für gerinnungsfaktoren für die gentherapie
AU2002305778A1 (en) 2001-05-31 2002-12-09 The Rockefeller University Method for generating replication defective viral vectors that are helper free
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
US20030129203A1 (en) 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
EP1442125A4 (en) 2001-10-04 2004-12-08 Carlos Estuard Aguilar-Cordova CHIMERAL VIRUS VECTORS FOR GENE THERAPY
CA2361462A1 (en) 2001-11-07 2003-05-08 Katherine A. High Induction of tolerance to a therapeutic polypeptide
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) * 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
CA2369985A1 (en) 2002-01-18 2003-07-18 Duke University Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
CA2426283C (en) 2002-04-29 2006-06-27 The Trustees Of The University Of Pennsylvania Method for direct rescue and amplification of integrated viruses from cellular dna of tissues
AU2003237159A1 (en) 2002-04-30 2003-11-17 University Of Florida Treatment for phenylketonuria
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
US20070202587A1 (en) 2002-09-23 2007-08-30 Applied Genetic Technologies Corporation Recombinant AAV production in mammalian cells
US7091029B2 (en) 2002-09-23 2006-08-15 Applied Genetics Technologies Corporation High titer recombinant AAV production
SI3235827T1 (sl) 2003-06-19 2021-05-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
US7482156B2 (en) 2003-10-15 2009-01-27 Cell Genesys, Inc. Hepatocellular carcinoma specific promoter and uses thereof
JP2007531537A (ja) 2004-04-06 2007-11-08 セダーズ−シナイ　メディカル　センター アポリポタンパク質ａ−ｉおよびアポリポタンパク質ａ−ｉｍｉｌａｎｏをコードする組換えアデノ随伴ウイルスベクターによる血管疾患の予防および処置
US8716461B2 (en) 2004-05-24 2014-05-06 Blood Systems, Inc. Human parvovirus
EP1804839B1 (en) 2004-09-22 2012-03-14 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
TWI293307B (en) 2004-09-30 2008-02-11 Ind Tech Res Inst A liver-specific chimeric regulatory sequence and use thereof
AU2006220500A1 (en) 2005-03-09 2006-09-14 Board Of Regents, The University Of Texas System Novel hTMC promoter and vectors for the tumor-selective and high-efficient expression of cancer therapeutic genes
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
EP1928504B1 (en) 2005-08-19 2013-04-24 University of Technology, Sydney Liver-directed gene therapy
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
WO2008150459A1 (en) 2007-05-30 2008-12-11 The Trustees Of The University Of Pennsylvania A method for transducing cells with primary cilia
EP2008656A1 (en) 2007-06-28 2008-12-31 Bergen Teknologioverforing AS Compositions for the treatment of hyperphenylalaninemia
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US20090215879A1 (en) 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
EP4154903A1 (en) 2008-04-22 2023-03-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010124180A1 (en) 2009-04-23 2010-10-28 Phasebio Pharmaceuticals, Inc. Phenylalanine hydroxylase fusion protein and methods for treating phenylketonuria
ES2724122T3 (es) 2009-04-30 2019-09-06 Univ Pennsylvania Composiciones para dirigir células de las vías respiratorias de conducción que comprenden construcciones de virus adenoasociado
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
US8476418B2 (en) 2009-05-28 2013-07-02 Deutsches Krebsforschungszentrum Modified AAV capsid polypeptides
HUE028341T2 (en) 2009-06-16 2016-12-28 Genzyme Corp Improved methods for purification of recombinant aav vectors
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
WO2011041502A1 (en) 2009-10-01 2011-04-07 The Trustees Of The University Of Pennsylvania Aav vectors expressing sec1o for treating kidney damage
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
DK2529020T3 (en) 2010-01-28 2018-08-06 Childrens Hospital Philadelphia SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY
WO2011117258A2 (en) 2010-03-22 2011-09-29 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
US8628966B2 (en) * 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
US20120014932A1 (en) 2010-07-14 2012-01-19 Golden William J Methods for treating disorders of amino acid metabolism
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
ES2685611T3 (es) 2011-02-14 2018-10-10 The Children's Hospital Of Philadelphia Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo
DK2675902T3 (da) 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
SG10201600912SA (en) 2011-03-04 2016-03-30 Intrexon Corp Vectors conditionally expressing protein
HRP20171334T1 (hr) 2011-04-22 2017-11-17 The Regents Of The University Of California Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
JP6188703B2 (ja) 2011-10-27 2017-08-30 サンガモ セラピューティクス， インコーポレイテッド Ｈｐｒｔ遺伝子座を修飾するための方法および組成物
JP6348064B2 (ja) 2011-11-22 2018-06-27 ザ チルドレンズ ホスピタル オブ フィラデルフィア 効率の高いトランスジーン送達のためのウイルスベクター
WO2013096955A1 (en) 2011-12-23 2013-06-27 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
AU2013221212B2 (en) 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
AU2013249202B2 (en) 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
WO2013158309A2 (en) 2012-04-18 2013-10-24 The Board Of Trustees Of The Leland Stanford Junior University Non-disruptive gene targeting
JP2016528873A (ja) * 2012-05-16 2016-09-23 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
BR112015000161A2 (pt) 2012-07-06 2017-06-27 Univ Iowa Res Found composições de vetor de vírus adeno-associado modificado
AU2013296425B2 (en) 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
US9636370B2 (en) 2012-09-28 2017-05-02 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
EP2911687B1 (en) 2012-10-26 2019-02-13 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
WO2014089212A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
EP2940131B1 (en) 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
US20150352228A1 (en) 2013-01-11 2015-12-10 Bruce TORBET Methods and compositions for enhancing transduction efficiency of retroviral vectors
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
EP2970946A4 (en) 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
MX384795B (es) 2013-03-15 2025-03-14 Childrens Hospital Philadelphia Vectores que comprenden secuencias de polinucleotidos rellenadoras/de relleno y metodos de uso.
