JPH09509564A - 高力価組換えａａｖベクターの生成 - Google Patents

高力価組換えａａｖベクターの生成

Info

Publication number
JPH09509564A
JPH09509564A JP7513877A JP51387794A JPH09509564A JP H09509564 A JPH09509564 A JP H09509564A JP 7513877 A JP7513877 A JP 7513877A JP 51387794 A JP51387794 A JP 51387794A JP H09509564 A JPH09509564 A JP H09509564A
Authority
JP
Japan
Prior art keywords
aav
vector
plasmid
packaging
rep
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP7513877A
Other languages
English (en)
Japanese (ja)
Inventor
アール． フロッティ，テレンス
ジェイ． カーター，バリー
ビー． グギーノ，ウィリアム
ソロー，リッキー
Original Assignee
ターゲテッド ジェネティックス コーポレイション
ジョンズ ホップキンズ ユニバーシティ
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 ターゲテッド ジェネティックス コーポレイション, ジョンズ ホップキンズ ユニバーシティ filed Critical ターゲテッド ジェネティックス コーポレイション
Publication of JPH09509564A publication Critical patent/JPH09509564A/ja
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/12—Drugs for disorders of the metabolism for electrolyte homeostasis
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Pulmonology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Compression, Expansion, Code Conversion, And Decoders (AREA)
  • Compression Or Coding Systems Of Tv Signals (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP7513877A 1993-11-09 1994-11-03 高力価組換えａａｖベクターの生成 Pending JPH09509564A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US14933293A 1993-11-09 1993-11-09
US08/149,332 1993-11-09
PCT/US1994/012587 WO1995013365A1 (en) 1993-11-09 1994-11-03 Generation of high titers of recombinant aav vectors

Publications (1)

Publication Number Publication Date
JPH09509564A true JPH09509564A (ja) 1997-09-30

Family

ID=22529805

Family Applications (2)

Application Number Title Priority Date Filing Date
JP7513877A Pending JPH09509564A (ja) 1993-11-09 1994-11-03 高力価組換えａａｖベクターの生成
JP2005000789A Pending JP2005110694A (ja) 1993-11-09 2005-01-05 高力価組換えａａｖベクターの生成

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2005000789A Pending JP2005110694A (ja) 1993-11-09 2005-01-05 高力価組換えａａｖベクターの生成

Country Status (11)

Country Link
US (1) US5658776A (https=)
EP (1) EP0733103B1 (https=)
JP (2) JPH09509564A (https=)
AT (1) ATE260980T1 (https=)
AU (1) AU688428B2 (https=)
CA (1) CA2176117C (https=)
DE (1) DE69433592T2 (https=)
DK (1) DK0733103T3 (https=)
ES (1) ES2216005T3 (https=)
PT (1) PT733103E (https=)
WO (1) WO1995013365A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2001500361A (ja) * 1994-12-16 2001-01-16 アヴァイジェン インコーポレーテッド 標的細胞への遺伝子の輸送および組込みのためのアデノ随伴由来ベクター系

