SG11202110146VA - Recombinant adeno-associated virus vectors - Google Patents

Recombinant adeno-associated virus vectors

Info

Publication number
SG11202110146VA
SG11202110146VA SG11202110146VA SG11202110146VA SG11202110146VA SG 11202110146V A SG11202110146V A SG 11202110146VA SG 11202110146V A SG11202110146V A SG 11202110146VA SG 11202110146V A SG11202110146V A SG 11202110146VA SG 11202110146V A SG11202110146V A SG 11202110146VA
Authority
SG
Singapore
Prior art keywords
associated virus
virus vectors
recombinant adeno
adeno
recombinant
Prior art date
Application number
SG11202110146VA
Other languages
English (en)
Inventor
Daniel Mccoy
Garrett E Berry
Original Assignee
Stridebio 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 Stridebio Inc filed Critical Stridebio Inc
Publication of SG11202110146VA publication Critical patent/SG11202110146VA/en

Links

Classifications

    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SG11202110146VA 2019-03-21 2020-03-20 Recombinant adeno-associated virus vectors SG11202110146VA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962821710P 2019-03-21 2019-03-21
PCT/US2020/023877 WO2020191300A1 (en) 2019-03-21 2020-03-20 Recombinant adeno-associated virus vectors

Publications (1)

Publication Number Publication Date
SG11202110146VA true SG11202110146VA (en) 2021-10-28

Family

ID=70289855

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202110146VA SG11202110146VA (en) 2019-03-21 2020-03-20 Recombinant adeno-associated virus vectors

Country Status (16)

Country Link
US (2) US20220064675A1 (de)
EP (1) EP3941929A1 (de)
JP (1) JP2022525955A (de)
KR (1) KR20220011616A (de)
CN (1) CN113727992A (de)
AR (1) AR118465A1 (de)
AU (1) AU2020241888A1 (de)
BR (1) BR112021018776A2 (de)
CA (1) CA3133453A1 (de)
CO (1) CO2021013878A2 (de)
EA (1) EA202192501A1 (de)
IL (1) IL286464A (de)
MX (1) MX2021011468A (de)
SG (1) SG11202110146VA (de)
TW (1) TW202102525A (de)
WO (1) WO2020191300A1 (de)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3773743A1 (de) 2018-04-03 2021-02-17 Stridebio, Inc. Virusvektoren gegen ophthalmisches gewebe
MX2020010466A (es) 2018-04-03 2021-01-08 Vectores de virus que evitan anticuerpos.
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
AU2021218411A1 (en) 2020-02-13 2022-10-06 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
KR20230029616A (ko) 2020-05-05 2023-03-03 듀크 유니버시티 종간 호환가능한 아데노-관련 바이러스 조성물 및 이의 사용 방법
BR112022023106A2 (pt) 2020-05-13 2023-01-17 Voyager Therapeutics Inc Redirecionamento de tropismo de capsídeos de aav
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
CN113025658B (zh) * 2021-03-10 2022-04-22 华侨大学 一种神经干细胞特异性基因的递送载体及其应用
CN112961220B (zh) * 2021-04-19 2023-11-17 信念医药科技(上海)有限公司 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体
JP2024523707A (ja) 2021-07-08 2024-06-28 テナヤ セラピューティクス， インコーポレイテッド 遺伝子治療のための最適化された発現カセット
WO2023060264A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2023150687A1 (en) * 2022-02-04 2023-08-10 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors, and methods of use thereof
WO2023168333A1 (en) * 2022-03-03 2023-09-07 California Institute Of Technology Compositions and methods for crossing blood brain barrier
KR102578015B1 (ko) 2022-07-18 2023-09-18 주식회사 마크헬츠 뇌혈관 내피세포에 특이적으로 발현되는 프로모터를 포함하고 뇌혈관 내피세포 표적용 나노-펩타이드가 코팅된 aav 벡터 및 이의 뇌혈관 내피세포 특이적 유전자 전달 용도
TW202421787A (zh) 2022-09-06 2024-06-01 美商特納亞治療股份有限公司 保護心臟之心臟病療法

