SG10201601110VA - Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer - Google Patents

Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer

Info

Publication number
SG10201601110VA
SG10201601110VA SG10201601110VA SG10201601110VA SG10201601110VA SG 10201601110V A SG10201601110V A SG 10201601110VA SG 10201601110V A SG10201601110V A SG 10201601110VA SG 10201601110V A SG10201601110V A SG 10201601110VA SG 10201601110V A SG10201601110V A SG 10201601110VA
Authority
SG
Singapore
Prior art keywords
aav9
compositions
improving
methods
gene transfer
Prior art date
Application number
SG10201601110VA
Inventor
James M Wilson
Christie L Bell
Luc H Vandenberghe
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of SG10201601110VA publication Critical patent/SG10201601110VA/en

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/66Phosphorus compounds
    • A61K31/683Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols
    • A61K31/685Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols one of the hydroxy compounds having nitrogen atoms, e.g. phosphatidylserine, lecithin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/861Adenoviral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14041Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14041Use of virus, viral particle or viral elements as a vector
    • C12N2750/14045Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
SG10201601110VA 2011-02-17 2012-02-17 Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer SG10201601110VA (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201161443879P 2011-02-17 2011-02-17

Publications (1)

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SG10201601110VA true SG10201601110VA (en) 2016-03-30

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SG10202007803QA SG10202007803QA (en) 2011-02-17 2012-02-17 Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
SG2013058920A SG192268A1 (en) 2011-02-17 2012-02-17 Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
SG10201601110VA SG10201601110VA (en) 2011-02-17 2012-02-17 Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer

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SG2013058920A SG192268A1 (en) 2011-02-17 2012-02-17 Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer

Country Status (16)

Country Link
US (5) US9884071B2 (en)
EP (1) EP2675902B1 (en)
JP (1) JP6224459B2 (en)
KR (1) KR101956885B1 (en)
CN (1) CN103502458B (en)
AU (1) AU2012219380B2 (en)
BR (1) BR112013020734A2 (en)
CA (1) CA2826316C (en)
DK (1) DK2675902T3 (en)
ES (1) ES2724800T3 (en)
HU (1) HUE043921T2 (en)
MX (1) MX353930B (en)
PL (1) PL2675902T3 (en)
SG (3) SG10202007803QA (en)
TR (1) TR201908020T4 (en)
WO (1) WO2012112832A1 (en)

