PT733103E - Criacao de elevados titulos de vectores de aav recombinantes - Google Patents

Criacao de elevados titulos de vectores de aav recombinantes

Info

Publication number
PT733103E
PT733103E PT95902421T PT95902421T PT733103E PT 733103 E PT733103 E PT 733103E PT 95902421 T PT95902421 T PT 95902421T PT 95902421 T PT95902421 T PT 95902421T PT 733103 E PT733103 E PT 733103E
Authority
PT
Portugal
Prior art keywords
aav
packaging
vectors
creation
gene therapy
Prior art date
Application number
PT95902421T
Other languages
English (en)
Portuguese (pt)
Inventor
Terence R Flotte
Barrie J Carter
William B Guggino
Rikki Solow
Original Assignee
Targeted Genetics Corp
Univ Johns Hopkins
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 Targeted Genetics Corp, Univ Johns Hopkins filed Critical Targeted Genetics Corp
Publication of PT733103E publication Critical patent/PT733103E/pt

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
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/12Drugs for disorders of the metabolism for electrolyte homeostasis
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • 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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (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)
PT95902421T 1993-11-09 1994-11-03 Criacao de elevados titulos de vectores de aav recombinantes PT733103E (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US14933293A 1993-11-09 1993-11-09

Publications (1)

Publication Number Publication Date
PT733103E true PT733103E (pt) 2004-07-30

Family

ID=22529805

Family Applications (1)

Application Number Title Priority Date Filing Date
PT95902421T PT733103E (pt) 1993-11-09 1994-11-03 Criacao de elevados titulos de vectores de aav recombinantes

Country Status (11)