SI2984166T1 (sl) 2013-03-15 2020-09-30 The Trustees Of The University Of Pennsylvania Sestavki za zdravljenje MPSI
KR102234672B1 (ko) 2013-05-21 2021-04-06 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
HK1222673A1 (zh) 2013-07-12 2017-07-07 The Children's Hospital Of Philadelphia Aav载体和用於抗aav(腺相关病毒)中和抗体的检测
KR20240090694A (ko) 2013-07-22 2024-06-21 더 칠드런스 호스피탈 오브 필라델피아 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
CN105899665B (zh) 2013-10-17 2019-10-22 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
BR112016019940A2 (pt) * 2014-03-21 2017-10-24 Univ Leland Stanford Junior edição de genoma sem nucleases
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
RS60359B1 (sr) * 2014-06-06 2020-07-31 Regeneron Pharma Postupci i kompozicije za modifikovanje ciljanog lokusa
BR112017005892A2 (pt) * 2014-09-24 2017-12-12 Hope City variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
CN107249646B (zh) 2014-12-16 2021-06-29 内布拉斯加大学董事会 用于青少年巴滕病的基因疗法
EP3233129B1 (en) 2014-12-17 2020-01-15 Fundacion para la Investigacion Medica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions
ES2876412T3 (es) 2014-12-17 2021-11-12 Fundacion Para La Investig Medica Aplicada Construcciones de ácido nucleico y vectores de terapia génica para su uso en el tratamiento de la enfermedad de Wilson
KR102554850B1 (ko) 2015-02-06 2023-07-13 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
EP3270944B1 (en) 2015-03-17 2019-10-23 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
CN115074366A (zh) 2015-04-16 2022-09-20 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
WO2017015154A1 (en) 2015-07-17 2017-01-26 Children's National Medical Center Methods and compositions for treating phenyltonuria
CA3002524A1 (en) 2015-10-28 2017-05-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof
US11389546B2 (en) 2015-12-09 2022-07-19 Modernatx, Inc. Heterologous UTR sequences for enhanced mRNA expression
CA3007330A1 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Composition for treatment of crigler-najjar syndrome
PL3411484T3 (pl) 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
GB201603577D0 (en) * 2016-03-01 2016-04-13 Oxford Genetics Ltd Promoter
EP3509628A1 (en) 2016-09-09 2019-07-17 Fondazione Telethon Combined therapy for mucopolysaccharidosis type vi (maroteaux-lamy-syndrome)
KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
MX2019007873A (es) 2016-12-30 2019-11-18 Univ Pennsylvania Terapia génica para tratar la enfermedad de wilson.
FI3568483T3 (fi) 2017-01-10 2025-11-21 Childrens Medical Res Institute Polynukleotideja ja vektoreita siirtogeenien ilmentämiseen
EP3596206A1 (en) 2017-03-16 2020-01-22 Pfizer Inc Tyrosine prototrophy
CA3057142A1 (en) 2017-04-03 2018-10-11 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
EP3589329B1 (en) 2017-05-11 2022-10-12 Zentrum für Forschungsförderung in der Pädiatrie GmbH Concept for the treatment of monogenetic disorders
EP3630200A4 (en) 2017-05-31 2021-02-24 Arcturus Therapeutics, Inc. THERAPEUTICS FOR PHENYLKETONURIA
JP7423521B2 (ja) * 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド

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