Family Cites Families (254)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4039388A (en) 1976-06-04 1977-08-02 The United States Of America As Represented By The Government Diagnostic test for Niemann-Pick disease
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
EP0322417A1 (de) 1986-09-08 1989-07-05 Applied Biotechnology, Inc. Leere virus kapsidvakzine
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
EP0442926A1 (de) 1988-11-10 1991-08-28 Imperial Cancer Research Technology Limited Polypeptide
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
DE69433592T2 (de) 1993-11-09 2005-02-10 Targeted Genetics Corp., Seattle Die erzielung hoher titer des rekombinanten aav-vektors
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
WO1995028493A1 (en) 1994-04-13 1995-10-26 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
CA2223837C (en) 1995-06-07 2010-05-25 University Of North Carolina At Chapel Hill Aav transduction of myoblasts
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
CA2240494C (en) 1995-12-15 2007-03-13 Lloyd G. Mitchell Therapeutic molecules generated by trans-splicing
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6426198B1 (en) 1997-07-03 2002-07-30 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Genes for Niemann-Pick type C disease
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
ATE402254T1 (de) 1998-05-28 2008-08-15 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
US6562958B1 (en) 1998-06-09 2003-05-13 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to Acinetobacter baumannii for diagnostics and therapeutics
US8299321B2 (en) 1998-06-16 2012-10-30 Monsanto Technology Llc Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20100293663A2 (en) 1998-06-16 2010-11-18 Thomas La Rosa Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
NZ511171A (en) 1998-09-22 2004-02-27 Univ Florida Methods for large-scale production of recombinant AAV vectors
GB9822763D0 (en) 1998-10-20 1998-12-16 Univ Sheffield Immunoglobin variant
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
PT1127150E (pt) 1998-11-05 2007-08-22 Univ Pennsylvania ''sequências de ácido nucleico do vírus adeno associado do serotipo 1, vectores e células hospedeiras que as contêm''
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
US6822071B1 (en) 1998-11-12 2004-11-23 The Regents Of The University Of California Polypeptides from Chlamydia pneumoniae and their use in the diagnosis, prevention and treatment of disease
EP1887081A2 (de) 1999-02-25 2008-02-13 Ceres Incorporated DNS-Sequenzen
EP1033405A3 (de) 1999-02-25 2001-08-01 Ceres Incorporated DNS-fragmente mit bestimmter Sequenz und die dadurch kodierte Polypeptide
US20130326723A1 (en) 1999-05-06 2013-12-05 Thomas J. La Rosa Soy nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20110214206A1 (en) 1999-05-06 2011-09-01 La Rosa Thomas J Nucleic acid molecules and other molecules associated with plants
US20080229439A1 (en) 1999-05-06 2008-09-18 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20090087878A9 (en) 1999-05-06 2009-04-02 La Rosa Thomas J Nucleic acid molecules associated with plants
US20110093981A9 (en) 1999-05-06 2011-04-21 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20040031072A1 (en) 1999-05-06 2004-02-12 La Rosa Thomas J. Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
AU2138601A (en) 1999-12-22 2001-07-03 Aventis Pasteur Limited Chlamydia antigens and corresponding DNA fragments and uses thereof
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US20140130203A1 (en) 2000-04-19 2014-05-08 Thomas J. La Rosa Rice nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20110131679A2 (en) 2000-04-19 2011-06-02 Thomas La Rosa Rice Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
AU2001255524A1 (en) 2000-04-21 2001-11-07 Corixa Corporation Compositions and methods for the therapy and diagnosis of acne vulgaris
WO2001092551A2 (en) 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
US7214786B2 (en) 2000-12-14 2007-05-08 Kovalic David K Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
US20040071659A1 (en) 2001-04-30 2004-04-15 Betty Chang Viral-mediated delivery and in vivo expression of polynucleotides encoding anti-angiogenic proteins
AU2002345250A1 (en) 2001-06-22 2003-01-08 Syngenta Participations Ag Plant disease resistance genes
EP1402042A2 (de) 2001-06-22 2004-03-31 Syngenta Participations AG Auf abiotischen stress ansprechende polynukleotide und polypeptide
