DK1127150T3 - Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse - Google Patents

Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse

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Publication number
DK1127150T3
DK1127150T3 DK99961559T DK99961559T DK1127150T3 DK 1127150 T3 DK1127150 T3 DK 1127150T3 DK 99961559 T DK99961559 T DK 99961559T DK 99961559 T DK99961559 T DK 99961559T DK 1127150 T3 DK1127150 T3 DK 1127150T3
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Prior art keywords
vectors
serotype
adeno
nucleic acid
host cells
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DK99961559T
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Danish (da)
English (en)
Inventor
James M Wilson
Weidong Xiao
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Univ Pennsylvania
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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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

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  • 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)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
DK99961559T 1998-11-05 1999-11-02 Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse DK1127150T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US10711498P 1998-11-05 1998-11-05
PCT/US1999/025694 WO2000028061A2 (en) 1998-11-05 1999-11-02 Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same

Publications (1)

Publication Number Publication Date
DK1127150T3 true DK1127150T3 (da) 2007-09-24

Family

ID=22314919

Family Applications (1)

Application Number Title Priority Date Filing Date
DK99961559T DK1127150T3 (da) 1998-11-05 1999-11-02 Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse

Country Status (11)

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

Families Citing this family (157)

* 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
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2745131C (en) 1998-05-28 2016-08-09 John A. Chiorini Aav5 vector and uses thereof
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
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
WO2001025253A2 (en) 1999-10-01 2001-04-12 The 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
CN101426935B (zh) * 2001-11-13 2013-08-21 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
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
PL222683B1 (pl) 2001-11-13 2016-08-31 Univ Pennsylvania Rekombinowane wirusy stowarzyszone z adenowirusem (AAV), sposoby ich wytwarzania, pakująca komórka gospodarza, kompozycja zawierająca wirus, zastosowanie wirusa, wyizolowane wirusy AAV, białka,sztuczne białka kapsydu, cząsteczki, komórki gospodarza, sposoby dostarczania transgenu, sposób identyfikacji serotypu, zestaw diagnostyczny, sposób izolacji nowych wirusów, rekombinowana komórka
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP1463805B1 (en) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
IL163988A0 (en) 2002-03-15 2005-12-18 Wyeth Corp Mutants of the p4 protein of nontypable haemophilus influenzae with reduced enzymatic activity
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
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
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
EP1742657B1 (en) 2004-04-28 2013-11-06 The Trustees of The University of Pennsylvania Immunization regimen with e4-deleted adenovirus prime and e1-deleted adenovirus boost
US8394386B2 (en) 2004-04-28 2013-03-12 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
DK2359867T3 (en) 2005-04-07 2015-01-05 Univ Pennsylvania A method for increasing an AAV vector function
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
EP1979485A2 (en) 2006-01-31 2008-10-15 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
CA2715765A1 (en) 2008-02-19 2009-08-27 Celladon Corporation Compositions for enhanced uptake of viral vectors in the myocardium
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
WO2010109053A1 (es) 2009-03-27 2010-09-30 Proyeto De Biomedicina Cima, S.L. Métodos y composiciones para el tratamiento de cirrosis y fibrosis hepática
CN102712933A (zh) 2009-11-05 2012-10-03 西马生物医学计划公司 调控的表达系统
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
JP5704361B2 (ja) * 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
DK2673289T3 (da) 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf
SG194583A1 (en) 2011-04-22 2013-12-30 Univ California Adeno-associated virus virions with variant capsid and methods of use thereof
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
EP2900686B1 (en) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
PT2968605T (pt) 2013-03-15 2022-09-22 Univ North Carolina Chapel Hill Métodos e composições para vetores aav de ligação dupla de glicano
EP3517612A1 (en) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