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

Families Citing this family (344)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5658785A (en) * 1994-06-06 1997-08-19 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
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
EP0796339A1 (en) * 1994-12-06 1997-09-24 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5843742A (en) * 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
US5795587A (en) * 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US6752987B1 (en) 1995-02-28 2004-06-22 The Regents Of The University Of California Adenovirus encoding human adenylylcyclase (AC) VI
CN100569297C (zh) 1995-02-28 2009-12-16 加利福尼亚大学董事会 基因转移介导的血管形成疗法
US20030069173A1 (en) * 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US6051429A (en) * 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV 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
EP0842287B1 (en) * 1995-08-03 2004-02-04 Avigen, Inc. High efficiency helper system for aav vector production
US6086913A (en) * 1995-11-01 2000-07-11 University Of British Columbia Liposomal delivery of AAV vectors
ES2219700T3 (es) * 1995-12-01 2004-12-01 Crucell Holland B.V. Expresion regulada de proteinas en celulas de mamiferos transfectadas de manera estable.
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
EP0931158A1 (en) * 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
AU722375B2 (en) * 1996-09-06 2000-08-03 Trustees Of The University Of Pennsylvania, The Methods using cre-lox for production of recombinant adeno-associated viruses
DE69738887D1 (de) 1996-11-06 2008-09-18 Univ California Tumor necrosis factor rezeptor abspaltendes enzym, dessen zubereitungen und verwendungen
CA2270285A1 (en) * 1996-12-18 1998-06-25 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
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
CA2285666C (en) * 1997-03-21 2010-12-14 Enzo Therapeutics, Inc. Vectors and viral vectors, and packaging cell lines for propagating same
DE69840439D1 (de) * 1997-04-24 2009-02-26 Univ Washington Zielgerichtete genveraenderung mit parvoviralen vektoren
AU7585998A (en) * 1997-05-22 1998-12-11 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
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999011764A2 (en) 1997-09-05 1999-03-11 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
AU9319198A (en) * 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The Methods and vector constructs useful for production of recombinant aav
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with 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
CA2308008A1 (en) * 1997-10-21 1999-04-29 Targeted Genetics Corporation Amplifiable adeno-associated virus (aav) packaging cassettes for the production of recombinant aav vectors
AU756827B2 (en) * 1997-10-21 2003-01-23 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRs) for use with 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
CA2326327C (en) * 1998-04-24 2009-06-16 University Of Florida Recombinant adeno-associated virus vector encoding alpha-1-antitrypsin serum protein
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
US6146874A (en) * 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
CA2335390A1 (en) * 1998-07-22 2000-02-03 Immusol Incorporated Substantially complete ribozyme libraries
EP2942393A1 (en) 1998-09-04 2015-11-11 Genzyme Corporation Methods for generating high titer helper-free preparations of released recombinant aav vectors
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
EP1129064B1 (en) 1998-11-12 2008-01-09 Invitrogen Corporation Transfection reagents
US6395511B1 (en) 1998-11-27 2002-05-28 Darwin Discovery, Ltd. Nucleic acids encoding a novel family of TGF-β binding proteins from humans
US20040009535A1 (en) 1998-11-27 2004-01-15 Celltech R&D, Inc. Compositions and methods for increasing bone mineralization
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
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
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
US6893865B1 (en) * 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
PT1180159E (pt) 1999-05-28 2008-12-05 Targeted Genetics Corp Métodos e composições para abaixamento do nível de factor de necrose tumoral (tnf) nos distúrbios associados a tnf
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
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
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
EP1224312A1 (en) * 1999-10-12 2002-07-24 The University of North Carolina at Chapel Hill Adeno-associated virus vectors encoding factor viii and methods of using the same
AU2460001A (en) 1999-12-27 2001-07-09 Regents Of The University Of California, The Gene therapy for congestive heart failure
