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)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (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 (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
IL295529A (en) 2020-02-13 2022-10-01 Tenaya Therapeutics Inc Gene therapy vectors to treat heart disease
BR112022023106A2 (pt) 2020-05-13 2023-01-17 Voyager Therapeutics Inc Redirecionamento de tropismo de capsídeos de aav
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
WO2023283649A1 (en) 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
TW202342759A (zh) * 2022-02-04 2023-11-01 美商史崔德生物公司 重組腺相關病毒載體及其使用方法
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
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies

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
ES2216005T3 (es) 1993-11-09 2004-10-16 Targeted Genetics Corporation Produccion de titulos elevados de vectores de aav recombinantes.
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
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
JP4002605B2 (ja) 1995-06-07 2007-11-07 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 筋組織のａａｖ形質導入
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
AU730305B2 (en) 1995-12-15 2001-03-01 Intronn Llc Therapeutic molecules generated by trans-splicing
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services 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
AU8286998A (en) 1997-07-03 1999-01-25 United States Of America, Represented By The Secretary, Department Of Health And Human Services, The Genes for niemann-pick type c disease
ES2313784T3 (es) 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
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
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
EP1115880A2 (de) 1998-09-22 2001-07-18 University of Florida Methoden zur herstellung von rekombinanten aav-vektoren in grossem massstab
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
JP4573437B2 (ja) 1998-11-05 2010-11-04 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血清型１核酸配列、ベクターおよび同一物を含有する宿主細胞
JP2002538770A (ja) 1998-11-10 2002-11-19 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル ウイルスベクターとその製造及び投与の方法
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
EP1033405A3 (de) 1999-02-25 2001-08-01 Ceres Incorporated DNS-fragmente mit bestimmter Sequenz und die dadurch kodierte Polypeptide
EP1887081A2 (de) 1999-02-25 2008-02-13 Ceres Incorporated DNS-Sequenzen
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
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
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
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
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
ATE403715T1 (de) 1999-08-09 2008-08-15 Targeted Genetics Corp Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
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
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
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
CA2407352A1 (en) 2000-04-21 2001-11-01 Corixa Corporation Compositions and methods for the therapy and diagnosis of acne vulgaris
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina 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
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
EP1437938A2 (de) 2001-04-30 2004-07-21 Cell Genesys, Inc. Virenvermittelte verabreichung und in-vivo-expression von polynukleotiden, die antiangione proteine codieren
EP1402042A2 (de) 2001-06-22 2004-03-31 Syngenta Participations AG Auf abiotischen stress ansprechende polynukleotide und polypeptide
AU2002345250A1 (en) 2001-06-22 2003-01-08 Syngenta Participations Ag Plant disease resistance genes
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
WO2002010210A2 (en) 2001-08-28 2002-02-07 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
EP1572893B1 (de) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Produktion von adenoassoziiertem virus in insektenzellen
DK1310571T3 (da) 2001-11-13 2006-06-19 Univ Pennsylvania Fremgangsmåde til identifikation af ukendte adeno-associerede virussekvenser (AAV-sekvenser) og et kit til fremgangsmåden
EP1463805B1 (de) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Sequenzen des adeno-assoziierten virus (aav) serotyp 9, diese enthaltende vektoren und verwendungen dafür
EP1453547B1 (de) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-assoziierte virus (aav) serotyp 8 sequenzen, diese enthaltende vektoren und ihre verwendung
AU2003223766A1 (en) 2002-04-30 2003-11-17 University Of North Carolina At Chapel Hill Secretion signal vectors
NZ536274A (en) 2002-05-01 2008-04-30 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
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
AU2003299817A1 (en) 2002-12-20 2004-07-22 Applera Corporation Genetic polymorphisms associated with stenosis, methods of detection and uses thereof