EP3567112A1 (en) 2013-06-13 2019-11-13 Translate Bio, Inc. Messenger rna based viral production
EP4039813A1 (en) 2013-07-12 2022-08-10 The Children's Hospital of Philadelphia Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies
EP3068889B1 (en) 2013-09-26 2019-04-17 Universitat Autònoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
PL3116900T3 (pl) 2014-03-09 2021-03-08 The Trustees Of The University Of Pennsylvania Kompozycje użyteczne w leczeniu niedoboru transkarbamylazy ornitynowej (otc)
US9458464B2 (en) 2014-06-23 2016-10-04 The Johns Hopkins University Treatment of neuropathic pain
CA2960912A1 (en) 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
US10532110B2 (en) 2014-11-21 2020-01-14 The University Of North Carolina At Chapel Hill AAV vectors targeted to the central nervous system
WO2016110518A1 (en) 2015-01-07 2016-07-14 Universitat Autònoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes
CA2972807C (en) 2015-01-14 2024-01-16 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3253786A4 (en) 2015-02-06 2018-10-17 The University of North Carolina at Chapel Hill Optimized human clotting factor viii gene expression cassettes and their use
CN108137655B (zh) 2015-09-28 2022-04-22 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
US10548947B2 (en) 2015-11-05 2020-02-04 Bamboo Therapeutics, Inc. Modified Friedreich ataxia genes and vectors for gene therapy
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
WO2017106236A1 (en) 2015-12-14 2017-06-22 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
HRP20231451T1 (hr) 2016-02-05 2024-03-01 Emory University Ubrizgavanje jednolančanog ili samo-komplementarnog adeno-povezanog virusa 9 u cerebrospinalnu tekućinu
CN108697774A (zh) 2016-02-22 2018-10-23 北卡罗来纳大学教堂山分校 治疗mps i-相关失明的aav-idua载体
EP3449250B1 (en) 2016-04-28 2020-11-04 Indiana University Research & Technology Corporation Methods and compositions for resolving components of a virus preparation
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
ES2908467T3 (es) 2016-04-29 2022-04-29 Adverum Biotechnologies Inc Evasión de anticuerpos neutralizantes por un virus adenoasociado recombinante
EP3448875A4 (en) * 2016-04-29 2020-04-08 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11866462B2 (en) * 2016-05-04 2024-01-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
UA124343C2 (uk) 2016-05-13 2021-09-01 4Д Молекьюлар Терапьютікс Інк. Капсиди аденоасоційованого вірусу і спосіб його використання
WO2017218450A1 (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
CN110167958A (zh) 2016-07-26 2019-08-23 北卡罗来纳大学教堂山分校 载体介导的眼睛免疫耐受性
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
WO2018083606A1 (en) 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
WO2018134168A1 (en) 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
JP2020510424A (ja) * 2017-02-28 2020-04-09 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 改変ａａｖカプシドおよびその使用
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
EP3388520A1 (en) 2017-04-11 2018-10-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for reducing the expression of nkcc1 in a subject in need thereof
WO2019021233A1 (en) 2017-07-27 2019-01-31 Novartis Ag TREM2 VARIANTS RESISTANT TO SHEDDASE
WO2019060454A2 (en) 2017-09-20 2019-03-28 4D Molecular Therapeutics Inc. CAPSID VARIANT ADENO-ASSOCIATED VIRUSES AND METHODS OF USE
EP3685168A1 (en) 2017-09-20 2020-07-29 The Trustees Of Indiana University Methods for resolving lipoproteins with mass spectrometry
JP7420710B2 (ja) 2017-10-20 2024-01-23 アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） 組換えａａｖ９由来ベクターの治療有効量の網膜下送達を含む、対象の錐体視細胞において目的のポリヌクレオチドを発現させる方法
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
CR20200282A (es) 2017-11-27 2021-02-18 4D Molecular Therapeutics Inc Cápsides variantes de virus adenoasociados y su uso para inhibir la angiogénesis
WO2019140233A1 (en) 2018-01-12 2019-07-18 The Trustees Of Indiana University Electrostatic linear ion trap design for charge detection mass spectrometry
EP3746153A4 (en) 2018-02-21 2021-11-10 Alcyone Lifesciences, Inc. FLUID DISTRIBUTION SYSTEMS AND METHODS
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス 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
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
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
CN112703579A (zh) 2018-06-04 2021-04-23 印地安纳大学理事会 用于高通量电荷检测质谱分析的离子阱阵列
WO2019236141A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for capturing ions in an electrostatic linear ion trap
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
KR20210035103A (ko) 2018-06-04 2021-03-31 더 트러스티즈 오브 인디애나 유니버시티 실시간 분석 및 신호 최적화를 통한 전하 검출 질량 분광분석법
WO2019236143A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for calibrating or resetting a charge detector
AU2019287468B2 (en) 2018-06-12 2024-10-10 The University Of North Carolina At Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