WO2001062942A2 (en) * 2000-02-25 2001-08-30 Ludwig Institute For Cancer Research MATERIALS AND METHODS INVOLVING HYBRID VASCULAR ENDOTHELIAL GROWTH FACTOR DNAs AND PROTEINS AND SCREENING METHODS FOR MODULATORS
EP1365786A4 (en) * 2000-07-28 2005-06-08 Michele Trucksis CHOLERA-ENTEROTOXIN AND ITS ANALOGA AS ACTIVATORS OF THE CALCIUM-DEPENDENT CHLORIDE CHANNEL
JP4353701B2 (ja) 2001-05-08 2009-10-28 ダーウィン モレキュラー コーポレイション Foxp3蛋白質を用いた霊長類における免疫機能の調節方法
US7119172B2 (en) 2001-05-21 2006-10-10 The Brigham And Women's Hospital, Inc. P. aeruginosa mucoid exopolysaccharide specific binding peptides
WO2002094854A2 (en) 2001-05-21 2002-11-28 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
AU2003223214B2 (en) 2002-03-01 2008-09-18 Celltech R & D, Inc. Methods to increase or decrease bone density
WO2007068784A1 (en) 2005-12-14 2007-06-21 Licentia Ltd Novel neurotrophic factor protein and uses thereof
US7510872B2 (en) * 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
PL1620133T3 (pl) 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
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
JP2007505603A (ja) * 2003-09-12 2007-03-15 ヴァーテックス ファーマシューティカルズ、 インコーポレイテッド プロテアーゼ活性および肝障害のための動物モデル
ATE507240T1 (de) 2004-03-05 2011-05-15 Vegenics Pty Ltd Materialien und verfahren für wachstumsfaktorbindende konstrukte
US7319015B2 (en) * 2004-03-16 2008-01-15 The Regents Of The University Of Michigan Methods and compositions for using alveolar macrophage phospholipase A2
US7582442B2 (en) * 2004-03-16 2009-09-01 The Regents Of The University Of Michigan Methods and compositions for using aleveolar macrophage phospholipase A2
WO2006014798A2 (en) * 2004-07-27 2006-02-09 Mount Sinai School Of Medicine Methods and compositions for using sax2
FI20050753A7 (fi) 2004-09-03 2006-03-04 Licentia Oy Uudet peptidit
US7531523B2 (en) * 2005-02-17 2009-05-12 Vertex Pharmaceuticals Incorporated Sodium channel protein type III alpha-subunit splice variant
MX2007013734A (es) * 2005-05-02 2008-03-14 Genzyme Corp Terapia genica para trastornos de la medula espinal.
CN107007842A (zh) 2005-05-02 2017-08-04 建新公司 神经代谢疾病的基因治疗
WO2007022287A2 (en) 2005-08-15 2007-02-22 Vegenics Limited Modified vegf and pdgf with improved angiogenic properties
US7972813B2 (en) * 2005-09-30 2011-07-05 Vertex Pharmaceuticals Incorporated Tetrodotoxin-resistant sodium channel alpha subunit
US20080200408A1 (en) * 2005-09-30 2008-08-21 Mccormack Kenneth Deletion mutants of tetrodotoxin-resistant sodium channel alpha subunit
US20090214496A1 (en) * 2006-01-30 2009-08-27 Licentia Ltd. Bmx/etk tyrosine kinase gene therapy materials and methods
AR059371A1 (es) 2006-02-08 2008-03-26 Genzyme Corp Terapia genica para la enfermedad de niemann-pick tipo a
WO2007098281A2 (en) 2006-02-27 2007-08-30 Regents Of The University Of California Oxysterol compounds and the hedgehog pathway
EP2548579B1 (en) 2006-05-17 2015-09-02 The Ludwig Institute for Cancer Research Targeting VEGF-B regulation of fatty acid transporters to modulate human diseases
ES2596885T3 (es) 2006-06-07 2017-01-12 Genzyme Corporation Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la medula espinal
WO2008011344A2 (en) * 2006-07-17 2008-01-24 Nationwide Children's Hospital Inc. Disruption of programmed death-1 (pd-1) ligands to adjuvant adeno-associated virus vector vaccines
SI2497500T1 (sl) 2006-10-03 2017-01-31 Genzyme Corporation Genska terapija za spinalno mišično atrofijo
ES2569365T3 (es) 2006-11-29 2016-05-10 Nationwide Children's Hospital Inhibición de miostatina para la potenciación de músculo y/o la mejora de la función muscular
AU2008331808B2 (en) 2007-03-16 2014-08-21 The Regents Of The University Of California Oxysterols for activation of hedgehog signaling, osteoinduction, antiadipogenesis, and Wnt signaling
HUE027278T2 (en) 2007-05-16 2016-11-28 Brigham & Womens Hospital Inc Treatment of synucleinopathies
PT3252161T (pt) 2007-06-06 2022-02-01 Genzyme Corp Terapia genética para doenças do armazenamento lisossomal
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
US8052970B2 (en) * 2008-06-30 2011-11-08 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
RS67077B1 (sr) 2009-05-02 2025-08-29 Genzyme Corp Genska terapija za neurodegenerativne poremećaje
EP2643045A4 (en) 2010-11-23 2016-01-13 Presage Biosciences Inc THERAPEUTIC PROCESSES AND COMPOSITIONS FOR SOLID FORM DELIVERY
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
EP2699673B1 (en) 2011-04-21 2018-09-19 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
US10196636B2 (en) 2011-04-21 2019-02-05 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
AU2012255143A1 (en) 2011-05-19 2014-02-20 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
ES2990023T3 (es) 2011-07-25 2024-11-28 Nationwide Childrens Hospital Inc Productos de virus recombinantes y métodos para inhibición de la expresión de DUX4
