ATE362542T1 - Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten - Google Patents

Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten

Info

Publication number
ATE362542T1
ATE362542T1 AT99961559T AT99961559T ATE362542T1 AT E362542 T1 ATE362542 T1 AT E362542T1 AT 99961559 T AT99961559 T AT 99961559T AT 99961559 T AT99961559 T AT 99961559T AT E362542 T1 ATE362542 T1 AT E362542T1
Authority
AT
Austria
Prior art keywords
vectors
adeno
nucleic acid
host cells
acid sequences
Prior art date
Application number
AT99961559T
Other languages
English (en)
Inventor
James Wilson
Weidong Xiao
Original Assignee
Univ Pennsylvania
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Application granted granted Critical
Publication of ATE362542T1 publication Critical patent/ATE362542T1/de

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AT99961559T 1998-11-05 1999-11-02 Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten ATE362542T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US10711498P 1998-11-05 1998-11-05

Publications (1)

Publication Number Publication Date
ATE362542T1 true ATE362542T1 (de) 2007-06-15

Family

ID=22314919

Family Applications (1)

Application Number Title Priority Date Filing Date
AT99961559T ATE362542T1 (de) 1998-11-05 1999-11-02 Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten

Country Status (11)

Country Link
EP (1) EP1127150B1 (de)
JP (1) JP4573437B2 (de)
AT (1) ATE362542T1 (de)
AU (2) AU768729B2 (de)
CA (1) CA2349838C (de)
CY (1) CY1106815T1 (de)
DE (1) DE69936104T2 (de)
DK (1) DK1127150T3 (de)
ES (1) ES2288037T3 (de)
PT (1) PT1127150E (de)
WO (1) WO2000028061A2 (de)