Families Citing this family (101)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1856576B (en) * 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2011133901A2 (en) 2010-04-23 2011-10-27 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
CA2833908C (en) 2010-04-23 2021-02-09 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
DK2673289T3 (en) * 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill VIRUS VECTORS WITH MODIFIED TRANSDUCTION PROFILES AND METHODS FOR THEIR PRODUCTION AND USE
BR112013020734A2 (en) 2011-02-17 2017-06-13 Univ Pennsylvania compositions and methods for altering tissue specificity and enhancing aav9-mediated gene transfer
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
EP2749569A1 (en) 2012-12-28 2014-07-02 Annibale Alessandro Puca Variant of BPIFB4 protein
US20140219964A1 (en) 2013-02-07 2014-08-07 Children's Medical Center Corporation Methods for inducing cardiomyocyte proliferation
WO2014160092A1 (en) * 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
JP6396988B2 (en) * 2013-03-15 2018-09-26 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Methods and compositions for double glycan binding AAV vectors
EP2984166B1 (en) 2013-03-15 2020-04-22 The Trustees Of The University Of Pennsylvania Compositions for treating mpsi
EP3628334B1 (en) 2014-03-21 2023-06-28 Genzyme Corporation Gene therapy for retinitis pigmentosa
WO2015164757A1 (en) * 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
WO2016044478A1 (en) 2014-09-16 2016-03-24 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
EP4012035A1 (en) 2014-09-16 2022-06-15 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
RU2738421C2 (en) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Versions of recombinant aav and use thereof
WO2016073693A2 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
JP6863891B2 (en) 2014-11-14 2021-04-21 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. Regulatory polynucleotide
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
CN107646052A (en) 2015-01-20 2018-01-30 建新公司 For characterizing the analytical supercentrifugation of recombinant virus particle
MX2017010369A (en) 2015-02-10 2017-12-14 Genzyme Corp VARIANT RNAi.
JP6928558B2 (en) 2015-02-10 2021-09-01 ジェンザイム・コーポレーション Enhanced delivery of viral particles to the striatum and cortex
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
TWI707951B (en) 2015-04-08 2020-10-21 美商健臻公司 Production of oversized adeno-associated vectors
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
PT3298134T (en) 2015-05-16 2023-08-18 Genzyme Corp Gene editing of deep intronic mutations
US20180214576A1 (en) * 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
ES2918998T3 (en) 2015-12-11 2022-07-21 Univ Pennsylvania Scalable purification method for AAVrh10
IL259964B (en) 2015-12-15 2022-09-01 Genzyme Corp Adeno-associated viral vectors for treating mucolipidosis type ii
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
KR20180121899A (en) 2016-02-03 2018-11-09 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Gene therapy to treat type I mucopolysaccharidosis
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
EP3452104A1 (en) 2016-04-15 2019-03-13 The Trustees of The University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR102234930B1 (en) 2016-05-13 2021-04-02 4디 몰레큘러 테라퓨틱스 아이엔씨. Adeno-associated virus variant capsid and uses thereof
EP3458589A4 (en) 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc Modulatory polynucleotides
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CN106093369B (en) * 2016-06-29 2017-10-20 福建农林大学 Method for evaluating safety of the Bt paddy rice to non-target insects brown paddy plant hopper
MA55748A (en) 2016-08-15 2022-03-02 Genzyme Corp AAV DETECTION METHODS
CN110650673B (en) 2016-08-30 2024-04-09 加利福尼亚大学董事会 Methods for biomedical targeting and delivery and devices and systems for practicing the methods
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
MX2019013172A (en) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease.
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
JP2020526514A (en) 2017-07-06 2020-08-31 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 Gene therapy to treat mucopolysaccharidosis type I
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP4218828A3 (en) 2017-09-20 2023-09-27 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US11819539B2 (en) 2017-09-22 2023-11-21 The Trustees Of The University Of Pennsylvania Gene therapy for treating Mucopolysaccharidosis type II
SG11202002488UA (en) 2017-09-22 2020-04-29 Genzyme Corp Variant rnai
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CA3077426A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7184894B2 (en) 2017-11-27 2022-12-06 4ディー モレキュラー セラピューティクス インコーポレイテッド Adeno-associated virus mutated capsid and use for inhibition of angiogenesis
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
BR112020015511A2 (en) * 2018-02-01 2021-01-26 Homology Medicines, Inc. adeno-associated virus compositions for gene transfer from pah and methods of using them
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
US20210107948A1 (en) 2018-04-05 2021-04-15 Genethon Hybrid Recombinant Adeno-Associated Virus Serotype Between AAV9 and AAVrh74 with Reduced Liver Tropism
WO2019213668A1 (en) * 2018-05-04 2019-11-07 Oregon Health & Science University Human anti-aav2 capsid polyclonal antibody epitopes
US20210301305A1 (en) 2018-06-13 2021-09-30 Voyager Therapeutics, Inc. Engineered untranslated regions (utr) for aav production
JP2021530548A (en) 2018-07-24 2021-11-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. Systems and methods for producing gene therapy products
KR20210062627A (en) 2018-08-03 2021-05-31 젠자임 코포레이션 Variant RNAi for alpha-synuclein
TW202035689A (en) 2018-10-04 2020-10-01 美商航海家醫療公司 Methods for measuring the titer and potency of viral vector particles
EP3861107A1 (en) 2018-10-05 2021-08-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
JP2022504740A (en) 2018-10-12 2022-01-13 ジェンザイム・コーポレーション Generation of improved human PAHs for the treatment of severe PKU by gene replacement therapy directed to the liver
CA3116701A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
UY38407A (en) 2018-10-15 2020-05-29 Novartis Ag TREM2 STABILIZING ANTIBODIES
CA3128230A1 (en) 2019-01-31 2020-08-06 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