Families Citing this family (349)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2187626C (en) 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
WO1996012010A1 (de) * 1994-10-13 1996-04-25 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Bereitstellung von rep-negativen aav-mutanten und hierfür verwendbare zellen
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US6342390B1 (en) 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
US6326356B1 (en) 1996-10-18 2001-12-04 Board Of Regents, The University Of Texas System Suppression of neu overexpression using a mini-E1A gene
AU707866B2 (en) * 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5795587A (en) * 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
WO1996026742A1 (en) 1995-02-28 1996-09-06 The Regents Of The University Of California Gene transfer-mediated angiogenesis therapy
US6752987B1 (en) 1995-02-28 2004-06-22 The Regents Of The University Of California Adenovirus encoding human adenylylcyclase (AC) VI
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US20030069173A1 (en) * 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
WO1997006272A2 (en) * 1995-08-03 1997-02-20 Avigen, Inc. High efficiency helper system for aav vector production
US6027931A (en) * 1995-08-03 2000-02-22 Avigen, Inc. High-efficiency AA V helper functions
US5622856A (en) * 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6086913A (en) * 1995-11-01 2000-07-11 University Of British Columbia Liposomal delivery of AAV vectors
DK0870044T3 (da) * 1995-12-01 2004-08-02 Crucell Holland Bv Reguleret proteinekspression i stabilt transfekterede pattedyrsceller
WO1997032990A1 (en) * 1996-03-04 1997-09-12 Targeted Genetics Corporation Methods for transducing cells in blood vessels using recombinant aav vectors
US6245735B1 (en) 1996-07-29 2001-06-12 The Brigham And Women's Hospital, Inc. Methods and products for treating pseudomonas infection
AU722624B2 (en) * 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
IL128736A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
BR9712900A (pt) 1996-11-06 2000-11-28 Univ California Enzima liberadora de receptor de fator de necrose de tumor isolado, composições que compreendem a enzima e métodos de utilização da mesma
AU741605B2 (en) * 1996-12-18 2001-12-06 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
EP0856585A1 (en) * 1997-01-29 1998-08-05 Introgene B.V. A conditional replication and expression system
US6403370B1 (en) 1997-02-10 2002-06-11 Genstar Therapeutics Corporation Oncolytic/immunogenic complementary-adenoviral vector system
EP1005561A4 (en) * 1997-03-21 2004-03-31 Enzo Therapeutics Inc A Fully VECTOR, VIRAL VECTORS AND PACKAGING CELL LINES TO THEIR REPRODUCTION
US8703480B1 (en) 1997-03-21 2014-04-22 Enzo Therapeutics, Inc. Biological function effecting viral vectors and chimeric cells useful as packaging cell lines and target cells
AU744445B2 (en) * 1997-04-24 2002-02-21 University Of Washington Targeted gene modification by parvoviral vectors
WO1998052615A1 (en) * 1997-05-22 1998-11-26 Uab Research Foundation Controlling immune response to specific antigens
US6689605B1 (en) * 1997-05-22 2004-02-10 Uab Research Foundation Controlling immune response to specific antigens
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2303768C (en) * 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant aav
DE69840948D1 (de) * 1997-10-21 2009-08-13 Targeted Genetics Corp Invertierte terminale repeats von aav, die für die transkription aktiviert sind, zur verwendung in rekombinanten aav-vektoren
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
WO1999020779A1 (en) * 1997-10-21 1999-04-29 Targeted Genetics Corporation Amplifiable adeno-associated virus (aav) packaging cassettes for the production of recombinant aav vectors
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
US6294379B1 (en) * 1998-02-25 2001-09-25 The Regents Of The University Of California Efficient AAV vectors
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US20060183228A1 (en) * 1998-03-24 2006-08-17 Enzo Therapeutics, Inc. Viral vectors with surface or envelope components
ES2219011T3 (es) 1998-04-24 2004-11-16 University Of Florida Vector viral adeno-asociado recombinante que codifica alfa-1 antitripsina para terapia genica.
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
WO1999061643A1 (en) * 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
EP1097244A4 (en) * 1998-07-22 2002-08-07 Immusol Inc SUBSTANTIALLY COMPLETE RIBOZYME BANKS
DK1930418T3 (da) 1998-09-04 2015-07-13 Genzyme Corp Fremgangsmåder til frembringelse af hjælperfri præparater med høj titer af frigjorte rekombinante AAV-vektorer
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US7129043B1 (en) * 1998-10-22 2006-10-31 Duke University Methods of screening for risk of proliferative disease and methods for the treatment of proliferative disease
DK1129064T3 (da) 1998-11-12 2008-04-28 Invitrogen Corp Transfektionsreagenser
US20040009535A1 (en) 1998-11-27 2004-01-15 Celltech R&D, Inc. Compositions and methods for increasing bone mineralization
DE69933044T3 (de) 1998-11-27 2016-11-24 Ucb Pharma, S.A. Zusammensetzungen und verfahren zur erhöhung der knochenmineralisierung
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
EP1939300A1 (en) 1999-05-28 2008-07-02 Targeted Genetics Corporation Methods and compositions for lowering the level of tumor necrosis factor (TNF) in TNF-associated disorders
DE60040147D1 (de) * 1999-05-28 2008-10-16 Targeted Genetics Corp Methoden und zusammensetzungen zur reduktion des tumor-nekrosis-faktor-spiegels (tnf) bei tnf-assoziierten erkrankungen
EP1916258B1 (en) 1999-08-09 2014-04-23 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
PT1916258E (pt) 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
AU7879000A (en) * 1999-10-12 2001-04-23 University Of North Carolina At Chapel Hill, The Adeno-associated virus vectors encoding factor viii and methods of using the same
CA2395839A1 (en) 1999-12-27 2001-07-05 The Regents Of The University Of California Gene therapy for congestive heart failure