Families Citing this family (152)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
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
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP4060531B2 (ja) 1998-05-28 2008-03-12 アメリカ合衆国 Aav5ベクターおよびその使用
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
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
US6627617B1 (en) 1999-10-01 2003-09-30 University Of North Carolina At Chapel Hill Temperature-sensitive regulation of viral vector production
DE10120265A1 (de) * 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren
CN112029800A (zh) * 2001-11-13 2020-12-04 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
ATE520707T1 (de) 2001-11-13 2011-09-15 Univ Pennsylvania Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
AU2008202344B2 (en) * 2001-11-13 2011-08-25 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
AU2002360291A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
JP2005532789A (ja) 2002-03-15 2005-11-04 ワイス・ホールデイングス・コーポレーシヨン 減少した酵素活性を有する型別不能なインフルエンザ菌(Haemophilusinfluenza)のP4タンパク質の変異体
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
PL3211085T3 (pl) 2003-09-30 2021-11-15 The Trustees Of The University Of Pennsylvania Klady wirusa towarzyszącego adenowirusom (aav), sekwencje, wektory je zawierające, oraz ich zastosowania
WO2005056807A2 (en) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
WO2006078279A2 (en) 2004-04-28 2006-07-27 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
JP2007535541A (ja) 2004-04-28 2007-12-06 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 E4欠失アデノウイルス初回免疫およびe1欠失アデノウイルス追加免疫を用いる免疫化方式
US7892809B2 (en) 2004-12-15 2011-02-22 The University Of North Carolina At Chapel Hill Chimeric vectors
EP4282956A3 (de) 2005-04-07 2024-03-13 The Trustees of the University of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
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
WO2008016391A2 (en) * 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
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
US8221738B2 (en) 2008-02-19 2012-07-17 Celladon Corporation Method for enhanced uptake of viral vectors in the myocardium
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CN102448501A (zh) 2009-03-27 2012-05-09 西马生物医学计划公司 治疗肝硬化和肝纤维化的方法和组合物
JP2014503173A (ja) 2009-11-05 2014-02-13 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 制御された発現系
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
WO2012007458A1 (en) 2010-07-12 2012-01-19 Universidad Autónoma De Barcelona Gene therapy composition for use in diabetes treatment
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
US9193956B2 (en) 2011-04-22 2015-11-24 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP2692868A1 (de) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
CN111621507A (zh) 2012-09-28 2020-09-04 北卡罗来纳-查佩尔山大学 靶向少突胶质细胞的aav载体
PT2968605T (pt) 2013-03-15 2022-09-22 Univ North Carolina Chapel Hill Métodos e composições para vetores aav de ligação dupla de glicano
EP2970966A4 (de) 2013-03-15 2016-10-26 Univ North Carolina Synthetische adenoassoziierte virusinvertierte endwiederholungssequenzen
EP3567112A1 (de) 2013-06-13 2019-11-13 Translate Bio, Inc. Virenproduktion auf messenger-rna-basis
EP4039813A1 (de) * 2013-07-12 2022-08-10 The Children's Hospital of Philadelphia Aav-vektor und assay für anti-aav (adeno-assoziierter virus)-neutralisierende antikörper
EP3068889B1 (de) 2013-09-26 2019-04-17 Universitat Autònoma De Barcelona Gentherapiezusammensetzungen zur verwendung bei der vorbeugung und/oder behandlung von nicht alkoholbedingter fettleber
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
KR102390075B1 (ko) 2014-03-09 2022-04-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 오르니틴 트랜스카르바밀라아제(otc) 결핍증의 치료에 유용한 조성물
US9458464B2 (en) 2014-06-23 2016-10-04 The Johns Hopkins University Treatment of neuropathic pain
EP3194430A1 (de) 2014-09-16 2017-07-26 Universitat Autònoma De Barcelona Adeno-assoziierte virusvektoren zur gentheraüie von stoffwechselkrankheiten
CN107532173A (zh) 2014-11-21 2018-01-02 北卡罗来纳-查佩尔山大学 靶向中枢神经系统的aav载体
US11033638B2 (en) 2015-01-07 2021-06-15 Universität Autonoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes
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
US10308705B2 (en) 2015-02-06 2019-06-04 The University Of North Carolina At Chapel Hill Optimized human clotting factor VIII gene expression cassettes and their use
IL299901B1 (en) 2015-09-28 2024-08-01 Univ Florida Methods and compositions for stealth virus antibody vectors
MX2018005084A (es) 2015-11-05 2019-05-16 Bamboo Therapeutics Inc Genes de ataxia de friedreich modificados y vectores para terapia genica.
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
LT3411484T (lt) 2016-02-05 2023-11-27 Emory University Viengrandžio arba sau pačiam komplementaraus adeno-asocijuoto viruso 9 injekcija į smegenų skystį
EP3419639A4 (de) 2016-02-22 2019-08-07 The University of North Carolina at Chapel Hill Aav-idua-vektor zur behandlung von blindheit im zusammenhang mit mps i