CA2921578C (en) 2002-12-24 2017-02-14 Rinat Neuroscience Corp. Anti-ngf antibodies and methods using same
ES2712695T3 (es) 2003-06-02 2019-05-14 Univ Massachusetts Métodos y composiciones para controlar la eficacia de la silenciación del ARN
ES2629087T3 (es) 2003-06-19 2017-08-07 Genzyme Corporation Viriones de AAV con inmunorreactividad reducida y usos de los mismos
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
WO2009105612A2 (en) 2008-02-20 2009-08-27 Ceres, Inc. Nucleotide sequences and corresponding polypeptides conferring improved nitrogen use efficiency characteristics in plants
ES2411479T3 (es) 2003-09-30 2013-07-05 The Trustees Of The University Of Pennsylvania Clados de virus adeno-asociados (AAV), secuencias, vectores que los contienen, y usos de los mismos
US20060107345A1 (en) 2003-09-30 2006-05-18 Nickolai Alexandrov Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
KR20060135060A (ko) 2004-04-07 2006-12-28 리나트 뉴로사이언스 코퍼레이션 신경성장인자 길항제의 투여에 의한 골암 통증의 치료방법
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
CA2579519A1 (en) 2004-09-09 2006-03-16 The General Hospital Corporation Modulating phosphatase activity in cardiac cells
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
US20080213759A1 (en) 2005-01-05 2008-09-04 Shionogi & Co., Ltd. Novel Angiogenesis Inhibitor
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
EP2478760A1 (de) 2005-05-10 2012-07-25 Monsanto Technology LLC Gene und ihre Verwendung zur Pflanzenverbesserung
WO2007011682A2 (en) 2005-07-15 2007-01-25 Regents Of The University Of California 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
WO2007092563A2 (en) 2006-02-08 2007-08-16 Genzyme Corporation Gene therapy for niemann-pick disease type a
US7989606B2 (en) 2006-02-10 2011-08-02 The University Of Cincinnati Phosphatase inhibitor protein-1 as a regulator of cardiac function
EP2007795B1 (de) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav-capsid-proteine
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
CN101495624A (zh) 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 衣壳免疫原性降低的经修饰aav载体及其用途
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
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
EP2191001B1 (de) 2007-04-09 2016-06-08 University of Florida Research Foundation, Inc. Raav-vektorzusammensetzungen mit tyrosin-modifizierten capsidproteinen und verwendungsverfahren
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
EP2193201A1 (de) 2007-09-18 2010-06-09 BASF Plant Science GmbH Pflanzen mit gesteigertem 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
WO2009108274A2 (en) 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
ES2538468T3 (es) 2008-05-20 2015-06-22 Eos Neuroscience, Inc. Vectores para la administración de proteínas sensibles a la luz y métodos para su utilización
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
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
PT3421603T (pt) 2009-05-02 2022-01-10 Genzyme Corp Terapia génica para distúrbios neurodegenerativos
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US20120252877A1 (en) 2009-08-14 2012-10-04 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
ES2698203T3 (es) 2010-04-23 2019-02-01 Univ Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
US8927514B2 (en) 2010-04-30 2015-01-06 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
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2634253B1 (de) 2010-10-27 2016-05-11 Jichi Medical University Adeno-assoziierte virus-virionen zur übertragung von genen in nervenzellen
ES2684307T3 (es) 2010-11-05 2018-10-02 The Board Of Trustees Of The Leland Stanford Junior University Proteínas estabilizadas de tipo opsina de función escalonada y métodos de uso de las mismas
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
US9409953B2 (en) 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
CA2827375C (en) 2011-02-14 2022-07-19 The Children's Hospital Of Philadelphia Improved 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
WO2013016315A1 (en) 2011-07-28 2013-01-31 Trustees Of Dartmouth College Methods for treating niemann-pick type c disease
BR112014004130A8 (pt) 2011-08-23 2022-05-17 Evogene Ltd Polinucleotídeos e polipeptídeos isolados e métodos de uso dos mesmos para o aumento da prooução da planta e/ou características agrícolas
US10640785B2 (en) 2011-11-22 2020-05-05 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
CN108929880A (zh) 2012-03-27 2018-12-04 库瑞瓦格股份公司 包含5′toputr的人工核酸分子
EP2839014B1 (de) 2012-04-18 2020-12-16 The Children's Hospital of Philadelphia Zusammensetzung und verfahren für hocheffizienten gentransfer mittels aav-kapsidvarianten
EP2660325A3 (de) 2012-05-02 2014-02-12 Christian Medical College AAV-Vektoren und dazugehörige Nukleotidsequenzen und Verfahren
JP6385920B2 (ja) 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
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