WO2013063383A2 (en) 2011-10-27 2013-05-02 Wellstat Ophthalmics Corporation Vectors encoding rod-derived cone viability factor
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
WO2013079687A1 (en) 2011-11-30 2013-06-06 The Chancellor, Masters And Scholars Of The University Of Oxford Inkt cell modulators and methods of using the same
GB201120860D0 (en) 2011-12-05 2012-01-18 Cambridge Entpr Ltd Cancer immunotherapy
EP3492586B1 (en) 2012-02-17 2024-04-17 Schepens Eye Research Institute Phenotype profile of human retinal progenitor cells
RU2632191C2 (ru) 2012-05-07 2017-10-03 Те Риджентс Оф Те Юниверсити Оф Калифорния Аналог оксистерола oxy133 индуцирует остеогенез и сигнальный путь hedgehog и ингибирует липогенез
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
EP3769789A1 (en) 2012-08-01 2021-01-27 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
PT3421602T (pt) 2012-09-06 2021-05-31 Univ Chicago Polinucleótidos antisenso para induzir o salto de exão e métodos de tratamentos de distrofias
EP2895606A4 (en) 2012-09-17 2016-07-06 Res Inst Nationwide Childrens Hospital COMPOSITIONS AND METHOD FOR THE TREATMENT OF AMYOTROPHER LATERAL SCLEROSIS (ALS)
US9745558B2 (en) 2013-02-18 2017-08-29 Vegenics Pty Limited VEGFR-3 ligand binding molecules and uses thereof
EP2983707B1 (en) 2013-04-08 2019-06-12 University of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
MX388221B (es) 2013-04-20 2025-03-19 Res Institute At Nationwide Children´S Hospital ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2.
CA2911205A1 (en) 2013-05-02 2014-11-06 The Regents Of The University Of California Bone-selective osteogenic oxysterol-bone targeting agents
RU2692251C2 (ru) 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
WO2014191630A2 (en) 2013-05-28 2014-12-04 Helsingin Yliopisto Non-human animal model encoding a non-functional manf gene
PL3702466T3 (pl) 2013-08-27 2023-04-24 Research Institute At Nationwide Children's Hospital Produkty i sposoby do leczenia stwardnienia zanikowego bocznego
US11078464B2 (en) * 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
JP2016538276A (ja) 2013-11-05 2016-12-08 ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルThe Research Institute At Nationwide Children’S Hospital 筋萎縮性側索硬化症の処置のためのNF−κBおよびSOD−1を阻害する組成物および方法
WO2015142984A1 (en) 2014-03-18 2015-09-24 Washington University Methods and compositions for red-shifted chromophore substitution for optogenetic applications
US10195280B2 (en) 2014-07-15 2019-02-05 Life Technologies Corporation Compositions and methods for efficient delivery of molecules to cells
WO2016025339A2 (en) 2014-08-09 2016-02-18 Research Institute At Nationwide Children's Hospital 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
WO2016073739A1 (en) 2014-11-05 2016-05-12 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
EP3250279B1 (en) 2015-01-30 2020-08-19 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
HUE052604T2 (hu) 2015-04-08 2021-05-28 Univ Chicago Készítmények és eljárások 2C típusú végtag-öv izomdisztrófia javítására exon-átugrás alkalmazásával
US20180104289A1 (en) 2015-04-08 2018-04-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Viral gene therapy as treatment for cholesterol storage disease or disorder
AU2016256895B2 (en) 2015-05-07 2022-05-26 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
WO2016187017A1 (en) 2015-05-15 2016-11-24 Mcivor R Scott Adeno-associated for therapeutic delivery to central nervous system
US10017832B2 (en) 2015-08-25 2018-07-10 Washington University Compositions and methods for site specific recombination at asymmetric sites
IL258005B2 (en) 2015-09-17 2025-01-01 Res Inst Nationwide Childrens Hospital Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2
JP2018532402A (ja) 2015-09-24 2018-11-08 クリスパー セラピューティクス アーゲー Rnaプログラム可能エンドヌクレアーゼの新規のファミリーならびにゲノム編集および他の適用におけるそれらの使用
ES3040945T3 (en) 2015-10-28 2025-11-06 Vertex Pharma Materials and methods for treatment of duchenne muscular dystrophy
CA2999649A1 (en) 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
KR102877920B1 (ko) 2015-11-16 2025-10-30 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
JP6932698B2 (ja) 2015-12-01 2021-09-08 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 アルファ1アンチトリプシン欠乏症の治療のための材料および方法
US20210260219A1 (en) 2015-12-23 2021-08-26 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
WO2017120589A1 (en) 2016-01-08 2017-07-13 Washington University Compositions comprising chemerin and methods of use thereof
US20190038771A1 (en) 2016-02-02 2019-02-07 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
EP3416689B1 (en) 2016-02-18 2023-01-18 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
WO2017147467A1 (en) 2016-02-26 2017-08-31 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inducing dux4 exon skipping