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
AU2001289843A1 (en) 2001-08-28 2002-02-13 Bayer Cropscience Ag Polypeptides for identifying herbicidally active compounds
WO2003033515A1 (en) 2001-10-15 2003-04-24 Corixa Corporation Compositions and methods for the therapy and diagnosis of acne vulgaris
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ578982A (en) 2001-11-13 2011-03-31 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
JP4769417B2 (ja) 2001-12-17 2011-09-07 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 アデノ随伴ウイルス（ａａｖ）血清型９の配列、それを含むベクターおよびその使用
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
WO2003093295A2 (en) 2002-04-30 2003-11-13 University Of North Carolina At Chapel Hill Secretion signal vectors
NZ561656A (en) 2002-05-01 2009-03-31 Univ Florida Improved rAAV expression systems for genetic modification of specific capsid proteins
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
JP2005185101A (ja) 2002-05-30 2005-07-14 National Institute Of Agrobiological Sciences 植物の全長ｃＤＮＡおよびその利用
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
US7306913B2 (en) 2002-12-20 2007-12-11 Applera Corporation Genetic polymorphisms associated with coronary stenosis, methods of detection and uses thereof
EP1575517B1 (de) 2002-12-24 2012-04-11 Rinat Neuroscience Corp. Anti-ngf-antikörper und verfahren zu ihrer verwendung
CA2528012C (en) 2003-06-02 2015-11-24 University Of Massachusetts Methods and compositions for controlling efficacy of rna silencing
SI2657248T1 (sl) 2003-06-19 2017-07-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in njihova uporaba
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
US7473531B1 (en) 2003-08-08 2009-01-06 Colora Corporation Pancreatic cancer targets and uses thereof
US20060107345A1 (en) 2003-09-30 2006-05-18 Nickolai Alexandrov Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
AU2005243247B2 (en) 2004-04-07 2012-03-01 Regents Of The University Of Minnesota Methods for treating bone cancer pain by administering a nerve growth factor antagonist
US20060171926A1 (en) 2004-04-30 2006-08-03 Passini Marco A Gene therapy for neurometabolic disorders
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
JP4346526B2 (ja) 2004-08-31 2009-10-21 株式会社東芝 半導体集積回路装置
WO2006029319A2 (en) 2004-09-09 2006-03-16 The General Hospital Corporation Modulating phosphatase activity in cardiac cells
NZ555830A (en) 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
EP1839669A4 (de) 2005-01-05 2012-12-12 Shionogi & Co Neue angiogenesehemmer
US20140199313A1 (en) 2005-03-02 2014-07-17 Metanomics Gmbh Process for the Production of Fine Chemicals
CA2598792A1 (en) 2005-03-02 2006-09-08 Metanomics Gmbh Process for the production of fine chemicals
JP2008539698A (ja) 2005-04-29 2008-11-20 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 転写後レベルでの核酸発現調節のための方法および組成物
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
EP1882392A4 (de) 2005-05-10 2009-07-01 Monsanto Technology Llc Gene und ihre verwendung zur pflanzenverbesserung
US20100095387A1 (en) 2005-07-15 2010-04-15 Martin Smith Methods and reagents for screening new drugs and for treating ion pump associated disorders and diseases
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
PL1986661T3 (pl) 2006-02-08 2019-02-28 Genzyme Corporation Terapia genowa choroby Niemanna-Picka typu A
EP2441770A1 (de) 2006-02-10 2012-04-18 The University of Cincinnati Phosphatasehemmer-Protein 1 als Regulator der Herzfunktion
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
JP2009535339A (ja) 2006-04-28 2009-10-01 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 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
CN101711164B (zh) 2007-01-18 2014-06-04 密苏里-哥伦比亚大学 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
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
KR100911624B1 (ko) 2007-05-14 2009-08-12 연세대학교 산학협력단 Ｉｌ－１２ 및 ｉｌ－２３의 효율적인 공동발현 방법
DE112008002435T5 (de) 2007-09-18 2010-07-22 Basf Plant Science Gmbh Pflanzen mit erhöhtem Ertrag
EP2058401A1 (de) 2007-10-05 2009-05-13 Genethon Weitverbreitete Genverabreichung an Motorneuronen unter Verwendung einer peripheren Injektion von AAV-Vektoren
US10526627B2 (en) 2007-11-30 2020-01-07 Corn Products Development, Inc Method for producing high molecular weight reduced viscosity starch pastes
US20090221620A1 (en) 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