EP3449250B1 (de) 2016-04-28 2020-11-04 Indiana University Research & Technology Corporation Verfahren und zusammensetzungen zur auflösung von komponenten einer viruspräparation
EP3448875A4 (de) * 2016-04-29 2020-04-08 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017190125A1 (en) 2016-04-29 2017-11-02 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11866462B2 (en) * 2016-05-04 2024-01-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
FI3445773T3 (fi) 2016-05-13 2023-03-30 4D Molecular Therapeutics Inc Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
CA3024536A1 (en) 2016-06-13 2017-12-21 The University Of North Carolina At Chapel Hill Optimized cln1 genes and expression cassettes and their use
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
BR112019001344A2 (pt) 2016-07-26 2019-04-30 The University Of North Carolina At Chapel Hill tolerância imunológica mediada por vetor no olho
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US20180245065A1 (en) 2016-11-01 2018-08-30 Novartis Ag Methods and compositions for enhancing gene editing
EP3570895A1 (de) 2017-01-17 2019-11-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren zur expression eines bestimmten polynukleotids in den zapfen-fotorezeptoren
EP3589277A4 (de) * 2017-02-28 2020-08-26 Adverum Biotechnologies, Inc. Modifizierte aav-kapside und verwendungen davon
AU2018234695A1 (en) 2017-03-15 2019-09-12 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
EP3388520A1 (de) 2017-04-11 2018-10-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren und pharmazeutische zusammensetzung zur reduzierung der expression von nkcc1 in einer darauf angewiesenen person
US20200207830A1 (en) 2017-07-27 2020-07-02 Novartis Ag Trem2 mutants resistant to sheddase cleavage
EP3685168A1 (de) 2017-09-20 2020-07-29 The Trustees Of Indiana University Verfahren zur auflösung von lipoproteinen mit massenspektrometrie
PE20201264A1 (es) 2017-09-20 2020-11-19 4D Molecular Therapeutics Inc Variantes de capsides de virus adenoasociados y metodos de uso de estas
CN111867635A (zh) 2017-10-20 2020-10-30 法国国家卫生及研究医学协会 包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法
WO2019094061A1 (en) 2017-11-07 2019-05-16 The University Of North Carolina At Chapel Hill Optimized aga genes and expression cassettes and their use
UA127831C2 (uk) 2017-11-27 2024-01-17 4Д Молекьюлар Терапьютикс Інк. Капсид аденоасоційованого вірусу та його застосування для інгібування ангіогенезу
EP3738137A1 (de) 2018-01-12 2020-11-18 The Trustees of Indiana University Konstruktion einer elektrostatischen linearen ionenfalle für massenspektrometrie mit ladungsdetektion
WO2019165058A1 (en) 2018-02-21 2019-08-29 Alcyone Lifesciences, Inc. Fluid delivery systems and methods
AU2019247746B2 (en) 2018-04-03 2024-08-15 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
US20210371469A1 (en) 2018-04-03 2021-12-02 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
EP3774854A1 (de) 2018-04-03 2021-02-17 Stridebio, Inc. Antikörperausweichende virusvektoren
AR126019A1 (es) 2018-05-30 2023-09-06 Novartis Ag Anticuerpos frente a entpd2, terapias de combinación y métodos de uso de los anticuerpos y las terapias de combinación
CN112673451B (zh) 2018-06-04 2024-07-19 印地安纳大学理事会 具有实时分析和信号优化的电荷检测质谱分析
JP7398811B2 (ja) 2018-06-04 2023-12-15 ザ・トラスティーズ・オブ・インディアナ・ユニバーシティー 高スループット電荷検出質量分光分析のためのイオン・トラップ・アレイ
CN112673452B (zh) 2018-06-04 2024-08-23 印地安纳大学理事会 用于在静电线性离子阱中捕获离子的设备和方法
WO2019236139A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Interface for transporting ions from an atmospheric pressure environment to a low pressure environment
WO2019236143A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for calibrating or resetting a charge detector
EP3807404A1 (de) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Manipulierte 5'-unübersetzte regionen (5' utr) zur herstellung von aav
US20210269829A1 (en) 2018-06-28 2021-09-02 The University Of North Carolina At Chapel Hill Optimized cln5 genes and expression cassettes and their use
CN112770812A (zh) 2018-07-24 2021-05-07 沃雅戈治疗公司 产生基因治疗制剂的系统和方法
EP3833767A4 (de) 2018-08-10 2022-05-04 The University of North Carolina at Chapel Hill Optimierte cln7-gene und expressionskassetten sowie deren verwendung
CN112867529B (zh) 2018-08-27 2023-06-02 亚克安娜医疗有限公司 流体递送系统和方法
EP3861113A1 (de) 2018-10-04 2021-08-11 Voyager Therapeutics, Inc. Verfahren zur messung des titers und der potenz von viralen vektorpartikeln
US20210348194A1 (en) 2018-10-05 2021-11-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
AU2019360937A1 (en) 2018-10-15 2021-05-20 Voyager Therapeutics, Inc. Expression vectors for large-scale production of rAAV in the baculovirus/Sf9 system
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
AU2019384065A1 (en) 2018-11-20 2021-06-03 The Trustees Of Indiana University Orbitrap for single particle mass spectrometry