AU3988401A (en) * 2000-02-25 2001-09-03 Ludwig Inst Cancer Res Materials and methods involving hybrid vascular endothelial growth factor dnas and proteins
AU2001280809A1 (en) * 2000-07-28 2002-02-13 University Of Maryland, Baltimore Accessory cholera enterotoxin and analogs thereof as activators of calcium dependent chloride channel
US7507542B2 (en) 2001-05-08 2009-03-24 Ucb Sa Method for regulating immune function using the FOXP3 protein
US7119172B2 (en) 2001-05-21 2006-10-10 The Brigham And Women's Hospital, Inc. P. aeruginosa mucoid exopolysaccharide specific binding peptides
US6962813B2 (en) 2001-05-21 2005-11-08 The Brigham And Women's Hospital, Inc. P. aeruginosa mucoid exopolysaccharide specific binding peptides
US6844192B2 (en) 2001-06-29 2005-01-18 Wake Forest University Adenovirus E4 protein variants for virus production
US7332276B2 (en) * 2002-03-01 2008-02-19 Celltech R&D, Inc. Methods to increase or decrease bone density
WO2004075861A2 (en) * 2003-02-26 2004-09-10 Children's Hospital, Inc. Recombinant adeno-associated virus production
ES2562490T3 (es) 2003-05-01 2016-03-04 Genzyme Corporation Terapia génica para trastornos neurometabólicos
US20070037284A1 (en) * 2003-06-04 2007-02-15 Enzo Therapeutics, Inc. Vectors for expressing exogenous gene or exogenous nucleic acid sequences
US9532994B2 (en) 2003-08-29 2017-01-03 The Regents Of The University Of California Agents and methods for enhancing bone formation by oxysterols in combination with bone morphogenic proteins
WO2005025517A2 (en) * 2003-09-12 2005-03-24 Vertex Pharmaceuticals Incorporated Animal model for protease activity and liver damage
WO2005087808A2 (en) 2004-03-05 2005-09-22 Ludwig Institute For Cancer Research Growth factor binding constructs materials and methods
US7582442B2 (en) 2004-03-16 2009-09-01 The Regents Of The University Of Michigan Methods and compositions for using aleveolar macrophage phospholipase A2
US7319015B2 (en) * 2004-03-16 2008-01-15 The Regents Of The University Of Michigan Methods and compositions for using alveolar macrophage phospholipase A2
US20090233986A1 (en) * 2004-07-27 2009-09-17 Mount Sinai School Of Medicine Methods and compositions for using sax2
FI20050753A7 (fi) 2004-09-03 2006-03-04 Licentia Oy Uudet peptidit
WO2006101629A2 (en) * 2005-02-17 2006-09-28 Vertex Pharmaceuticals Incorporated SODIUM CHANNEL PROTEIN TYPE III α-SUBUNIT SPLICE VARIANT
PL1879623T3 (pl) * 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
EP1879624B1 (en) 2005-05-02 2011-09-21 Genzyme Corporation Gene therapy for neurometabolic disorders
EP1919944B1 (en) 2005-08-15 2011-03-23 Vegenics Pty Ltd Modified vegf and pdgf with improved angiogenic properties
US20080200408A1 (en) * 2005-09-30 2008-08-21 Mccormack Kenneth Deletion mutants of tetrodotoxin-resistant sodium channel alpha subunit
US7972813B2 (en) * 2005-09-30 2011-07-05 Vertex Pharmaceuticals Incorporated Tetrodotoxin-resistant sodium channel alpha subunit
DE602005023550D1 (de) 2005-12-14 2010-10-21 Licentia Ltd Verwendungen eines neurotrophischen Faktors
WO2007089780A2 (en) * 2006-01-30 2007-08-09 Licentia, Ltd. Bmx/etk tyrosine kinase gene therapy materials and methods
JP5706602B2 (ja) 2006-02-08 2015-04-22 ジェンザイム・コーポレーション ニーマンピック病ａ型に対する遺伝子治療
US9670244B2 (en) 2006-02-27 2017-06-06 The Regents Of The University Of California Oxysterol compounds and the hedgehog pathway
AU2007254243B2 (en) 2006-05-17 2013-06-13 Ludwig Institute For Cancer Research Targeting VEGF-B regulation of fatty acid transporters to modulate human diseases
WO2007146046A2 (en) 2006-06-07 2007-12-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
US20100035973A1 (en) * 2006-07-17 2010-02-11 Nationwide Children's Hospital, Inc. Disruption of programmed death 1 (pd-1) ligand to adjuvant adeno-associated virus vector vaccines
PL2066791T3 (pl) 2006-10-03 2013-02-28 Genzyme Corp Terapia Genowa do leczenia stwardnienia zanikowego bocznego i innych zaburzeń rdzenia kręgowego
CA2693178C (en) 2006-11-29 2018-12-04 Nationwide Children's Hospital, Inc. Myostatin inhibition for enhancing muscle and/or improving muscle function
WO2009073186A1 (en) 2007-12-03 2009-06-11 The Regents Of The University Of California Oxsterols for activation of hedgehog signaling, osteoinduction, antiadipogenesis, and wnt signaling
EP2154969B1 (en) 2007-05-16 2015-11-18 The Brigham and Women's Hospital, Inc. Treatment of synucleinopathies
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
FI20070808A0 (fi) 2007-10-25 2007-10-25 Mart Saarma GDNF:n silmukointivariantit ja niiden käytöt
US20090196854A1 (en) * 2008-02-04 2009-08-06 Kytos Biosystems S.A. Methods and compositions for use of crl 5803 cells for expression of biotherapeutics and encapsulated cell-based delivery
FI20080326A0 (fi) 2008-04-30 2008-04-30 Licentia Oy Neurotroofinen tekijä MANF ja sen käytöt
EP2318036B1 (en) * 2008-06-30 2015-06-03 The Regents of the University of Michigan Lysosomal phospholipase a2 (lpla2) activity as a diagnostic and therapeutic target for identifying and treating systemic lupus erythematosis
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
CN103492016A (zh) 2010-11-23 2014-01-01 普莱萨格生命科学公司 用于实体递送的治疗方法和组合物
WO2012145509A2 (en) 2011-04-19 2012-10-26 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors, and viruses
US10196636B2 (en) 2011-04-21 2019-02-05 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
ES2702496T3 (es) 2011-04-21 2019-03-01 Nationwide Childrens Hospital Inc Productos de virus recombinante y procedimientos para la inhibición de la expresión de la miotilina
CA2840175C (en) 2011-05-19 2021-11-16 The Regents Of The University Of Michigan Integrin alpha-2 binding agents and use thereof to inhibit cancer cell proliferation
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
AU2012286994B2 (en) 2011-07-25 2017-08-10 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of DUX4
KR102000141B1 (ko) 2011-10-27 2019-07-15 웰스테이트 옵탈믹스 코퍼레이션 간상세포 유래 원추세포 생존능 인자를 인코딩하는 벡터
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
US9365496B2 (en) 2011-11-30 2016-06-14 Ludwig Institute For Cancer Research iNKT cell modulators and methods of using the same
GB201120860D0 (en) 2011-12-05 2012-01-18 Cambridge Entpr Ltd Cancer immunotherapy