JP6417322B2 (ja) 2012-06-21 2018-11-07 アソシアシオン・アンスティテュ・ドゥ・ミオロジーＡｓｓｏｃｉａｔｉｏｎ Ｉｎｓｔｉｔｕｔ Ｄｅ Ｍｙｏｌｏｇｉｅ 遺伝子治療ベクターの広範な遺伝子送達
IN2015KN00074A (de) 2012-07-06 2015-07-31 Univ Iowa Res Found
AU2013289206B2 (en) 2012-07-11 2018-08-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
WO2014047261A1 (en) 2012-09-19 2014-03-27 Beth Israel Deaconess Medical Center, Inc. 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
US9636370B2 (en) 2012-09-28 2017-05-02 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
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
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US9983200B2 (en) 2013-03-14 2018-05-29 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
IL293294B2 (en) 2013-03-15 2023-04-01 Univ North Carolina Chapel Hill Methods and compounds for ligation of paired aav glycan vectors
EP2999791B1 (de) 2013-05-21 2018-12-05 University of Florida Research Foundation, Inc. Kapsidmodifizierte, raav3-vektorzusammensetzungen und verfahren zur verwendung in der gentherapie von leberkrebs bei menschen
WO2014194132A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Modified Aav and preparations, methods and uses for gene transfer to cells, organs and tissues
DE102013215817A1 (de) 2013-08-09 2015-02-12 Universitätsklinikum Hamburg-Eppendorf Neue peptide mit spezifität für die lunge
EP3044318B1 (de) * 2013-09-13 2019-05-01 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
JP6985795B2 (ja) 2013-09-26 2021-12-22 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー
US20160256571A1 (en) 2013-09-30 2016-09-08 Sanofi Invention
ES2857751T3 (es) 2013-10-11 2021-09-29 Massachusetts Eye & Ear Infirmary Métodos para predecir secuencias de virus ancestrales y usos de los mismos
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
WO2015126972A1 (en) 2014-02-21 2015-08-27 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
US10746742B2 (en) 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
PE20170260A1 (es) 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
US20170088852A1 (en) 2014-05-28 2017-03-30 Evogene Ltd. Isolated polynucleotides, polypeptides and methods of using same for increasing abiotic stress tolerance, biomass and yield of plants
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
RS63848B1 (sr) 2014-06-25 2023-01-31 Acuitas Therapeutics Inc Novi lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina
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
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
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
JP6938377B2 (ja) 2015-01-14 2021-09-22 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 標的化遺伝子移入のための方法および組成物
CA2970730A1 (en) 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
GB201502305D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
GB201502306D0 (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
SG11201707063TA (en) 2015-03-02 2017-09-28 Adverum Biotechnologies Inc Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
WO2016150964A1 (en) 2015-03-23 2016-09-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia
CN107532177A (zh) 2015-03-24 2018-01-02 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
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
CA2982129A1 (en) 2015-04-08 2016-10-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
EP3285788A4 (de) 2015-04-23 2018-12-05 University of Massachusetts Modulation der transgenexpression eines aav-vektors
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
WO2016183297A1 (en) 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
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
EP3907231A1 (de) 2015-07-19 2021-11-10 Yeda Research and Development Co., Ltd. Selektive inhibitoren der alpha2-haltigen isoformen der na, k-atpase und deren verwendung zur reduzierung des augeninnendruckes
CA2988471A1 (en) 2015-07-28 2017-02-02 University Of Massachusetts Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector
AU2016298394C1 (en) 2015-07-30 2024-02-15 Massachusetts Eye And Ear Infirmary Ancestral virus sequences and uses thereof
IL257628B2 (en) * 2015-09-28 2023-04-01 Univ North Carolina Chapel Hill Methods and compositions for stealth virus antibody 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
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
KR102200642B1 (ko) 2015-11-05 2021-01-12 뱀부 테라퓨틱스 인코포레이티드 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터
SG11201804400SA (en) 2015-12-01 2018-06-28 Spark Therapeutics Inc Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
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
ES2869284T3 (es) 2015-12-11 2021-10-25 California Inst Of Techn Péptidos de diana para dirigir virus adenoasociados (AAV)
MX2018007237A (es) 2015-12-14 2018-12-11 Univ North Carolina Chapel Hill Proteinas de cápside modificadas para un suministro mejorado de vectores de parvovirus.
