[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

EA202092362A1 - Вирусные векторы для нацеливания на ткани глаза - Google Patents

Вирусные векторы для нацеливания на ткани глаза

Info

Publication number
EA202092362A1
EA202092362A1 EA202092362A EA202092362A EA202092362A1 EA 202092362 A1 EA202092362 A1 EA 202092362A1 EA 202092362 A EA202092362 A EA 202092362A EA 202092362 A EA202092362 A EA 202092362A EA 202092362 A1 EA202092362 A1 EA 202092362A1
Authority
EA
Eurasian Patent Office
Prior art keywords
viral vectors
eye tissue
targeting
aav capsid
targeting eye
Prior art date
Application number
EA202092362A
Other languages
English (en)
Inventor
Дэниел Маккой
Гаррет И. Бэрри
Original Assignee
Страйдбайо, Инк.
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 Страйдбайо, Инк. filed Critical Страйдбайо, Инк.
Publication of EA202092362A1 publication Critical patent/EA202092362A1/ru

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

В настоящем изобретении предложены капсидные белки AAV, содержащие модификацию в аминокислотной последовательности, и вирусные векторы, содержащие модифицированный капсидный белок AAV. В изобретении также предложены способы введения вирусных векторов и вирусных капсидов по изобретению в клетку или субъекту in vivo.
EA202092362A 2018-04-03 2019-04-03 Вирусные векторы для нацеливания на ткани глаза EA202092362A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862652108P 2018-04-03 2018-04-03
PCT/US2019/025584 WO2019195423A1 (en) 2018-04-03 2019-04-03 Virus vectors for targeting ophthalmic tissues

Publications (1)

Publication Number Publication Date
EA202092362A1 true EA202092362A1 (ru) 2021-01-11

Family

ID=66397437

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202092362A EA202092362A1 (ru) 2018-04-03 2019-04-03 Вирусные векторы для нацеливания на ткани глаза

Country Status (13)

Country Link
US (1) US12116384B2 (ru)
EP (1) EP3773743A1 (ru)
JP (2) JP7425738B2 (ru)
KR (1) KR20210006358A (ru)
CN (1) CN112533644A (ru)
AU (1) AU2019247191A1 (ru)
BR (1) BR112020020341A2 (ru)
CA (1) CA3094465A1 (ru)
EA (1) EA202092362A1 (ru)
IL (1) IL277667A (ru)
MX (1) MX2020010465A (ru)
SG (1) SG11202009450SA (ru)
WO (1) WO2019195423A1 (ru)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，インコーポレイテッド 抗体を回避するウイルスベクター
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
EA202092362A1 (ru) 2018-04-03 2021-01-11 Страйдбайо, Инк. Вирусные векторы для нацеливания на ткани глаза
MA51353B1 (fr) * 2018-04-05 2022-09-30 Univ Sorbonne Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit
JP2022525955A (ja) 2019-03-21 2022-05-20 ストライドバイオ，インコーポレイテッド 組換えアデノ随伴ウイルスベクター
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
EP4045525A1 (en) 2019-10-17 2022-08-24 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
US11400039B2 (en) * 2020-01-16 2022-08-02 James W. Hill Changing eye color by gene transduction
MX2022013963A (es) * 2020-05-05 2023-02-22 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas.
WO2022018516A1 (en) * 2020-07-21 2022-01-27 Inspirar Limited Composition and method for treating eye diseases
US20230304038A1 (en) 2020-08-19 2023-09-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
TW202242124A (zh) 2021-01-14 2022-11-01 美商史崔德生物公司 靶向ｔ細胞之ａａｖ載體
CA3234664A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
CN117801116A (zh) * 2022-09-30 2024-04-02 上海玮美基因科技有限责任公司 一种融合型新型腺相关病毒及其应用
WO2024124019A2 (en) 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells

Family Cites Families (253)