CN108588025A (zh) 2012-02-17 2018-09-28 斯格本斯眼科研究所 人视网膜祖细胞的表型谱
KR20150013232A (ko) 2012-05-07 2015-02-04 더 리전트 오브 더 유니버시티 오브 캘리포니아 골형성 및 헤지호그 신호전달을 유도하고 지방생성을 억제하는 옥시스테롤 유사체 oxy133
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
AU2013296425B2 (en) 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
CA2884245C (en) 2012-09-06 2023-03-14 The University Of Chicago Antisense polynucleotides to induce exon skipping and methods of treating dystrophies
AU2013315007A1 (en) 2012-09-17 2015-04-09 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
PT2956476T (pt) 2013-02-18 2020-02-21 Vegenics Pty Ltd Moléculas de ligação e usos destas
AU2014251099B2 (en) 2013-04-08 2019-01-17 The University Of Kansas Chimeric adeno-associated virus/ bocavirus parvovirus vector
KR102413498B1 (ko) 2013-04-20 2022-06-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달
CN105263500A (zh) 2013-05-02 2016-01-20 加利福尼亚大学董事会 骨选择性成骨性氧固醇-骨靶向剂
CN105377039A (zh) 2013-05-15 2016-03-02 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
WO2014191630A2 (en) 2013-05-28 2014-12-04 Helsingin Yliopisto Non-human animal model encoding a non-functional manf gene
LT3702466T (lt) 2013-08-27 2023-02-27 Research Institute At Nationwide Children's Hospital Produktai ir būdai, skirti šoninės amiotrofinės sklerozės gydymui
EP3039129B1 (en) * 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
WO2015069647A1 (en) 2013-11-05 2015-05-14 The Research Institute At Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS
WO2015142984A1 (en) 2014-03-18 2015-09-24 Washington University Methods and compositions for red-shifted chromophore substitution for optogenetic applications
WO2016011203A1 (en) 2014-07-15 2016-01-21 Life Technologies Corporation Compositions with lipid aggregates and methods for efficient delivery of molecules to cells
CA2957661A1 (en) 2014-08-09 2016-02-18 Kevin FLANIGAN Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
WO2016057975A2 (en) 2014-10-10 2016-04-14 Research Institute At Nationwide Children's Hospital Guided injections for aav gene transfer to muscle
EP3215602B1 (en) 2014-11-05 2019-12-25 The Research Institute at Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
CA2975447C (en) 2015-01-30 2021-02-23 The Regents Of The University Of California Spinal subpial gene delivery system
MA41451A (fr) 2015-02-04 2017-12-12 Univ Washington Constructions anti-tau
AU2016225178B2 (en) 2015-02-23 2022-05-05 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3280451B8 (en) 2015-04-08 2019-09-25 The United States of America, as represented by the Secretary, Department of Health and Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
TN2017000427A1 (en) 2015-04-08 2019-04-12 Univ Northwestern Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping
EP3292206B8 (en) 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
HK1256341A1 (zh) 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
US10017832B2 (en) 2015-08-25 2018-07-10 Washington University Compositions and methods for site specific recombination at asymmetric sites
EA201890543A1 (ru) 2015-09-17 2018-08-31 Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл Способы и материалы для генной терапии galgt2
US20190048340A1 (en) 2015-09-24 2019-02-14 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
JP2019507579A (ja) 2015-10-28 2019-03-22 クリスパー セラピューティクス アーゲー デュシェンヌ型筋ジストロフィーの処置のための材料および方法
CA2999649A1 (en) 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
CA3005633C (en) 2015-11-16 2023-11-21 Research Institute Of Nationwide Children's Hospital Materials and methods for treatment of titin-based myopathies and other titinopathies
CA3006618A1 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
BR112018012894A2 (en) 2015-12-23 2018-12-04 Crispr Therapeutics Ag Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration
WO2017120589A1 (en) 2016-01-08 2017-07-13 Washington University Compositions comprising chemerin and methods of use thereof
WO2017134529A1 (en) 2016-02-02 2017-08-10 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
WO2017141109A1 (en) 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
US11066456B2 (en) 2016-02-25 2021-07-20 Washington University Compositions comprising TREM2 and methods of use thereof
AU2017222653B2 (en) 2016-02-26 2023-06-01 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inducing DUX4 exon skipping
CA3198936A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
US11083799B2 (en) 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
AU2017241534A1 (en) 2016-03-28 2018-10-04 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability
BR112018070249A2 (pt) 2016-04-02 2019-01-29 Research Institute At Nationwide Children's Hospital sistema promotor de u6 modificada para expressão específica de tecido
PT3442600T (pt) 2016-04-15 2024-05-09 Res Inst Nationwide Childrens Hospital Distribuição de vetor de vírus adeno-associado de bsarcoglicano e microrna-29 e o tratamento da distrofia muscular
AU2017250791B2 (en) 2016-04-15 2022-11-24 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
DK3445388T3 (da) 2016-04-18 2024-06-03 Vertex Pharma Materialer og fremgangsmåder til behandling af hæmoglobinopatier
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
EP3478828B1 (en) 2016-06-29 2024-09-04 CRISPR Therapeutics AG Materials and methods for treatment of friedreich ataxia and other related disorders
WO2018002762A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
JP7305534B2 (ja) 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
AU2017292173B2 (en) 2016-07-06 2022-01-13 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
WO2018020323A2 (en) 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
EP3541946A1 (en) 2016-11-15 2019-09-25 Regents Of The University Of Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
BR112019009834B1 (pt) 2016-11-17 2022-05-17 Ohio State Innovation Foundation Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav
JP7206214B2 (ja) 2016-12-13 2023-01-17 シアトル チルドレンズ ホスピタル （ディービーエイ シアトル チルドレンズ リサーチ インスティテュート） インビトロ及びインビボで操作された細胞において発現された化学誘導シグナル伝達複合体の外因性薬物活性化の方法
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