EP3413928B1 (de) 2016-02-12 2022-04-20 University of Massachusetts Anti-angiogene mirna-therapeutika zur hemmung der neovaskularisation der hornhaut
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
WO2017147123A1 (en) 2016-02-22 2017-08-31 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i-associated blindness
SG11201808812RA (en) 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
US11866462B2 (en) 2016-05-04 2024-01-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
PT3445773T (pt) 2016-05-13 2023-03-13 4D Molecular Therapeutics Inc Cápsides variantes de vírus adeno-associado e métodos de utilização das mesmas
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc modulatory polynucleotides
CN109790549A (zh) 2016-07-21 2019-05-21 星火治疗有限公司 生产高产重组腺相关病毒(rAAV)载体可扩展高回收法及其产生的重组腺相关病毒(rAAV)载体
CN109715650B (zh) 2016-07-26 2024-03-08 生物马林药物股份有限公司 新颖腺相关病毒衣壳蛋白
US20190262373A1 (en) 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3510157B1 (de) 2016-09-08 2023-05-31 2seventy bio, Inc. Pd-1-homing-endonukleasevarianten, zusammensetzungen und verfahren zur verwendung
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
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
WO2018119330A2 (en) 2016-12-22 2018-06-28 Oregon Health & Science University Adeno associated viral vectors
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
SG10201912976WA (en) 2017-02-28 2020-02-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
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
CA3054711A1 (en) 2017-03-15 2018-09-20 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
WO2018204764A1 (en) 2017-05-05 2018-11-08 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
WO2018209154A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (app)-dependence of viruses
MX2019014760A (es) 2017-06-06 2020-08-03 Univ Massachusetts Vectores de aav autoregulables para la expresión segura de mecp2 en el síndrome de rett.
EP3642345A1 (de) 2017-06-20 2020-04-29 The U.S.A. As Represented By The Secretary, Department Of Health And Human Services Codon-optimierte humane npc1-gene zur behandlung von morbus niemann-pick typ c1 und verwandten erkrankungen
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
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
AU2018311504A1 (en) 2017-07-31 2020-03-12 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
MX2020006764A (es) 2018-01-17 2021-01-08 Meiragtx Uk Ii Ltd Una proteína de la cápside de raav modificada para terapia génica.
WO2019168961A1 (en) 2018-02-27 2019-09-06 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
BR112020017278A2 (pt) 2018-02-27 2020-12-22 The Trustees Of The University Of Pennsylvania Vetores de vírus adenoassociado (aav), vetores de aav tendo desamidação de capsídeos reduzida e usos para os mesmos
AU2019227896A1 (en) 2018-02-28 2020-08-20 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2019173434A1 (en) 2018-03-06 2019-09-12 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
KR20200130337A (ko) 2018-03-06 2020-11-18 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 Aav 키메라
AU2019234918A1 (en) 2018-03-16 2020-09-17 Research Institute At Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
EA202092362A1 (ru) 2018-04-03 2021-01-11 Страйдбайо, Инк. Вирусные векторы для нацеливания на ткани глаза
WO2019195444A1 (en) * 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
EP3883954A4 (de) 2018-11-21 2022-08-10 Stridebio, Inc. Rekombinante virale vektoren und nukleinsäuren zur herstellung davon
KR20210112339A (ko) 2019-01-04 2021-09-14 울트라제닉스 파마수티컬 인코포레이티드 윌슨병을 치료하기 위한 유전자 요법 구축물
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
AU2020274200A1 (en) 2019-05-14 2021-11-25 Duke University Compositions and methods for the treatment of ATPase-mediated diseases
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease
US20240131185A1 (en) 2019-10-17 2024-04-25 Stridebio, Inc. Aav transfer cassette
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome

Also Published As

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

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
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
HK1251612A1 (zh) 包含2a肽的重組載體
SG11202011145UA (en) Liver targeting adeno-associated viral vectors
IL277666A (en) Virus vector evasion antibody
EP3221456A4 (de) Genommodifizierte rekombinante adeno-assoziierte virusvektoren
IL282885A (en) AAV viral vectors and their uses
EP4061427A4 (de) Adeno-assoziierte virusvektorvarianten
IL277664A (en) Virus vector evasion antibody
IL268291A (en) Multiple transgene recombinant adenovirus
ZA202100859B (en) Recombinant protein variants
IL288419A (en) Recombinant viral herpes vector
IL289489A (en) A chemically modified adeno-associated virus
ZA201904705B (en) Recombinant bcg overexpressing phop-phor
IL288412A (en) peptides
IL272655A (en) Recombinant adeno-associated vectors
IL286475A (en) Methods for the production of recombinant viral vectors
IL285639A (en) Adeno-associated virus vectors for drug delivery
EP3710046A4 (de) Rekombinante vektoren, die für proteinuntereinheiten des zika-virus codieren
EP3751000A4 (de) Verbesserter adeno-assoziierter virusvektor
IL290019A (en) Peptides to regulate melanocytes
GB201906982D0 (en) Peptides