* 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
WO1988002026A1 (en) 1986-09-08 1988-03-24 Applied Biotechnology, Inc. Empty viral capsid vaccines
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
JPH04501719A (ja) 1988-11-10 1992-03-26 インペリアル・キヤンサー・リサーチ・テクノロジー・リミテツド ポリペプチド
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
CA2176117C (en) 1993-11-09 2006-01-03 Terence R. Flotte Generation of high titers of recombinant aav vectors
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
EP0755454B1 (en) 1994-04-13 2008-02-13 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
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
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
EP0883344B2 (en) 1995-12-15 2010-06-09 VIRxSYS Corporation 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
WO1999001555A1 (en) 1997-07-03 1999-01-14 The United States Of America Represented By The Secretary, 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
DE69939169D1 (de) 1998-05-28 2008-09-04 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
AU774706B2 (en) 1998-09-22 2004-07-08 Johns Hopkins University, The Methods for large-scale production of recombinant AAV vectors
GB9822763D0 (en) 1998-10-20 1998-12-16 Univ Sheffield Immunoglobin variant
DK1127150T3 (da) 1998-11-05 2007-09-24 Univ Pennsylvania Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse
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
DE69941905D1 (de) 1998-11-10 2010-02-25 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
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 (en) 1999-02-25 2001-08-01 Ceres Incorporated Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
EP1887081A2 (en) 1999-02-25 2008-02-13 Ceres Incorporated DNA Sequences
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
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
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
US20110214206A1 (en) 1999-05-06 2011-09-01 La Rosa Thomas J Nucleic acid molecules and other molecules associated with plants
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
DK1204739T3 (da) 1999-08-09 2008-10-20 Targeted Genetics Corp Forögelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar
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
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
CA2410901C (en) 2000-06-01 2012-03-20 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
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
WO2002088173A2 (en) 2001-04-30 2002-11-07 Cell Genesys, Inc. Viral-mediated delivery and in vivo expression of polynucleotides encoding anti-angiogenic proteins
US7777097B2 (en) 2001-06-22 2010-08-17 Syngenta Participations Ag Plant disease resistance genes
AU2002341541A1 (en) 2001-06-22 2003-03-03 Syngenta Participations Ag Abiotic stress responsive polynucleotides and polypeptides
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
CA2467959C (en) 2001-11-09 2009-03-10 Robert M. Kotin Production of adeno-associated virus in insect cells
KR101015854B1 (ko) 2001-11-13 2011-02-23 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아데노-결합 바이러스 (ａａｖ) 서열을 검출 및/또는확인하는 방법 및 그 방법에 의해 확인된 신규한 서열을분리하는 방법
WO2003052051A2 (en) 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
PT2573170T (pt) 2001-12-17 2018-03-26 Univ Pennsylvania Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações
US7071172B2 (en) 2002-04-30 2006-07-04 The 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
CA2510895A1 (en) 2002-12-20 2004-07-15 Applera Corporation Genetic polymorphisms associated with stenosis, methods of detection and uses thereof
ES2697876T3 (es) 2002-12-24 2019-01-29 Rinat Neuroscience Corp Anticuerpos anti-NGF y procedimientos de uso de los mismos
EP3502252B1 (en) 2003-06-02 2023-04-05 University of Massachusetts Methods and compositions for controlling efficacy of rna silencing
HUE034546T2 (en) 2003-06-19 2018-02-28 Genzyme Corp Reduced immunoreactivity of AAV virions and their applications
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
AU2005282352B2 (en) 2004-09-09 2011-12-01 The General Hospital Corporation Modulating phosphatase activity in cardiac cells
CA2591544A1 (en) 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Chimeric vectors
US20080213759A1 (en) 2005-01-05 2008-09-04 Shionogi & Co., Ltd. Novel Angiogenesis Inhibitor
EP2194140A2 (en) 2005-03-02 2010-06-09 Metanomics GmbH Process for the production of fine chemicals
US20140199313A1 (en) 2005-03-02 2014-07-17 Metanomics Gmbh Process for the Production of Fine Chemicals
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
CN101213203A (zh) 2005-04-29 2008-07-02 教堂山北卡罗莱纳州大学 在转录后水平调节核酸表达的方法和组合物
WO2006138005A2 (en) 2005-05-10 2006-12-28 Monsanto Technology, Llc Genes and uses for plant improvement
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
US20090191597A1 (en) 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. 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
AU2007221470B2 (en) 2006-02-10 2013-02-14 The University Of Cincinnati Phosphatase inhibitor Protein-1 as a regulator of cardiac function
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
ES2400235T3 (es) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania Método de producción escalable de AAV
WO2008027084A2 (en) 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
DK3023500T3 (da) 2006-06-21 2020-04-20 Uniqure Ip Bv Insektceller til fremstilling af AAV-vektorer