WO2020006200A1 (en) 2018-06-28 2020-01-02 The University Of North Carolina At Chapel Hill Optimized cln5 genes and expression cassettes and their use
JP2021530548A (ja) 2018-07-24 2021-11-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. 遺伝子治療製剤を生産するための系および方法
WO2020033833A1 (en) 2018-08-10 2020-02-13 The University Of North Carolina At Chapel Hill Optimized cln7 genes and expression cassettes and their use
CA3110345A1 (en) 2018-08-27 2020-03-05 Alcyone Lifesciences, Inc. Fluid delivery systems and methods
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
JP7285023B2 (ja) 2018-11-20 2023-06-01 ザ・トラスティーズ・オブ・インディアナ・ユニバーシティー 単一粒子質量分光分析のためのオービトラップ
US11562896B2 (en) 2018-12-03 2023-01-24 The Trustees Of Indiana University Apparatus and method for simultaneously analyzing multiple ions with an electrostatic linear ion trap
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
MX2021011468A (es) 2019-03-21 2021-12-15 Vectores de virus adenoasociados recombinantes.
SG11202110607WA (en) 2019-04-01 2021-10-28 Tenaya Therapeutics Inc Adeno-associated virus with engineered capsid
US20220280655A1 (en) 2019-04-23 2022-09-08 Institut National de la Santé et de la Recherche Médicale New Adeno-Associated Virus (AAV) Variants and Uses Thereof for Gene Therapy
WO2020219527A1 (en) 2019-04-23 2020-10-29 The Trustees Of Indiana University Identification of sample subspecies based on particle charge behavior under structural change-inducing sample conditions
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
CA3137961A1 (en) 2019-04-26 2020-10-29 Sangamo Therapeutics, Inc. Engineering aav
BR112021023692A2 (pt) 2019-05-24 2022-01-04 Regeneron Pharma Partícula viral de vírus adeno-associado recombinante, proteína de capsídeo de vírus adeno-associado, molécula de ácido nucleico, célula empacotadora, métodos de produção de uma partícula viral e de distribuição de um nucleotídeo, e, composição farmacêutica
US20230241248A1 (en) 2019-06-27 2023-08-03 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
TW202124446A (zh) 2019-09-18 2021-07-01 瑞士商諾華公司 與ｅｎｔｐｄ２抗體之組合療法
JP2022548881A (ja) 2019-09-18 2022-11-22 ノバルティス アーゲー Ｅｎｔｐｄ２抗体、組合せ療法並びに抗体及び組合せ療法を使用する方法
WO2021061650A1 (en) 2019-09-25 2021-04-01 The Trustees Of Indiana University Apparatus and method for pulsed mode charge detection mass spectrometry
GB201913974D0 (en) 2019-09-27 2019-11-13 King S College London Vector
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
WO2021170784A1 (en) 2020-02-28 2021-09-02 INSERM (Institut National de la Santé et de la Recherche Médicale) Gene therapy for maple syrup urine disease
EP4121168A1 (en) 2020-03-20 2023-01-25 University of Florida Research Foundation, Inc. Gene therapy for cockayne syndrome
WO2021194915A1 (en) 2020-03-23 2021-09-30 The University Of North Carolina At Chapel Hill Aav-naglu vectors for treatment of mucopolysaccharidosis iiib
US20240043494A1 (en) 2020-08-07 2024-02-08 Amicus Therapeutics, Inc. Vesicle Targeting Proteins And Uses Of Same
MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
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
CN116490514A (zh) 2020-10-28 2023-07-25 北卡罗来纳-查佩尔山大学 用于双重聚糖结合aav2.5载体的方法和组合物
JP2023548816A (ja) 2020-11-03 2023-11-21 ファイザー・インク 陰イオン交換クロマトグラフィーによるａａｖベクターの精製方法
WO2022130172A1 (en) 2020-12-15 2022-06-23 Pfizer Inc. Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of AAV vectors by affinity chromatography
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 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
JP2024515626A (ja) 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル， インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドａａｖビリオン
CN117377500A (zh) 2021-04-26 2024-01-09 阿雷克森国际制药有限公司 具有改善的组织向性的腺相关病毒载体衣壳
EP4349852A1 (en) 2021-05-28 2024-04-10 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof
JP2023002483A (ja) 2021-06-22 2023-01-10 ファイザー・インク 昆虫細胞におけるアデノ随伴ウイルスベクターの産生
WO2023279073A1 (en) 2021-07-01 2023-01-05 Amicus Therapeutics, Inc. Neurotensin variants and tagged proteins comprising neurotensin or sortilin propeptide
MX2024000175A (es) 2021-07-08 2024-03-05 Tenaya Therapeutics Inc Casetes de expresión optimizada para genoterapia.
EP4384195A2 (en) 2021-08-11 2024-06-19 King's College London Compositions and methods for improved treatment of disorders affecting the central nervous system
AU2022371414A1 (en) 2021-10-20 2024-05-02 University Of Copenhagen Isolated glial progenitor cells for use in the competition treatment of age-related white matter loss
US20230270818A1 (en) 2021-11-02 2023-08-31 University Of Rochester Tcf7l2 mediated remyelination in the brain
EP4426359A1 (en) 2021-11-04 2024-09-11 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
AU4645697A (en) * 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof

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