EP3426787A1 (en) 2016-03-07 2019-01-16 University of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
WO2017158422A1 (en) 2016-03-16 2017-09-21 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
JP6949867B2 (ja) 2016-03-28 2021-10-13 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California 神経の過剰興奮を治療するための方法および組成物
JP7253379B2 (ja) 2016-04-02 2023-04-06 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 組織特異的発現のための改変u6プロモーターシステム
HUE066442T2 (hu) 2016-04-15 2024-08-28 Res Institute At Nationwide ß-szarkoglikán és mikroRNS-29 adenoasszociáltvírus-vektorral történõ bejuttatása és izomdisztrófia kezelése
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
SG11201809144XA (en) 2016-04-18 2018-11-29 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
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
EP4484443A3 (en) 2016-06-29 2025-03-26 CRISPR Therapeutics AG Materials and methods for treatment of friedreich ataxia and other related disorders
JP7548696B2 (ja) 2016-07-06 2024-09-10 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
CA3029132A1 (en) 2016-07-06 2018-01-11 Crispr Therapeutics Ag 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
KR20190086503A (ko) 2016-11-15 2019-07-22 리젠츠 오브 더 유니버시티 오브 미네소타 Mpsi 및 mpsii에서의 신경계 기능 및 다른 신경계 장애를 개선하는 방법
CA3044291A1 (en) 2016-11-17 2018-05-24 Nationwide Children's Hospital Inc. Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
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
CA3053304A1 (en) 2017-02-17 2018-08-23 Lonza Ltd. Mammalian cells for producing adeno-associated viruses
US11920148B2 (en) 2017-02-22 2024-03-05 Crispr Therapeutics Ag Compositions and methods for gene editing
EP3585807A1 (en) 2017-02-22 2020-01-01 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
US20200216857A1 (en) 2017-02-22 2020-07-09 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
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
WO2018154459A1 (en) 2017-02-22 2018-08-30 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
JP7162021B2 (ja) 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
WO2018170408A1 (en) 2017-03-17 2018-09-20 Research Institute At Nationwide Children's Hospital, Inc. Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
MY201573A (en) 2017-05-12 2024-03-02 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
WO2018237066A1 (en) 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services CODON OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF C1-TYPE NIEMANN-PICK DISEASE AND ASSOCIATED STATES
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
EP3668997A4 (en) 2017-10-02 2021-05-19 Research Institute at Nationwide Children's Hospital SYSTEM FOR MIRNA DETARGETING FOR TISSUE-SPECIFIC INTERFERENCE
EP3697907A1 (en) 2017-10-17 2020-08-26 CRISPR Therapeutics AG Compositions and methods for gene editing for hemophilia a
US11534501B2 (en) 2017-10-18 2022-12-27 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
EP3697916A4 (en) 2017-10-20 2021-08-11 Research Institute at Nationwide Children's Hospital METHODS AND MATERIALS FOR NT-3 GENE THERAPY
US20210180091A1 (en) 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN120505370A (zh) 2017-11-08 2025-08-19 诺华股份有限公司 制备病毒载体的手段和方法及其用途
WO2019092507A2 (en) 2017-11-09 2019-05-16 Crispr Therapeutics Ag Crispr/cas systems for treatment of dmd
US20190153440A1 (en) 2017-11-21 2019-05-23 Casebia Therapeutics Llp Materials and methods for treatment of autosomal dominant retinitis pigmentosa
SG11202004488TA (en) 2017-11-27 2020-06-29 Coda Biotherapeutics Inc Compositions and methods for neurological diseases
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
CA3084632A1 (en) 2017-12-21 2019-06-27 Crispr Therapeutics Ag 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)
EP3737762A1 (en) 2018-01-12 2020-11-18 CRISPR Therapeutics AG Compositions and methods for gene editing by targeting transferrin
SG11202006722RA (en) 2018-01-31 2020-08-28 Res Inst Nationwide Childrens Hospital Gene therapy for limb-girdle muscular dystrophy type 2c
MA51787A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes de traitement d'hémoglobinopathies
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
US20210130824A1 (en) 2018-02-16 2021-05-06 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting fibrinogen-alpha
CA3092497A1 (en) 2018-03-19 2019-09-26 Crispr Therapeutics Ag Novel rna-programmable endonuclease systems and uses thereof
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
EP3773743A1 (en) 2018-04-03 2021-02-17 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
EP3801638A1 (en) 2018-06-08 2021-04-14 Novartis AG Cell-based assay for measuring drug product potency