EP2125006B1 (en) 2007-01-18 2013-10-16 University of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
WO2008124724A1 (en) 2007-04-09 2008-10-16 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for 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
AU2008300579B2 (en) 2007-09-18 2014-11-13 Basf Plant Science Gmbh Plants with increased yield
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
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
BRPI0907786B1 (pt) 2008-02-20 2021-06-01 Ceres, Inc. Método de produção de uma planta e método de aumentar o nível de tolerância a baixo teor de nitrogênio em uma planta
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
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
EP2396343B1 (en) 2009-02-11 2017-05-17 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
BRPI1010868A2 (pt) 2009-05-02 2018-06-12 Genzyme Corp terapia gênica para distúrbios neurodegenerativos
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 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
ES2605305T3 (es) 2010-04-23 2017-03-13 University Of Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
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
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
CN107828820B (zh) 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
CN105941328B (zh) 2010-11-05 2019-04-09 斯坦福大学托管董事会 一种鉴定抑制前额叶皮质中的兴奋性或抑制性神经元的去极化的化合物的系统
US10415056B2 (en) 2010-11-10 2019-09-17 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
CA2826273C (en) 2011-02-10 2021-11-02 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
EP2675484B1 (en) 2011-02-14 2018-05-30 The Children's Hospital of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
EP3693025B1 (en) * 2011-04-22 2021-10-13 The Regents of The University of California Adeno-associated virus virions with variant capsid 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
EP2723393A4 (en) 2011-06-24 2015-02-25 Ct Se Llc EXTRACELLULAR TARGETED CONSTITUENT CONJUGATES
US9206425B2 (en) 2011-07-28 2015-12-08 Trustees Of Dartmouth College Methods for treating Niemann-Pick type C disease
CA3177742A1 (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
AU2012340567B2 (en) 2011-11-22 2017-11-23 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
ES2660129T3 (es) 2012-03-27 2018-03-20 Curevac Ag Moléculas de ácido nucleico artificiales que comprenden un 5'UTR-TOP
US9909142B2 (en) 2012-04-18 2018-03-06 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
EP2660325A3 (en) 2012-05-02 2014-02-12 Christian Medical College AAV vectors and corresponding nucleotide sequences and methods
WO2013170078A1 (en) 2012-05-09 2013-11-14 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 (en) 2012-05-22 2012-08-29 Laboratorios Del. Dr. Esteve, S.A. Methods for the production of vectors
EP2864488A1 (en) 2012-06-21 2015-04-29 Association Institut de Myologie Widespread gene delivery of gene therapy vectors
EP2870237B1 (en) 2012-07-06 2018-09-26 University of Iowa Research Foundation Modified adeno-associated virus vector compositions
CA2875618C (en) 2012-07-11 2021-04-27 Sangamo Biosciences, 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
JP5971661B2 (ja) 2012-09-19 2016-08-17 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
DK2900686T3 (da) 2012-09-28 2020-08-17 Univ North Carolina Chapel Hill Aav-vektorer targeteret til oligodendrocytter
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
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
PT2968605T (pt) 2013-03-15 2022-09-22 Univ North Carolina Chapel Hill Métodos e composições para vetores aav de ligação dupla de glicano
KR20210111878A (ko) 2013-05-21 2021-09-13 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
SG10201809075XA (en) 2013-07-22 2018-11-29 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015044704A1 (en) 2013-09-30 2015-04-02 Sanofi Use of neuroglobin agonist for preventing or treating mitochondrial rcci and/or rcciii deficiency disease
CA3182790A1 (en) 2013-10-11 2015-04-16 Massachusetts Eye & Ear Infirmary Ancestral adeno-associated virus sequences and uses thereof
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
US10308957B2 (en) 2014-03-04 2019-06-04 University Of Florida Research Foundation, Inc. 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
EA034355B1 (ru) 2014-05-02 2020-01-30 Джензим Корпорейшн Векторы на основе aav для генной терапии в сетчатке и cns
BR112016027911A2 (pt) 2014-05-28 2017-10-24 Evogene Ltd polinucleotídeos isolados, polipeptídeos e métodos de uso dos mesmos para aumento da tolerância ao estresse abiótico, biomassa e produção da planta
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
IL298516B1 (en) 2014-06-25 2024-11-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
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
CN107295802B (zh) 2014-09-24 2021-06-29 希望之城 用于高效基因组编辑的腺相关病毒载体变异体和其方法
WO2016054557A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Novel high efficiency library-identified aav vectors
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
AU2015349759B2 (en) 2014-11-21 2022-01-06 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
AU2016206624B2 (en) * 2015-01-14 2020-08-06 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3632923A1 (en) 2015-01-16 2020-04-08 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
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