JP2022520875A (en) 2019-02-25 2022-04-01 ノバルティス アーゲー Compositions and Methods for Treating Bietti Crystallin Retinopathy
US20220047618A1 (en) 2019-02-28 2022-02-17 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
CN114008209A (en) * 2019-04-12 2022-02-01 马萨诸塞大学 AAV-mediated Maple Syrup Urine Disease (MSUD) gene therapy
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
WO2021087366A1 (en) * 2019-11-01 2021-05-06 Sangamo Therapeutics, Inc. Compositions and methods for genome engineering
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
IL301677A (en) 2020-10-01 2023-05-01 Genzyme Corp Human pah expression cassette for treatment of pku by liver-directed gene replacement therapy
AU2022214429A1 (en) 2021-02-01 2023-09-14 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CA3227103A1 (en) 2021-07-30 2023-02-02 Matthew P. GEMBERLING Compositions and methods for modulating expression of frataxin (fxn)
AU2022318664A1 (en) 2021-07-30 2024-02-29 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
WO2023147304A1 (en) * 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2024015881A2 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5774090A (en) 1980-10-27 1982-05-10 Shionogi & Co Ltd Novel beta-galactosidase inhibitor, gt-2558, its derivative, and production
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
US4542104A (en) 1983-04-06 1985-09-17 The Board Of Trustees Of The Leland Stanford Jr. Univ. Phycobiliprotein fluorescent conjugates
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
EP0162067B1 (en) 1983-10-28 1992-07-15 Genetics Institute, Inc. Production of factor viii and related products
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
FI86885C (en) 1984-04-20 1992-10-26 Genentech Inc Method for Preparation of Human Recombinant Factor VIII and Nucleic Acid Sequences and Vectors Used thereto
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
FI98829C (en) 1986-01-27 1997-08-25 Chiron Corp Process for Preparation of Recombinant Protein Complex with Human Factor VIII: C Activity
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
IE69026B1 (en) 1987-06-12 1996-08-07 Immuno Ag Novel proteins with factor VIII activity process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
FR2619314B1 (en) 1987-08-11 1990-06-15 Transgene Sa FACTOR VIII ANALOG, PREPARATION METHOD AND PHARMACEUTICAL COMPOSITION CONTAINING THE SAME
US5004803A (en) 1988-11-14 1991-04-02 Genetics Institute, Inc. Production of procoagulant proteins
EP0670332A3 (en) 1989-11-17 1995-10-25 Novo Nordisk As Protein complexes having factor VIII:C activity and production thereof.
SE465222C5 (en) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab A recombinant human factor VIII derivative and process for its preparation
US5171846A (en) 1990-05-21 1992-12-15 Coulter Corporation Method of preferential labelling of a phycobiliprotein with a second dye for use in a multiple color assay and product for such use
US5272257A (en) 1990-05-21 1993-12-21 Coulter Corporation Method of preferential labelling of a phycobiliprotein with an amine-reactive dye for use in a multiple color assay and product for such use
US5240640A (en) 1990-06-04 1993-08-31 Coulter Corporation In situ use of gelatin or an aminodextran in the preparation of uniform ferrite particles
US5466609A (en) 1990-10-31 1995-11-14 Coulter Corporation Biodegradable gelatin-aminodextran particle coatings of and processes for making same
US5639620A (en) 1990-10-31 1997-06-17 Coulter Corporation Polymeric particles having a biodegradable gelatin or aminodextran coating and process for making same
SE468050C (en) 1991-03-15 1998-02-11 Pharmacia & Upjohn Ab Recombinant human factor VIII derivative
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
US5248771A (en) 1991-06-26 1993-09-28 Triumf Process for preparing no-carrier-added radiohalogenated vinyl nucleosides
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
US5248772A (en) 1992-01-29 1993-09-28 Coulter Corporation Formation of colloidal metal dispersions using aminodextrans as reductants and protective agents
AU5602394A (en) 1992-11-13 1994-06-08 Duke University Chimeric blood coagulation proteins
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0874057B1 (en) 1993-06-10 2004-07-28 Bayer Corporation Vector and mammalian cell line having improved productivity
CA2164646A1 (en) 1993-06-11 1994-12-22 Wade E. Bolton Anti-cd3 antibody-aminodextran conjugates for induction of t-cell activation and proliferation
US5681746A (en) 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
AU6486196A (en) 1995-07-11 1997-02-10 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6096273A (en) 1996-11-05 2000-08-01 Clinical Micro Sensors Electrodes linked via conductive oligomers to nucleic acids
US5945293A (en) 1997-10-09 1999-08-31 Coulter International Corp. Protein-colloidal metal-aminodextran coated particle and methods of preparation and use
US6074884A (en) 1997-10-09 2000-06-13 Coulter International Corp. Stable protein-nickel particles and methods of production and use thereof
US6117643A (en) 1997-11-25 2000-09-12 Ut Battelle, Llc Bioluminescent bioreporter integrated circuit
US6197503B1 (en) 1997-11-26 2001-03-06 Ut-Battelle, Llc Integrated circuit biochip microsystem containing lens
US6287857B1 (en) * 1998-02-09 2001-09-11 Genzyme Corporation Nucleic acid delivery vehicles
WO2000014197A1 (en) 1998-09-09 2000-03-16 Hitachi Software Engineering Co., Ltd. Biochip and method of using biochip
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
CN1856576B (en) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
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
CN102439157B (en) 2009-04-30 2015-09-16 宾夕法尼亚大学托管会 Comprise the target conducting airways cell composition of adeno associated virus construct
JP5458245B2 (en) 2010-06-25 2014-04-02 国立大学法人九州工業大学 Operation control apparatus, method thereof, and program
WO2012109214A1 (en) 2011-02-07 2012-08-16 The University Of North Carolina At Chapel Hill Targeted transduction of aav vectors
DK2673289T3 (en) 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill VIRUS VECTORS WITH MODIFIED TRANSDUCTION PROFILES AND METHODS FOR THEIR PRODUCTION AND USE
BR112013020734A2 (en) 2011-02-17 2017-06-13 Univ Pennsylvania compositions and methods for altering tissue specificity and enhancing aav9-mediated gene transfer

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