EP2250268A4 (de) 2008-02-20 2011-08-24 Ceres Inc Verbesserte stickstoffverwendungseffizienzeigenschaften bei pflanzen verleihende nukleotidsequenzen und entsprechende polypeptide
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
CA2762118A1 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
JP2010002479A (ja) 2008-06-18 2010-01-07 Crossfor:Kk メガネレンズ用装飾体およびメガネ用装飾体装着具
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP2396343B1 (de) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modifizierte virusvektoren sowie verfahren zu ihrer herstellung und verwendung
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
CA2759801C (en) 2009-05-02 2019-04-02 Marco A. Passini Gene therapy for neurodegenerative disorders
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2011020118A1 (en) 2009-08-14 2011-02-17 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
EP2292781A1 (de) 2009-08-17 2011-03-09 Genethon Baculovirus-basierte Herstellung von Biopharmazeutika ohne kontaminierende baculovirale Virionen
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
EP2561073B1 (de) 2010-04-23 2016-08-24 University of Massachusetts Auf das zentralnervensystem abzielende vektoren und verfahren zu ihrer verwendung
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
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
JP5704361B2 (ja) 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
AU2011323199B2 (en) 2010-11-05 2016-01-28 The Board Of Trustees Of The Leland Stanford Junior University Stabilized step function opsin proteins and methods of using the same
WO2012064960A2 (en) 2010-11-10 2012-05-18 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
EP4234571A3 (de) 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Virale vektoren mit modifizierten transduktionsprofilen sowie verfahren zu ihrer herstellung und verwendung
US10392632B2 (en) 2011-02-14 2019-08-27 The Children's Hospital Of Philadelphia AAV8 vector with enhanced functional activity and methods of use thereof
WO2012159006A2 (en) 2011-05-18 2012-11-22 University Of Florida Research Foundation, Inc. Polypeptides and vectors for targeting her2/neu expressing cells and uses thereof
US9206425B2 (en) 2011-07-28 2015-12-08 Trustees Of Dartmouth College Methods for treating Niemann-Pick type C disease
WO2013027223A2 (en) 2011-08-23 2013-02-28 Evogene Ltd. Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics
WO2013078400A1 (en) 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
FI124176B (en) 2011-12-01 2014-04-15 Helsingin Yliopisto Polypeptide, polynucleotide, expression cassette, vector, host cell, plant and uses
US20140350087A9 (en) 2012-03-22 2014-11-27 Halozyme, Inc. Oncovector Nucleic Acid Molecules and Methods of Use
CA2866945C (en) 2012-03-27 2021-05-04 Curevac Gmbh Artificial nucleic acid molecules comprising a 5'top utr
MY172457A (en) 2012-04-18 2019-11-26 Childrens Hospital Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
EP2660325A3 (de) 2012-05-02 2014-02-12 Christian Medical College AAV-Vektoren und dazugehörige Nukleotidsequenzen und Verfahren
US9677088B2 (en) 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP2492347A1 (de) 2012-05-22 2012-08-29 Laboratorios Del. Dr. Esteve, S.A. Verfahren zur Herstellung von Vektoren
US20150182637A1 (en) 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
WO2014007858A1 (en) 2012-07-06 2014-01-09 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions
EP3196301B1 (de) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur behandlung von monogener krankheiten
US9683268B2 (en) 2012-09-19 2017-06-20 Beth Israel Deaconess Viruses associated with immunodeficiency and enteropathy and methods using same
WO2014045674A1 (ja) 2012-09-19 2014-03-27 国立大学法人東京大学 神経活動依存的プロモータ活性を有するdna、及びこれを含むベクター
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
US9066966B2 (en) 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
WO2014153083A1 (en) 2013-03-14 2014-09-25 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Compositions and methods for predicting age of onset of a lysosomal storage disease or a disease associated with a lysosomal defect
AU2014227766B2 (en) 2013-03-15 2018-10-04 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV vectors
AU2014274457B2 (en) 2013-05-21 2019-10-24 University Of Florida Research Foundation, Inc. Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3024498B1 (de) 2013-07-22 2019-12-04 The Children's Hospital of Philadelphia Aav-variante und zusammensetzungen, verfahren und verwendungen für gentransfer zu zellen, organen und gewebe