EP3891777A1 (de) 2018-12-03 2021-10-13 The Trustees of Indiana University Vorrichtung und verfahren zur gleichzeitigen analyse mehrerer ionen mit einer elektrostatischen linearen ionenfalle
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
CR20210444A (es) 2019-02-25 2021-11-02 Novartis Ag Composiciones y métodos para tratar distrofia cristalina de bietti
SG11202110146VA (en) 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
CA3136872A1 (en) 2019-04-23 2020-10-29 Inserm (Institut National De La Sante Et De La Recherche Medicale) New adeno-associated virus (aav) variants and uses thereof for gene therapy
EP3959741A1 (de) 2019-04-23 2022-03-02 The Trustees of Indiana University Identifizierung von probensubspezies basierend auf partikelladungsverhalten unter strukturveränderungen induzierenden probenbedingungen
JP2022529662A (ja) 2019-04-26 2022-06-23 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 二重グリカン結合aav2.5ベクターのための方法および組成物
SG11202111616RA (en) 2019-04-26 2021-11-29 Sangamo Therapeutics Inc Engineering aav
MX2021014338A (es) 2019-05-24 2022-01-06 Regeneron Pharma Particulas virales modificadas y usos de estas.
US20230241248A1 (en) 2019-06-27 2023-08-03 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
WO2021053559A1 (en) 2019-09-18 2021-03-25 Novartis Ag Entpd2 antibodies, combination therapies, and methods of using the antibodies and combination therapies
TW202124446A (zh) 2019-09-18 2021-07-01 瑞士商諾華公司 與entpd2抗體之組合療法
GB201913974D0 (en) 2019-09-27 2019-11-13 King S College London Vector
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
JP2023515820A (ja) 2020-02-28 2023-04-14 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル メープルシロップ尿症の遺伝子治療
WO2021188960A1 (en) 2020-03-20 2021-09-23 University Of Florida Research Foundation, Inc. Gene therapy for cockayne syndrome
AU2021244191A1 (en) 2020-03-23 2022-09-22 The University Of North Carolina At Chapel Hill AAV-NAGLU vectors for treatment of mucopolysaccharidosis IIIB
KR20230042754A (ko) 2020-08-07 2023-03-29 아미쿠스 세라퓨틱스, 인코포레이티드 소포 표적화 단백질 및 이의 용도
EP4010356B1 (de) 2020-10-28 2024-09-04 The University of North Carolina at Chapel Hill Verfahren und zusammensetzungen für dualen glycanbindenden aav2.5-vektor
WO2022093769A1 (en) 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
MX2023005218A (es) 2020-11-03 2023-05-16 Pfizer Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico.
US20240052322A1 (en) 2020-12-15 2024-02-15 Pfizer Inc. HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins
EP4267726A1 (de) 2020-12-23 2023-11-01 Pfizer Inc. Verfahren zur reinigung von aav-vektoren durch affinitätschromatografie
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
EP4284335A1 (de) 2021-02-01 2023-12-06 RegenxBio Inc. Gentherapie für neuronale ceroid-lipofuscinosen
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
AU2022256479A1 (en) 2021-04-16 2023-11-30 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
EP4329820A1 (de) 2021-04-26 2024-03-06 Alexion Pharma International Operations Limited Adeno-assoziierte virale vektorkapside mit verbessertem gewebetropismus
JP2024519888A (ja) 2021-05-28 2024-05-21 上海瑞宏迪医薬有限公司 カプシド変異を有する組換えアデノ随伴ウイルス及びその応用
EP4359547A1 (de) 2021-06-22 2024-05-01 Pfizer Inc. Herstellung eines adeno-assoziierten virusvektors in insektenzellen
JP2024526286A (ja) 2021-07-01 2024-07-17 アミカス セラピューティックス インコーポレイテッド ニューロテンシン変異体及びニューロテンシン又はソルチリンプロペプチドを含むタグ付きタンパク質
WO2023283649A1 (en) 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
JP2024530050A (ja) 2021-08-11 2024-08-14 キングス・カレッジ・ロンドン 中枢神経系に影響を及ぼす障害の改善された治療のための組成物及び方法
AU2022368907A1 (en) 2021-10-20 2024-05-02 University Of Copenhagen Rejuvenation treatment of age-related white matter loss
US20230270818A1 (en) 2021-11-02 2023-08-31 University Of Rochester Tcf7l2 mediated remyelination in the brain
WO2023081850A1 (en) 2021-11-04 2023-05-11 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to skeletal muscle
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
WO2023150687A1 (en) 2022-02-04 2023-08-10 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors, and methods of use thereof
TW202404993A (zh) 2022-04-11 2024-02-01 美商特納亞治療股份有限公司 具經工程化蛋白殼之腺相關病毒
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2023220603A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024026494A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to transferrin receptor 1
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024124019A2 (en) 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells
WO2024130175A2 (en) 2022-12-16 2024-06-20 Regeneron Pharmaceuticals, Inc. Antigen-binding molecules that bind to aav particles and uses
WO2024147114A1 (en) 2023-01-06 2024-07-11 Bloomsbury Genetic Therapies Limited Compositions and methods for treating parkinson's disease
WO2024163747A2 (en) 2023-02-02 2024-08-08 University Of Rochester Competitive replacement of glial cells
WO2024173248A1 (en) 2023-02-13 2024-08-22 Regeneron Pharmaceuticals, Inc. Treatment of muscle related disorders with anti-human cacng1 antibodies