US12258395B2 (en) 2018-06-11 2025-03-25 University Of Florida Research Foundation, Incorporated 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
EP3807309A1 (en) 2018-06-18 2021-04-21 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
US12492402B2 (en) 2018-08-22 2025-12-09 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3′ untranslated region of the DMPK gene
CA3110665A1 (en) 2018-08-29 2020-03-05 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of mutant gars protein
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
BR112021009739A2 (pt) 2018-11-30 2021-10-19 Novartis Ag Vetores virais aav e usos dos mesmos
WO2020127813A1 (en) 2018-12-21 2020-06-25 Genethon Expression cassettes for gene therapy vectors
JP2022516515A (ja) 2018-12-31 2022-02-28 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 Rna標的化crispr-cas13bを使用するdux4 rna発現停止
TW202045530A (zh) 2019-02-04 2020-12-16 美國全美兒童醫院之研究學會 腺相關病毒對cln3聚核苷酸的遞送
SG11202107983TA (en) 2019-02-04 2021-08-30 Res Inst Nationwide Childrens Hospital Adeno-associated virus delivery of cln6 polynucleotide
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc Gene editing for hemophilia A with improved Factor VIII expression
CA3131390A1 (en) 2019-02-26 2020-09-03 Research Institute Of Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
WO2020186059A2 (en) 2019-03-12 2020-09-17 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
WO2020214668A1 (en) 2019-04-15 2020-10-22 University Of Iowa Research Foundation Compositions and methods for treatment of cystic fibrosis
SG11202111353QA (en) 2019-04-15 2021-11-29 Univ Iowa Res Found Methods and compositions for transgene expression
EP3955969A1 (en) 2019-04-15 2022-02-23 Sanford Research Gene therapy for treating or preventing visual effects in batten disease
WO2020225606A1 (en) 2019-05-08 2020-11-12 Crispr Therapeutics Ag Crispr/cas all-in-two vector systems for treatment of dmd
KR20220009427A (ko) 2019-05-17 2022-01-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 글리코시드 가수분해효소를 사용하는 망막 세포에 대한 유전자 치료 벡터의 개선된 전달
WO2020264254A1 (en) 2019-06-28 2020-12-30 Crispr Therapeutics Ag Materials and methods for controlling gene editing
EP4004213A1 (en) 2019-07-25 2022-06-01 Novartis AG Regulatable expression systems
CA3151920A1 (en) 2019-08-21 2021-02-25 Coda Biotherapeutics, Inc. Compositions and methods for neurological diseases
HRP20240505T1 (hr) 2019-08-21 2024-07-05 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana vektorom povezanim s adenovirusom i liječenje mišićne distrofije
US20230416776A1 (en) 2019-10-08 2023-12-28 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療c型尼曼—匹克病之腺相關病毒載體
WO2021077115A1 (en) 2019-10-18 2021-04-22 Research Institute At Nationwide Children's Hospital Gene therapy targeting cochlear cells
US20220389453A1 (en) 2019-10-18 2022-12-08 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of disorders associated with the irf2bpl gene
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
BR112022010373A2 (pt) 2019-11-29 2022-08-16 Paros Bio Inc Terapia genética para doenças neurodegenerativas
WO2021127655A1 (en) 2019-12-20 2021-06-24 Research Institute At Nationwide Children's Hospital Optimized gene therapy for targeting muscle in muscle diseases
CN115485382A (zh) 2020-02-18 2022-12-16 全国儿童医院研究所 X连锁病症的治疗中的AAV介导的MiRNA靶向
WO2021209521A1 (en) 2020-04-14 2021-10-21 Genethon Vectors for the treatment of acid ceramidase deficiency
WO2021257595A1 (en) 2020-06-15 2021-12-23 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
WO2022011099A1 (en) 2020-07-08 2022-01-13 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
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
US20230414787A1 (en) 2020-08-27 2023-12-28 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
TWI887479B (zh) 2020-09-08 2025-06-21 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
AU2021345112A1 (en) 2020-09-15 2023-04-27 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
WO2022067257A1 (en) 2020-09-28 2022-03-31 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
US20220290136A1 (en) 2020-09-30 2022-09-15 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis
US20230374542A1 (en) 2020-10-07 2023-11-23 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 (lgmd21)
US20240026356A1 (en) 2020-11-30 2024-01-25 Research Institute At Nationwide Children's Hospital Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
EP4263829A1 (en) 2020-12-17 2023-10-25 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
KR20230138949A (ko) 2021-02-03 2023-10-05 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Dux4 과발현과 연관된 질환을 치료하기 위한 조성물 및 방법
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
EP4305157A1 (en) 2021-03-09 2024-01-17 Huidagene Therapeutics (Singapore) Pte. Ltd. Engineered crispr/cas13 system and uses thereof
WO2022221424A1 (en) 2021-04-13 2022-10-20 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