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 (en) 2015-03-23 2018-01-31 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
US10883117B2 (en) 2015-03-24 2021-01-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
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
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
US11104917B2 (en) 2015-05-08 2021-08-31 Children's Medical Research Institute Promoters for expression of heterologous genes
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
US11447797B2 (en) 2015-05-12 2022-09-20 The United States Of America,As Represented By The Secretary, Department Of Health And Human Service AAV with unique capsid protein VP1 and methods of using for treatment
US20180216133A1 (en) 2015-07-17 2018-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
EP3907231A1 (en) 2015-07-19 2021-11-10 Yeda Research and Development Co., Ltd. Selective inhibitors of alpha2-containing isoforms of na,k-atpase and use thereof for reducing intraocular pressure
US20180214576A1 (en) 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
CN108603235A (zh) 2015-07-30 2018-09-28 马萨诸塞眼科耳科诊所 祖先病毒序列及其用途
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
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
KR102200642B1 (ko) 2015-11-05 2021-01-12 뱀부 테라퓨틱스 인코포레이티드 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터
MY205245A (en) 2015-12-01 2024-10-09 Spark Therapeutics Inc Scalable method for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
WO2017096164A1 (en) 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
CO2018007203A2 (es) 2015-12-11 2018-09-20 California Inst Of Techn Focalización de péptidos para dirigir virus adenoasociados (aavs)
EP3390429A4 (en) 2015-12-14 2019-07-17 The University of North Carolina at Chapel Hill MODIFIED CAPSID PROTEINS FOR IMPROVED DELIVERY OF PARVOVIRUS VECTORS
EP4094780A3 (en) 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
JP6947739B2 (ja) 2016-02-16 2021-10-13 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド
EP3419639A4 (en) 2016-02-22 2019-08-07 The University of North Carolina at Chapel Hill AAV IDUA VECTOR FOR TREATING BLINDNESS IN CONNECTION WITH MPS I
CN109661470A (zh) 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 新型aav8突变衣壳和含有其的组合物
WO2017192750A1 (en) 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
RU2768045C2 (ru) 2016-07-21 2022-03-23 Спарк Терапьютикс, Инк. СПОСОБ ПОЛУЧЕНИЯ ЧАСТИЦ РЕКОМБИНАНТНОГО АДЕНОАССОЦИИРОВАННОГО ВИРУСНОГО ВЕКТОРА (rAAV)
WO2018022608A2 (en) 2016-07-26 2018-02-01 Biomarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
JP7226801B2 (ja) 2016-08-16 2023-02-21 ザ 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 標的遺伝子導入のための方法および組成物
IL265045B2 (en) 2016-09-08 2023-09-01 Bluebird Bio Inc Variants of endonuclease pd-1, compositions and methods of use
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 (en) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. MODIFIED AAV CASPIDS AND USES THEREOF
WO2018119330A2 (en) 2016-12-22 2018-06-28 Oregon Health & Science University Adeno associated viral vectors
SG11201907298WA (en) 2017-02-15 2019-09-27 Univ North Carolina Chapel Hill Methods and compositions for gene transfer across the vasculature
IL268644B1 (en) 2017-02-28 2025-02-01 Univ Pennsylvania Adeno-associated virus group F vector and uses for it
CN110770346B (zh) 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
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
WO2018204764A1 (en) 2017-05-05 2018-11-08 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
CA3061968A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses
US11680275B2 (en) 2017-06-06 2023-06-20 University Of Massachusetts Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome
CA3068010A1 (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 niemann-pick type c1 deficiency and related conditions
CN111511375A (zh) 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
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
CA3071769A1 (en) 2017-07-31 2019-02-07 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
KR20200135768A (ko) 2018-01-17 2020-12-03 메이라지티엑스 유케이 Ii 리미티드 유전자 치료를 위한 변형된 rAAV 캡시드 단백질
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
SG11202008278WA (en) 2018-02-28 2020-09-29 Univ North Carolina Chapel Hill Methods and compositions for antibody-evading virus vectors
US20210010028A1 (en) 2018-03-06 2021-01-14 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
US11905524B2 (en) 2018-03-06 2024-02-20 University Of Florida Research Foundation, Incorporated AAV chimeras
WO2019178412A1 (en) 2018-03-16 2019-09-19 Research Institute At Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，インコーポレイテッド 抗体を回避するウイルスベクター
EA202092362A1 (ru) 2018-04-03 2021-01-11 Страйдбайо, Инк. Вирусные векторы для нацеливания на ткани глаза
US20210214749A1 (en) 2018-05-16 2021-07-15 Voyager Therapeutics, Inc. Directed evolution
TW202039533A (zh) 2018-11-21 2020-11-01 美商史崔德生物公司 重組病毒載體及用於生產其之核酸
EP4269579A3 (en) 2019-01-04 2024-01-24 Ultragenyx Pharmaceutical Inc. Gene therapy constructs for treating wilson disease
JP2022525955A (ja) 2019-03-21 2022-05-20 ストライドバイオ，インコーポレイテッド 組換えアデノ随伴ウイルスベクター
CN114072514A (zh) 2019-05-14 2022-02-18 杜克大学 用于治疗atp酶介导的疾病的组合物和方法
EP4045525A1 (en) 2019-10-17 2022-08-24 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CA3157702A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Aav transfer cassette
US20230304038A1 (en) 2020-08-19 2023-09-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome

Also Published As

Publication number Publication date
JP2021519583A (ja) 2021-08-12
JP2024054142A (ja) 2024-04-16
SG11202009450SA (en) 2020-10-29
AU2019247191A1 (en) 2020-10-15
KR20210006358A (ko) 2021-01-18
MX2020010465A (es) 2021-01-08
CN112533644A (zh) 2021-03-19
CA3094465A1 (en) 2019-10-10
WO2019195423A1 (en) 2019-10-10
US20210371469A1 (en) 2021-12-02
US12116384B2 (en) 2024-10-15
IL277667A (en) 2020-11-30
JP7425738B2 (ja) 2024-01-31
BR112020020341A2 (pt) 2021-01-05
EP3773743A1 (en) 2021-02-17

Similar Documents

Publication Publication Date Title
EA202092362A1 (ru) Вирусные векторы для нацеливания на ткани глаза
EA201991911A1 (ru) Способы и композиции для переноса генов по сосудистой сети
EA202192501A1 (ru) Рекомбинантные аденоассоциированные вирусные векторы
MX2020010466A (es) Vectores de virus que evitan anticuerpos.
MX2020010464A (es) Vectores de virus que evitan anticuerpos.
WO2017058892A3 (en) Methods and compositions for antibody-evading virus vectors
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
UA124343C2 (uk) Капсиди аденоасоційованого вірусу і спосіб його використання
BR112019002904A2 (pt) métodos e composições para transferência gênica direcionada
PH12020550706A1 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
EA202090332A1 (ru) Улучшенная продукция капсида aav в клетках насекомых
MX2022015079A (es) Variantes de hsd17b13 y usos de las mismas.
PH12020551938A1 (en) Liver targeting adeno-associated viral vectors
EA201892369A1 (ru) Способы селективной модуляции активности отдельных подтипов клеток
MX2020008932A (es) Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
MX2018006840A (es) Focalizacion de peptidos para dirigir virus adenoasociados.
PH12021552638A1 (en) Compositions useful for treatment of pompe disease
PH12020550117A1 (en) Variant rnai
EA202090700A1 (ru) Компетентные по репликации аденовирусные векторы
EA201890187A1 (ru) Рекомбинантные вирусоподобные частицы (vlp) с использованием протеина группового антигена (gag) вируса бычьего иммунодефицита
EA202092353A1 (ru) Уклоняющиеся от антител вирусные векторы
EA202092363A1 (ru) Уклоняющиеся от антител вирусные векторы
EA201992558A1 (ru) Варианты белка bmp7 человека
UY38881A (es) Métodos de tratamiento de enfermedades neovasculares oculares utilizando variantes de aav2 que codifican aflibercept
EA201990955A1 (ru) Разработка капсидов aav