WO2018150271A1 (en) 2017-02-17 2018-08-23 Lonza Ltd. Mammalian cells for producing adeno-associated viruses
US20200040061A1 (en) 2017-02-22 2020-02-06 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
WO2018154462A2 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
JP2020508056A (ja) 2017-02-22 2020-03-19 クリスパー・セラピューティクス・アクチェンゲゼルシャフトＣＲＩＳＰＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＡＧ 遺伝子編集のための組成物および方法
EP3585899A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders
US11559588B2 (en) 2017-02-22 2023-01-24 Crispr Therapeutics Ag Materials and methods for treatment of Spinocerebellar Ataxia Type 1 (SCA1) and other Spinocerebellar Ataxia Type 1 Protein (ATXN1) gene related conditions or disorders
MX2019011046A (es) 2017-03-17 2019-10-17 Res Inst Nationwide Childrens Hospital Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular.
WO2019012336A2 (en) 2017-03-17 2019-01-17 Newcastle University ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
JP2020513831A (ja) 2017-03-24 2020-05-21 ザ ユニバーシティ コート オブ ザ ユニバーシティ オブ エジンバラ ＭｅＣＰ２発現カセット
MX2019013514A (es) 2017-05-12 2020-01-20 Crispr Therapeutics Ag Materiales y metodos para modificar celulas por ingenieria genetica y usos de los mismos en inmunooncologia.
US12116577B2 (en) 2017-06-20 2024-10-15 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
US20200370069A1 (en) 2017-07-08 2020-11-26 Genethon Treatment of spinal muscular atrophy
CN110892064A (zh) 2017-07-25 2020-03-17 牛津遗传学有限公司 腺病毒载体
AU2018345772B2 (en) 2017-10-02 2025-04-17 Research Institute At Nationwide Children's Hospital MiRNA detargeting system for tissue specific interference
US20190247517A1 (en) 2017-10-17 2019-08-15 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing for hemophilia a
JP7213238B2 (ja) 2017-10-18 2023-01-26 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達
AU2018351528B2 (en) 2017-10-20 2025-02-27 Research Institute At Nationwide Children's Hospital Methods and materials for NT-3 gene therapy
WO2019081982A1 (en) 2017-10-26 2019-05-02 Crispr Therapeutics Ag SUBSTANCES AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
MX2020004830A (es) 2017-11-08 2020-11-11 Avexis Inc Medios y metodo para preparar vectores virales y usos de los mismos.
US20210363521A1 (en) 2017-11-09 2021-11-25 Vertex Pharmaceuticals Incorporated CRISPR/CAS Systems For Treatment of DMD
MA50877A (fr) 2017-11-21 2020-09-30 Bayer Healthcare Llc Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante
MX2020005470A (es) 2017-11-27 2020-11-09 Coda Biotherapeutics Inc Composiciones y metodos para enfermedades neurologicas.
JP2021506251A (ja) 2017-12-14 2021-02-22 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系、ならびにゲノム編集および他の適用におけるその使用
WO2019123429A1 (en) 2017-12-21 2019-06-27 Casebia Therapeutics Llp Materials and methods for treatment of usher syndrome type 2a
WO2019123430A1 (en) 2017-12-21 2019-06-27 Casebia Therapeutics Llp Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
CA3088180A1 (en) 2018-01-12 2019-07-18 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting transferrin
MX2020007876A (es) 2018-01-31 2020-12-03 Res Inst Nationwide Childrens Hospital Terapia genica para la distrofia muscular de cinturas tipo 2c.
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
MA51787A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes de traitement d'hémoglobinopathies
WO2019161310A1 (en) 2018-02-16 2019-08-22 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing by targeting fibrinogen-alpha
JP7550648B2 (ja) 2018-03-19 2024-09-13 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
WO2019204668A1 (en) 2018-04-18 2019-10-24 Casebia Therapeutics Limited Liability Partnership Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
CA3091490A1 (en) 2018-04-27 2019-10-31 Seattle Children's Hospital (dba Seattle Children's Research Institute) Rapamycin resistant cells
EP3796980A4 (en) 2018-05-22 2022-03-23 The Brigham & Women's Hospital, Inc. NEW THERAPY FOR ALZHEIMER'S DISEASE
CN112601557B (zh) 2018-06-08 2025-06-06 诺华股份有限公司 用于测量药物产品效力的基于细胞的测定
AU2019284362A1 (en) 2018-06-11 2021-01-07 University Of Florida Research Foundation, Inc. Materials and methods for treating stress-related disorders and cancer
GB201809588D0 (en) 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
WO2019246125A1 (en) 2018-06-18 2019-12-26 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
CN120393053A (zh) 2018-06-18 2025-08-01 全国儿童医院研究所 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
EP3841209A4 (en) 2018-08-22 2023-01-04 Research Institute at Nationwide Children's Hospital RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITING DYSTROPHIA MYOTONICA PROTEIN KINASE EXPRESSION AND/OR INTERFERING WITH A TRINUCLEOTIDE REPEAT EXPANSION IN THE 3'-UNTRANSLATED REGION OF THE DMPK GENE
JP2021534794A (ja) 2018-08-29 2021-12-16 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法
CN111315407B (zh) 2018-09-11 2023-05-02 上海市公共卫生临床中心 一种广谱抗流感疫苗免疫原及其应用
JP7520826B2 (ja) 2018-10-17 2024-07-23 クリスパー・セラピューティクス・アクチェンゲゼルシャフト 導入遺伝子を送達するための組成物および方法
CA3121191A1 (en) 2018-11-28 2020-06-04 Crispr Therapeutics Ag Optimized mrna encoding cas9 for use in lnps
WO2020113034A1 (en) 2018-11-30 2020-06-04 Avexis, Inc. Aav viral vectors and uses thereof
EP3898995A1 (en) 2018-12-21 2021-10-27 Genethon Expression cassettes for gene therapy vectors
AU2019419494B2 (en) 2018-12-31 2026-03-26 Research Institute At Nationwide Children's Hospital DUX4 RNA silencing using RNA targeting CRISPR-Cas13b
KR20210124299A (ko) 2019-02-04 2021-10-14 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Cln6 폴리뉴클레오티드의 아데노-관련 바이러스 전달
KR20210124300A (ko) 2019-02-04 2021-10-14 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Cln3 폴리뉴클레오티드의 아데노-관련 바이러스 전달
US11529427B2 (en) 2019-02-15 2022-12-20 Crispr Therapeutics Ag Gene editing for hemophilia A with improved factor VIII expression
MX2021010356A (es) 2019-02-26 2022-03-04 Res Inst Nationwide Childrens Hospital SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR.
WO2020186059A2 (en) 2019-03-12 2020-09-17 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