US20240115738A1 (en) 2021-04-15 2024-04-11 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis
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
WO2022226334A1 (en) 2021-04-23 2022-10-27 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
US20240425877A1 (en) 2021-05-07 2024-12-26 Ucl Business Ltd Abca4 genome editing
EP4341413A1 (en) 2021-05-17 2024-03-27 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
CN117980484A (zh) 2021-09-16 2024-05-03 诺华股份有限公司 新颖的转录因子
US20240401046A1 (en) 2021-10-07 2024-12-05 Research Institute At Nationwide Children' S Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
WO2023060233A1 (en) 2021-10-08 2023-04-13 Amicus Therapeutics, Inc. Biomarkers for lysosomal storage diseases
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
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
EP4198134A1 (en) 2021-12-16 2023-06-21 Genethon Gamma-sarcoglycan gene transfer increase using modified itr sequences
EP4198047A1 (en) 2021-12-16 2023-06-21 Genethon Fukutin related protein 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
EP4198048A1 (en) 2021-12-16 2023-06-21 Genethon Calpain-3 gene transfer increase using modified itr sequences
CA3242989A1 (en) 2021-12-21 2023-06-29 Res Inst Nationwide Childrens Hospital Materials and methods for the treatment of limb girdle muscular dystrophy
US20250084391A1 (en) 2021-12-23 2025-03-13 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
AU2023227443A1 (en) 2022-03-01 2024-10-10 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions
EP4486900A2 (en) 2022-03-03 2025-01-08 Research Institute at Nationwide Children's Hospital Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
EP4503923A1 (en) 2022-04-04 2025-02-12 The Regents of the University of California Genetic complementation compositions and methods
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2023240177A1 (en) 2022-06-08 2023-12-14 Research Instiitute At Nationwide Children's Hospital Products and methods for treating diseases or conditions associated with mutant or pathogenic kcnq3 expression
EP4536818A1 (en) 2022-06-10 2025-04-16 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2024011115A1 (en) 2022-07-06 2024-01-11 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
EP4590838A1 (en) 2022-09-23 2025-07-30 Sarepta Therapeutics, Inc. Recombinant aav vectors for treating muscular dystrophy
EP4602162A1 (en) 2022-10-11 2025-08-20 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)
AU2023364350A1 (en) 2022-10-19 2025-06-05 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
WO2024092126A1 (en) 2022-10-27 2024-05-02 Cargo Therapeutics, Inc. Compositions and methods for improved immunotherapies
AU2023398007A1 (en) 2022-12-13 2025-06-19 Bayer Aktiengesellschaft Engineered type v rna 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
EP4658796A1 (en) 2023-02-01 2025-12-10 Sarepta Therapeutics, Inc. Raav production methods
WO2024168276A2 (en) 2023-02-09 2024-08-15 Cargo Therapeutics, Inc. Compositions and methods for immunotherapies
WO2024206928A1 (en) 2023-03-30 2024-10-03 Pharma Cinq, Llc VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IgK SIGNAL SEQUENCE
AU2024256934A1 (en) 2023-04-18 2025-11-06 Research Institute At Nationwide Children's Hospital, Inc. Gene therapy for treating limb girdle muscular dystrophy r9 and congenital muscular dystrophy 1c
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
WO2024229211A2 (en) 2023-05-02 2024-11-07 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
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
WO2024254319A1 (en) 2023-06-07 2024-12-12 Research Institute At Nationwide Children's Hospital Gene therapy for lysosomal acid lipase deficiency (lal-d)
WO2024259064A1 (en) 2023-06-13 2024-12-19 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom
US20250027087A1 (en) 2023-07-21 2025-01-23 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
WO2025076291A1 (en) 2023-10-06 2025-04-10 Bluerock Therapeutics Lp Engineered type v rna programmable endonucleases and their uses
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
WO2025113643A1 (en) 2023-12-01 2025-06-05 Gilead Sciences Inc. Anti-fap-light fusion protein and use thereof
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
WO2025194042A1 (en) 2024-03-14 2025-09-18 Affinia Therapeutics Inc. Plasmid system for production of recombinant adenoassociated virus
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
WO2025227063A1 (en) 2024-04-26 2025-10-30 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
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
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
US20250369016A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle tropic raav