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
EP1127150A2 (de) 2001-08-29
CA2349838A1 (en) 2000-05-18
AU1811100A (en) 2000-05-29
WO2000028061A9 (en) 2000-11-02
AU2004201463C1 (en) 2008-07-24
AU2004201463A1 (en) 2004-05-06
PT1127150E (pt) 2007-08-22
JP4573437B2 (ja) 2010-11-04
DE69936104T2 (de) 2008-01-17
DE69936104D1 (de) 2007-06-28
EP1127150B1 (de) 2007-05-16
JP2002529098A (ja) 2002-09-10
CA2349838C (en) 2011-06-07
ES2288037T3 (es) 2007-12-16
WO2000028061A3 (en) 2000-08-03
CY1106815T1 (el) 2012-05-23
AU2004201463B2 (en) 2008-01-03
AU768729B2 (en) 2004-01-08
WO2000028061A2 (en) 2000-05-18
DK1127150T3 (da) 2007-09-24

Similar Documents

Publication Publication Date Title
DE69936104D1 (de) Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
CY1120209T1 (el) Αλληλουχιες αδενο-εξαρτωμενου ιου (aav) οροτυπου 9, φορεις που τον περιεχουν και χρησεις αυτων
DE59913833D1 (de) Strukturprotein von aav, seine herstellung und verwendung
NO962950L (no) Fremgangsmåte for fremstilling av adeno-assosierte viruser (AAV), og deres anvendelse
IL234063A0 (en) Sequences of adenoid virus serological type 7 and recombinant viruses containing them
WO2005033321A3 (en) Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
DE69535703D1 (de) Aav-vermittelte überbringung von dna in zellen des nervensystems
CY1106216T1 (el) 5´ ESTs ΓΙΑ ΜΗ ΙΣΤΟ-ΕΙΔΙΚΕΣ ΕΚΚΡΙΤΙΚΕΣ ΠΡΩΤΕΪΝΕΣ
WO1998011244A3 (en) AAV4 vector and uses thereof
MXPA03002413A (es) Moleculas tipo b7 y uso de las mismas.
EP0804609A4 (de) NEUARTIGE AUF p53 ANSPRECHENDE GENE
NO990025L (no) Hamster-EF-1<alfa> transkripsjonsregulatorisk DNA
WO1998028317A3 (en) Synthesis of nucleosides and polynucleotides
WO1998006841A3 (en) Two human nsp-like proteins
WO1999023216A3 (en) Hyperthermic inducible expression vectors for gene therapy and methods of use thereof
ATE420157T1 (de) Zielgerichtete genveraenderung mit parvoviralen vektoren
EP1845163A3 (de) Adeno-assoziierte Virus-Serotyp-I-Nukleinsäuresequenzen und diese enthaltende Vektoren und Wirtszellen
MX9806041A (es) Proteina purificada sr-p70.
WO1999047558A3 (en) T cell costimulatory proteins, sequences and uses thereof
MXPA03010728A (es) Moleculas de proteina-2 relacionadas con b7 y uso de las mismas.
GB2358865A (en) Targeted gene modification by parvoviral vectors
ATE435915T1 (de) Spezifische nukleinsäuren und polypeptide aus krankheitserregenden stämmen von neisseria
ATE262034T1 (de) Xylanasen, für diese kodierende gene und anwendungen derselben
WO1998012220A3 (en) Novel tumor proteins
EP0778347A3 (de) ATP- und Nukleinsäure-bindendes Protein mit putativen Helikase- und ATPase Eigenschaften

Legal Events

Date Code Title Description
UEP Publication of translation of european patent specification

Ref document number: 1127150

Country of ref document: EP