WO2020214737A1 (en) 2019-04-15 2020-10-22 Sanford Research Gene therapy for treating or preventing visual effects in batten disease
US20220241436A1 (en) 2019-04-15 2022-08-04 University Of Iowa Research Foundation Compositions and methods for treatment of cystic fibrosis
EP3955970A1 (en) 2019-04-15 2022-02-23 University Of Iowa Research Foundation Methods and compositions for transgene expression
EP3966327A1 (en) 2019-05-08 2022-03-16 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
JP2022533645A (ja) 2019-05-17 2022-07-25 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル グリコシドヒドロラーゼ酵素を使用する網膜細胞への遺伝子治療ベクターの送達の改善
WO2020242892A1 (en) 2019-05-24 2020-12-03 The Brigham And Women's Hospital, Inc. Gene therapy for alzheimer's disease
EP3990636A1 (en) 2019-06-28 2022-05-04 CRISPR Therapeutics AG Materials and methods for controlling gene editing
WO2021014428A1 (en) 2019-07-25 2021-01-28 Novartis Ag Regulatable expression systems
PT4017871T (pt) 2019-08-21 2024-04-22 Res Inst Nationwide Childrens Hospital Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular
JP7781052B2 (ja) 2019-08-21 2025-12-05 トレイムス バイオ， インコーポレイテッド 神経学的疾患のための組成物および方法
WO2021072115A1 (en) 2019-10-08 2021-04-15 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
AU2020366242A1 (en) 2019-10-18 2022-05-26 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of disorders associated mutations in the IRF2BPL gene
EP4045092A1 (en) 2019-10-18 2022-08-24 Research Institute at Nationwide Children's Hospital Gene therapy targeting cochlear cells
JP7684296B2 (ja) 2019-11-22 2025-05-27 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法
IL293285A (en) 2019-11-29 2022-07-01 Paros Bio Inc Gene therapy for neurodegenerative disorders
AU2020408225A1 (en) 2019-12-20 2022-07-14 Research Institute At Nationwide Children's Hospital Optimized gene therapy for targeting muscle in muscle diseases
KR20220155999A (ko) 2020-02-18 2022-11-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 X-연관 장애의 치료에서의 miRNA의 AAV-매개 표적화
EP4135776A1 (en) 2020-04-14 2023-02-22 Genethon Vectors for the treatment of acid ceramidase deficiency
EP4164668A1 (en) 2020-06-15 2023-04-19 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies
US20230242941A1 (en) 2020-06-30 2023-08-03 Eric Yuen Methods and compositions for administering recombinant viral vectors
US20230374483A1 (en) 2020-07-08 2023-11-23 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
WO2022018638A1 (en) 2020-07-21 2022-01-27 Crispr Therapeutics Ag Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
WO2022047201A1 (en) 2020-08-27 2022-03-03 University Of Iowa Research Foundation Gene knock-out for treatment of glaucoma
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
TW202541851A (zh) 2020-09-08 2025-11-01 美商薩羅塔治療公司 表現γ-肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療
WO2022060841A2 (en) 2020-09-15 2022-03-24 Research Institute At Nationwide Children's Hospital Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
EP4217375A1 (en) 2020-09-28 2023-08-02 Research Institute at Nationwide Children's Hospital Products and methods for treating muscular dystrophy
US20230392134A1 (en) 2020-09-30 2023-12-07 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis
AU2021358957A1 (en) 2020-10-07 2023-06-08 AskBio Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21
IL303230A (en) 2020-11-30 2023-07-01 Res Inst Nationwide Childrens Hospital Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
AU2021400745A1 (en) 2020-12-17 2023-07-20 Vertex Pharmaceuticals Incorporated Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
JP2024505885A (ja) 2021-01-27 2024-02-08 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル リソソーム酸リパーゼ欠損症（ｌａｌ－ｄ）の治療のための材料及び方法
WO2022169922A1 (en) 2021-02-03 2022-08-11 Research Institute At Nationwide Children's Hospital Compositions and methods for treating disease associated with dux4 overexpression
WO2022170082A1 (en) 2021-02-05 2022-08-11 Regents Of The University Of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses
WO2022170038A1 (en) 2021-02-05 2022-08-11 Amicus Therapeutics, Inc. Adeno-associated virus delivery of cln3 polynucleotide
WO2022187571A1 (en) 2021-03-04 2022-09-09 Research Institute At Nationwide Children's Hospital Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
CN115427561B (zh) 2021-03-09 2024-06-04 辉大(上海)生物科技有限公司 工程化CRISPR/Cas13系统及其用途
EP4323010A1 (en) 2021-04-13 2024-02-21 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus encoding methyl-cpg binding protein 2 for treating pitt hopkins syndrome via intrathecal delivery
US12275941B2 (en) 2021-04-15 2025-04-15 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of dynamin-1 variants
US20240115738A1 (en) 2021-04-15 2024-04-11 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis
EP4326752A1 (en) 2021-04-23 2024-02-28 Research Institute at Nationwide Children's Hospital Products and methods for treating muscular dystrophy
EP4334447A1 (en) 2021-05-07 2024-03-13 UCL Business Ltd Abca4 genome editing
CA3218350A1 (en) 2021-05-17 2022-11-24 Sarepta Therapeutics, Inc. Production of recombinant aav vectors for treating muscular dystrophy
EP4108263A3 (en) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
EP4101928A1 (en) 2021-06-11 2022-12-14 Bayer AG Type v rna programmable endonuclease systems
US20240141312A1 (en) 2021-06-11 2024-05-02 Bayer Aktiengesellschaft Type v rna programmable endonuclease systems
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
KR20240095165A (ko) 2021-08-11 2024-06-25 솔리드 바이오사이언시즈 인크. 근이영양증의 치료
EP4144841A1 (en) 2021-09-07 2023-03-08 Bayer AG Novel small rna programmable endonuclease systems with impoved pam specificity and uses thereof
WO2023042104A1 (en) 2021-09-16 2023-03-23 Novartis Ag Novel transcription factors
CA3234702A1 (en) 2021-10-07 2023-04-13 Research Institute At Nationwide Children's Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
US20240424137A1 (en) 2021-10-08 2024-12-26 Amicus Therapeutics, Inc. Biomarkers For Lysosomal Storage Diseases
JP2023059858A (ja) 2021-10-15 2023-04-27 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
EP4186919A1 (en) 2021-11-30 2023-05-31 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