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
WO1992010574A1 (en) * 1990-12-06 1992-06-25 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce Inhibition of human immunodeficiency virus by an adeno-associated virus gene for human cells
ATE272123T1 (de) * 1993-11-09 2004-08-15 Ohio Med College Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren

Also Published As

Publication number Publication date
DK0733103T3 (da) 2004-07-12
DE69433592T2 (de) 2005-02-10
EP0733103A4 (en) 1997-12-17
JPH09509564A (ja) 1997-09-30
CA2176117A1 (en) 1995-05-18
WO1995013365A1 (en) 1995-05-18
DE69433592D1 (de) 2004-04-08
EP0733103A1 (en) 1996-09-25
ATE260980T1 (de) 2004-03-15
ES2216005T3 (es) 2004-10-16
JP2005110694A (ja) 2005-04-28
AU688428B2 (en) 1998-03-12
CA2176117C (en) 2006-01-03
EP0733103B1 (en) 2004-03-03
US5658776A (en) 1997-08-19
AU1129395A (en) 1995-05-29

Similar Documents

Publication Publication Date Title
PT733103E (pt) Criacao de elevados titulos de vectores de aav recombinantes
Fischer et al. Leukocyte adhesion deficiency: molecular basis and functional consequences.
DE69232725D1 (de) Synthetische dna-sequenz mit verstärkter isektizider aktivität in mais
DE3484664D1 (de) Rekombinante immunoglobulin-praeparate, verfahren zu ihrer herstellung, dna-sequenzen, expressionsvektoren und rekombinante wirkzellen hierfuer.
AU4427389A (en) Peptides and antibodies that inhibit integrin-ligand binding
PT728214E (pt) Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
CA2435972A1 (en) Matrix attachment regions and methods for use thereof
AR018101A1 (es) UNA REGION VARIABLE DE UNA CADENA LIGERA Y DE UNA CADENA PESADA, MOLECULAS DE ACIDO NUCLEICO QUE LAS CODIFICAN, VECTORES DE EXPRESIoN, ANTICUERPOS QUIMERICOS QUE SE ENLAZAN A CD40 HUMANO, ANTICUERPOS HUMANIZADOS Y COMPOSICIONES FARMACEUTICAS QUE COMPRENDEN DICHAS CADENAS.
BRPI9811940A (pt) vetor de expressão para um polipeptídeo de ligação da interleucina-18 (il-18-bp), célula hospedeira procariótica transformada com o referido vetor, processo para produção de il-18-bp, proteína de fusão compreendendo o referido polipeptídeo, bem como il-18bp modificada.
DE59410380D1 (de) Endosomolytisch wirksame partikel
DE69933550D1 (de) Modifizierte adenoviren welche ein faserersatzprotein enthalten
BR9916069A (pt) Sistema de expressão para fator viii
ES2191011T3 (es) Acido nucleico, vector de expresion y composiciones para la identificacion y sintesis de sialiltransferasas recombinantes.
GB8818706D0 (en) Dna constructs & plants incorporating them
ATE321873T1 (de) Rekombinante aav-vektoren für die gentherapie von hämophilie a
BR9904475A (pt) Molécula tipo anticorpo anti-fas, humanizada, agente para a profilaxia e/ou tratamento de condições envolvendo uma anormalidade no sistema ligando fas/fas, uso de um anticorpo, dna, vetor de dna recombinante, célula hospedeira, processo para produzir um anticorpo anti-pas, cepa transformante de e.coli, e, polipeptìdeo.
Tamiya et al. Transgene inheritance and retroviral infection contribute to the efficiency of gene expression in solid tumors inoculated with retroviral vector producer cells.
SG54291A1 (en) Monoclonal antibodies to antigens expressed by hematopoietic facilitatory cells
ES2170751T3 (es) Promotor aox2 mutante, vector portador del mismo, transformante, y produccion de proteina heterologa.
Cornelissen et al. A retrospective Dutch study comparing T cell-depleted allogeneic blood stem cell transplantation vs T cell-depleted allogeneic bone marrow transplantation.
DE69535024D1 (de) Verpackungszellinie zur transkomplementierung von defektiven retroviren-vektoren
Wahren et al. NCA: a differentiation antigen of myelopoietic cells in humans and hominoid monkeys.
Harada et al. Aplastic anemia treated by isogeneic or allogeneic bone marrow transplantation (author's transl)
FI864071A0 (fi) Promoottorijärjestelmä streptomykeettavektoreita varten
WO1992001058A1 (en) Hybridomas