DE102013215817A1 (de) 2013-08-09 2015-02-12 Universitätsklinikum Hamburg-Eppendorf Neue peptide mit spezifität für die lunge
EP3561062A1 (de) 2013-09-13 2019-10-30 California Institute of Technology Selektive rückgewinnung
US9710484B2 (en) * 2013-09-17 2017-07-18 Here Global B.V. Method and apparatus for associating physical locations to online entities
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
US20160256571A1 (en) 2013-09-30 2016-09-08 Sanofi Invention
CA3182790A1 (en) 2013-10-11 2015-04-16 Massachusetts Eye & Ear Infirmary Ancestral adeno-associated virus sequences and uses thereof
CN104592364B (zh) * 2013-10-30 2018-05-01 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
US20170007720A1 (en) 2014-02-21 2017-01-12 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
HUE054768T2 (hu) 2014-05-02 2021-09-28 Genzyme Corp AAV vektorok retina és CNS génterápiára
AU2015265412B2 (en) 2014-05-28 2021-03-25 Evogene Ltd. Isolated polynucleotides, polypeptides and methods of using same for increasing abiotic stress tolerance, biomass and yield of plants
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
SI3766916T1 (sl) 2014-06-25 2023-01-31 Acuitas Therapeutics Inc. Formulacije novih lipidov in lipidnih nanodelcev za dostavo nukleinskih kislin
US9818383B2 (en) 2014-07-28 2017-11-14 University Of Connecticut Methods and systems for non-destructive analysis of objects and production of replica objects
KR102526711B1 (ko) 2014-09-24 2023-04-27 시티 오브 호프 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
EP3209311B1 (de) 2014-10-21 2024-03-06 University of Massachusetts Rekombinante aav-varianten und verwendungen davon
WO2016115382A1 (en) 2015-01-14 2016-07-21 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2016115503A1 (en) 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
GB201502306D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
GB201502305D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
US20180135074A1 (en) 2015-02-19 2018-05-17 University Of Florida Research Foundation, Incorporated Recombinant aav vectors for gene therapy of human hematopoietic disorders
CN107405507B (zh) 2015-03-02 2022-05-03 阿德夫拉姆生物技术股份有限公司 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
EP3273999A1 (de) 2015-03-23 2018-01-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren und pharmazeutische zusammensetzung zur behandlung und prävention von neurologischem phänotyp im zusammenhang mit friedreich-ataxie
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
ES2755725T3 (es) 2015-04-08 2020-04-23 Us Health Terapia génica viral como tratamiento para una enfermedad o un trastorno por almacenamiento de colesterol
WO2016172155A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
WO2016179644A1 (en) 2015-05-08 2016-11-17 Children's Medical Research Institute Promoters for expression of heterologous genes
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP3294894B8 (de) 2015-05-12 2019-09-25 The U.S.A. as represented by the Secretary, Department of Health and Human Services Aav-isolat und fusionsprotein mit nervenwachstumsfaktor-signalpeptid und parathormon
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
EP3325494B1 (de) 2015-07-19 2020-12-30 Yeda Research and Development Co., Ltd. Selektive inhibitoren der alpha2-haltigen isoformen der na, k-atpase und deren verwendung zur reduzierung des augeninnendruckes
US20180214576A1 (en) 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
WO2017019994A2 (en) 2015-07-30 2017-02-02 Massachusetts Eye And Ear Infirmary Ancestral virus sequences and uses thereof
WO2017058892A2 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
RU2021102893A (ru) 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
RU2766583C2 (ru) 2015-12-01 2022-03-15 Спарк Терапьютикс, Инк. Масштабируемые способы получения рекомбинантного вектора на основе аденоассоциированного вируса (aav) в системе бессывороточной суспензионной культуры клеток, подходящего для клинического применения
EP3384034B1 (de) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Neue rekombinante kapside des adeno-associated virus mit erhöhtem tropismus für menschliche skelett- muskulatur
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
BR112018011881A2 (pt) 2015-12-14 2018-12-04 The University Of North Carolina At Chapel Hill proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