EP4198047A1 (en) 2021-12-16 2023-06-21 Genethon Fukutin related protein gene transfer increase using modified itr sequences
EP4198048A1 (en) 2021-12-16 2023-06-21 Genethon Calpain-3 gene transfer increase using modified itr sequences
EP4198134A1 (en) 2021-12-16 2023-06-21 Genethon Gamma-sarcoglycan gene transfer increase using modified itr sequences
EP4198046A1 (en) 2021-12-16 2023-06-21 Genethon Alpha-sarcoglycan gene transfer increase using modified itr sequences
CA3242989A1 (en) 2021-12-21 2023-06-29 Research Institute At Nationwide Children's Hospital MATERIALS AND METHODS FOR THE TREATMENT OF MUSCULAR BELLY DYSTROPHY
WO2023118068A1 (en) 2021-12-23 2023-06-29 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2023166425A1 (en) 2022-03-01 2023-09-07 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions
JP2025508910A (ja) 2022-03-03 2025-04-10 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｅｉｆ２ｂ５の変異及びそれに起因する疾患の治療のための材料及び方法
CN119562762A (zh) 2022-04-04 2025-03-04 加利福尼亚大学董事会 遗传互补组合物和方法
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
US20250340887A1 (en) 2022-06-08 2025-11-06 Research Institute At Nationwide Children's Hospital Products and methods for treating diseases or conditions associated with mutant or pathogenic kcnq3 expression
AU2023284048A1 (en) 2022-06-10 2024-11-14 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
AU2023303015A1 (en) 2022-07-06 2025-01-23 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery of cln1 polynucleotide
WO2024035782A1 (en) 2022-08-10 2024-02-15 Aav Gene Therapeutics, Inc. Aav-mediated intramuscular delivery of insulin
KR20250090396A (ko) 2022-09-23 2025-06-19 사렙타 쎄러퓨틱스 인코퍼레이티드 근이영양증 치료를 위한 재조합 aav 벡터
AU2023360998A1 (en) 2022-10-11 2025-05-22 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)
WO2024086747A1 (en) 2022-10-19 2024-04-25 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2024092171A1 (en) 2022-10-26 2024-05-02 University Of Iowa Research Foundation Method to deliver large genes using virus and a dna recombination system
EP4608856A1 (en) 2022-10-27 2025-09-03 CARGO Therapeutics, Inc. Compositions and methods for improved immunotherapies
IL321270A (en) 2022-12-13 2025-08-01 Bayer Ag Engineered RNA-type programmable endonucleases and their uses
WO2024151982A1 (en) 2023-01-13 2024-07-18 Amicus Therapeutics, Inc. Gene therapy constructs for the treatment of pompe disease
JP2026504375A (ja) 2023-02-01 2026-02-05 サレプタ セラピューティクス， インコーポレイテッド ｒＡＡＶの製造方法
WO2024168276A2 (en) 2023-02-09 2024-08-15 Cargo Therapeutics, Inc. Compositions and methods for immunotherapies
CN121285568A (zh) 2023-03-30 2026-01-06 制药五有限责任公司 编码视杆细胞源性视锥细胞活力因子和人IgK信号序列的载体
WO2024220592A2 (en) 2023-04-18 2024-10-24 Research Institute At Nationwide Children's Hospital, Inc. Gene therapy for treating limb girdle muscular dystrophy r9 and congenital muscular dystrophy 1c
AU2024265701A1 (en) 2023-05-02 2025-12-04 Research Institute At Nationwide Children's Hospital A modular system to convert therapeutic microrna expression cassettes from polymerase iii-based to polymerase ii-based promoters
AU2024265705A1 (en) 2023-05-02 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases
WO2024229115A1 (en) 2023-05-02 2024-11-07 Research Institute At Nationwide Children's Hospital Treatment of multiple sclerosis using nt-3 gene therapy
WO2024228167A1 (en) 2023-05-03 2024-11-07 Iox Therapeutics Inc. Inkt cell modulator liposomal compositions and methods of use
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
IL325133A (en) 2023-06-07 2026-02-01 Res Inst Nationwide Childrens Hospital Gene therapy for lysosomal acid lipase deficiency (LAL-D)
AU2024305531A1 (en) 2023-06-13 2026-01-22 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom
AU2024301706A1 (en) 2023-07-21 2026-02-05 Crispr Therapeutics Ag Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene
EP4512403A1 (en) 2023-08-22 2025-02-26 Friedrich-Schiller-Universität Jena Neuropeptide b and w-receptor as a target for treating mood disorders and/or chronic stress
TW202521691A (zh) 2023-10-06 2025-06-01 美商藍岩醫療公司 經工程化之v型rna可程式核酸內切酶及其用途
WO2025096498A1 (en) 2023-10-30 2025-05-08 Research Institute At Nationwide Children's Hospital Compositions and methods for treating diseases or conditions associated with progerin expression
AR134514A1 (es) 2023-12-01 2026-01-21 Gilead Sciences Inc Proteína de fusión anti-fap-ligera y uso de esta
WO2025179121A1 (en) 2024-02-21 2025-08-28 Research Institute At Nationwide Children's Hospital Exon 17-targeted nucleic acids, compositions, and methods for treatment of dystrophin-based myopathies
WO2025186726A1 (en) 2024-03-05 2025-09-12 Crispr Therapeutics Ag Modulating expression of agt (angiotensinogen) gene
WO2025188993A2 (en) 2024-03-07 2025-09-12 Research Institute At Nationwide Children's Hospital Gene therapy for treating gne-related disorders
TW202548023A (zh) 2024-03-14 2025-12-16 美商雅菲納治療公司 用於生產重組腺相關病毒的質體系統
WO2025212838A1 (en) 2024-04-03 2025-10-09 Research Institute At Nationwide Children's Hospital Products and methods for treating diseases or disorders associated with dux4 overexpression
WO2025226343A1 (en) 2024-04-26 2025-10-30 Research Institute At Nationwide Children's Hospital Products and methods to inhibit expression of dynamin-1 variants and replace dynamin-1
WO2025227063A1 (en) 2024-04-26 2025-10-30 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2025235425A1 (en) 2024-05-06 2025-11-13 Research Institute At Nationwide Children's Hospital Improved proviral plasmids
WO2025235491A1 (en) 2024-05-07 2025-11-13 Affinia Therapeutics Inc. Recombinant aav for treatment of cardiac diseases
WO2025240690A2 (en) 2024-05-15 2025-11-20 Research Institute At Nationwide Children's Hospital Products and methods for treating diseases or conditions associated with progerin expression from an aberrant lmna gene
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav
US20260056209A1 (en) 2024-06-14 2026-02-26 Gilead Sciences, Inc. Anti-ccr8 antibodies and uses thereof
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
WO2026072752A1 (en) 2024-09-27 2026-04-02 Pharma Cinq, Llc Rod-derived cone viability factor fusion protein

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) * 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1991018088A1 (en) * 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) * 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
AU1150792A (en) * 1990-12-06 1992-07-08 United States of America, as represented by the Secretary, U.S. Department of Commerce, The Inhibition of human immunodeficiency virus by an adeno-associated virus gene for human cells
PT728214E (pt) * 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2001500361A (ja) * 1994-12-16 2001-01-16 アヴァイジェン インコーポレーテッド 標的細胞への遺伝子の輸送および組込みのためのアデノ随伴由来ベクター系

Also Published As

Publication number Publication date
ES2216005T3 (es) 2004-10-16
ATE260980T1 (de) 2004-03-15
AU1129395A (en) 1995-05-29
JP2005110694A (ja) 2005-04-28
EP0733103A1 (en) 1996-09-25
DE69433592D1 (de) 2004-04-08
DK0733103T3 (da) 2004-07-12
DE69433592T2 (de) 2005-02-10
WO1995013365A1 (en) 1995-05-18
CA2176117A1 (en) 1995-05-18
CA2176117C (en) 2006-01-03
EP0733103A4 (en) 1997-12-17
US5658776A (en) 1997-08-19
PT733103E (pt) 2004-07-30
EP0733103B1 (en) 2004-03-03
AU688428B2 (en) 1998-03-12

Similar Documents

Publication Publication Date Title
JPH09509564A (ja) 高力価組換えａａｖベクターの生成
US6924128B2 (en) Packaging cell lines for generation of high titers of recombinant AAV vectors
AU707866B2 (en) Packaging cell lines for generation of high titers of recombinant AAV vectors
US5252479A (en) Safe vector for gene therapy
US6541258B2 (en) AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
EP1696036B1 (en) Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
US20040087026A1 (en) Host cells for packing a recombinant adeno-associated virus (raav), method for the production and use thereof
WO1996017947A9 (en) Packaging cell lines for generation of high titers of recombinant aav vectors
CA3096293A1 (en) Gene therapy for cns degeneration
JP7598333B2 (ja) 組換えウイルスベクターの製造方法
WO1998027204A9 (en) Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
JP2001506132A (ja) Ａａｖベクターの産生における使用のためのリコンビナーゼ活性化可能ａａｖパッケージングカセット
AU4645697A (en) Aav4 vector and uses thereof
CN110914419A (zh) 糖原贮积病iii的治疗
KR20240045198A (ko) 캡시드 변이체 및 이의 사용 방법
TW202304954A (zh) 用於肌肉萎縮症之基因療法的aavrh74載體
TW202208407A (zh) 使用質膜修復蛋白(dysferlin)雙載體之基因療法
JP2025512534A (ja) カプシドバリアント及びそれを使用する方法
CN117940446A (zh) 衣壳变体及其使用方法
JP2023545384A (ja) 中枢神経系または筋肉送達のための組換えアデノ随伴ウイルス
CN117355603A (zh) 用于肌营养不良的基因治疗的aavrh74载体
CN121086028A (zh) 定向进化构建的aav衣壳变体及其用途
EP4508065A2 (en) Capsid variants and methods of using the same
HK40005500A (en) Adeno-associated virus variant capsids and methods of use thereof