US11060088B2 (en) 2016-02-12 2021-07-13 University Of Massachusetts Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization
EP3417055B1 (de) 2016-02-16 2021-10-13 The Board of Trustees of the Leland Stanford Junior University Gegen vorhandene humane neuartige antikörper resistente, neuartige rekombinante adeno-assoziierte virus-kapside
CN108697774A (zh) 2016-02-22 2018-10-23 北卡罗来纳大学教堂山分校 治疗mps i-相关失明的aav-idua载体
WO2017180854A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Novel aav8 mutant capsids and compositions containing same
WO2017192750A1 (en) 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
FI3445773T3 (fi) 2016-05-13 2023-03-30 4D Molecular Therapeutics Inc Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
JP7153009B2 (ja) 2016-07-21 2022-10-13 スパーク セラピューティクス インコーポレイテッド 高収率の組換えアデノ随伴ウイルス（ｒＡＡＶ）ベクターを生成するための、規模拡大可能な高回収率の方法、及びそれにより生成される組換えアデノ随伴ウイルス（ｒＡＡＶ）ベクター
EP3491008A2 (de) 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Neuartige adeno-assoziierte virus-kapsid-proteine
CA3033125A1 (en) 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
CN109923211A (zh) 2016-09-08 2019-06-21 蓝鸟生物公司 Pd-1归巢核酸内切酶变体、组合物及使用方法
US11078238B2 (en) 2016-09-29 2021-08-03 University Of Florida Research Foundation, Incorporated AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties
EP3528785A4 (de) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. Modifizierte aav-kaspide und verwendungen davon
US20180182497A1 (en) 2016-12-22 2018-06-28 DxRx, Inc. Creating engagement with an inner circle social network in substance abuse treatment
EA038695B1 (ru) 2017-02-15 2021-10-06 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Способы и композиции для переноса генов по сосудистой сети
LT3589730T (lt) 2017-02-28 2024-03-12 The Trustees Of The University Of Pennsylvania Adenoasocijuoto viruso (aav) monofiletinės grupės f vektorius, ir jo panaudojimo būdai
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
AU2018234695B2 (en) 2017-03-15 2024-10-03 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
US20210254056A1 (en) 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
JP7330899B2 (ja) 2017-05-10 2023-08-22 マサチューセッツ アイ アンド イヤー インファーマリー ウイルスのアセンブリ活性化タンパク質（ａａｐ）依存性を改変する方法および組成物
US11680275B2 (en) 2017-06-06 2023-06-20 University Of Massachusetts Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome
WO2018237066A1 (en) 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services CODON OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF C1-TYPE NIEMANN-PICK DISEASE AND ASSOCIATED STATES
EP3645021A4 (de) 2017-06-30 2021-04-21 Intima Bioscience, Inc. Adeno-assoziierte virale vektoren zur gentherapie
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
US20200255859A1 (en) 2017-07-31 2020-08-13 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
SG11202006056RA (en) 2018-01-17 2020-07-29 Meiragtx Uk Ii Ltd A modified raav capsid protein for gene therapy
EP3758724A4 (de) 2018-02-27 2022-07-06 The Trustees of The University of Pennsylvania Neuartige vekrtoren von adeno-assoziiertem virus (aav), aav-vektoren mit reduzierter kapsiddeamidierung und verwendungen davon
CN112236443A (zh) 2018-02-27 2021-01-15 宾夕法尼亚州大学信托人 新颖腺相关病毒(aav)载体、具有降低的衣壳脱酰胺化的aav载体及其用途
JP7393766B2 (ja) 2018-02-28 2023-12-07 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 抗体回避ウイルスベクターのための方法および組成物
JP2021514659A (ja) 2018-03-06 2021-06-17 ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド Ａａｖキメラ
EP3762500A1 (de) 2018-03-06 2021-01-13 Voyager Therapeutics, Inc. Aus insektenzelle hergestellte teilweise selbstkomplementäre aav-genome
SG11202008687XA (en) 2018-03-16 2020-10-29 Res Inst Nationwide Childrens Hospital Increasing tissue specific gene delivery by capsid modification
EP3773743A1 (de) 2018-04-03 2021-02-17 Stridebio, Inc. Virusvektoren gegen ophthalmisches gewebe
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
MX2020010466A (es) * 2018-04-03 2021-01-08 Vectores de virus que evitan anticuerpos.
SG11202105326WA (en) 2018-11-21 2021-06-29 Stridebio Inc Recombinant viral vectors and nucleic acids for producing the same
PL3906066T3 (pl) 2019-01-04 2024-09-02 Ultragenyx Pharmaceutical Inc. Konstrukty do terapii genowej do leczenia choroby wilsona
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
MX2021013913A (es) 2019-05-14 2022-03-17 Univ Duke Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa.
CN114585743A (zh) 2019-10-17 2022-06-03 斯特里迪比奥公司 Aav转移盒
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터

Also Published As

Publication number Publication date
KR20220011616A (ko) 2022-01-28
AU2020241888A1 (en) 2021-09-30
TW202102525A (zh) 2021-01-16
US20240067986A1 (en) 2024-02-29
AR118465A1 (es) 2021-10-06
EP3941929A1 (de) 2022-01-26
US11981914B2 (en) 2024-05-14
CO2021013878A2 (es) 2022-01-17
CA3133453A1 (en) 2020-09-24
CN113727992A (zh) 2021-11-30
US20220064675A1 (en) 2022-03-03
WO2020191300A1 (en) 2020-09-24
BR112021018776A2 (pt) 2021-11-30
IL286464A (en) 2021-12-01
EA202192501A1 (ru) 2021-12-16
JP2022525955A (ja) 2022-05-20
MX2021011468A (es) 2021-12-15

Similar Documents

Publication Publication Date Title
IL286464A (en) A virus of vectors related to recombinant DNA
IL286911A (en) Recombinant adeno-associated viruses and their uses
IL283546A (en) A recombinant adeno-associated virus vector for gene delivery
IL277666A (en) Virus vector evasion antibody
IL276859A (en) Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses thereof
IL276860A (en) Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses thereof
IL277664A (en) Virus vector evasion antibody
HK1251612A1 (zh) 包含2a肽的重組載體
SG11202011145UA (en) Liver targeting adeno-associated viral vectors
IL282885A (en) AAV viral vectors and their uses
EP3221456A4 (de) Genommodifizierte rekombinante adeno-assoziierte virusvektoren
EP4061427A4 (de) Adeno-assoziierte virusvektorvarianten
IL268291A (en) Multiple transgene recombinant adenovirus
IL289489A (en) A chemically modified adeno-associated virus
ZA202100859B (en) Recombinant protein variants
IL288419A (en) Recombinant viral herpes vector
IL286475A (en) Methods for the production of recombinant viral vectors
ZA201904705B (en) Recombinant bcg overexpressing phop-phor
IL288412A (en) peptides
IL272655A (en) Recombinant adeno-associated vectors
IL285639A (en) Adeno-associated virus vectors for drug delivery
IL284006A (en) Mitochondria-targeted peptides
EP3710046A4 (de) Rekombinante vektoren, die für proteinuntereinheiten des zika-virus codieren
EP3751000A4 (de) Verbesserter adeno-assoziierter virusvektor
IL290019A (en) Peptides to regulate melanocytes