DE69535749D1 - Vektoren für rekombinante aav-genome - Google Patents

Vektoren für rekombinante aav-genome

Info

Publication number
DE69535749D1
DE69535749D1 DE69535749T DE69535749T DE69535749D1 DE 69535749 D1 DE69535749 D1 DE 69535749D1 DE 69535749 T DE69535749 T DE 69535749T DE 69535749 T DE69535749 T DE 69535749T DE 69535749 D1 DE69535749 D1 DE 69535749D1
Authority
DE
Germany
Prior art keywords
raav
methods
genomes
useful
adeno
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.)
Expired - Lifetime
Application number
DE69535749T
Other languages
English (en)
Inventor
Philip R Johnson
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nationwide Childrens Hospital Inc
Original Assignee
Nationwide Childrens Hospital Inc
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 Nationwide Childrens Hospital Inc filed Critical Nationwide Childrens Hospital Inc
Application granted granted Critical
Publication of DE69535749D1 publication Critical patent/DE69535749D1/de
Anticipated expiration legal-status Critical
Expired - Lifetime 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
DE69535749T 1994-06-06 1995-06-06 Vektoren für rekombinante aav-genome Expired - Lifetime DE69535749D1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US08/254,358 US5658785A (en) 1994-06-06 1994-06-06 Adeno-associated virus materials and methods
PCT/US1995/007178 WO1995034670A2 (en) 1994-06-06 1995-06-06 Recombinant aav genome encoding immunodeficiency virus protein

Publications (1)

Publication Number Publication Date
DE69535749D1 true DE69535749D1 (de) 2008-06-19

Family

ID=22963991

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69535749T Expired - Lifetime DE69535749D1 (de) 1994-06-06 1995-06-06 Vektoren für rekombinante aav-genome

Country Status (11)

Country Link
US (3) US5658785A (de)
EP (2) EP0764213B1 (de)
JP (3) JP3720845B2 (de)
AT (1) ATE394498T1 (de)
AU (1) AU710804B2 (de)
CA (2) CA2576396A1 (de)
DE (1) DE69535749D1 (de)
DK (1) DK0764213T3 (de)
ES (1) ES2302331T3 (de)
PT (1) PT764213E (de)
WO (1) WO1995034670A2 (de)

Families Citing this family (336)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
US6924128B2 (en) * 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US7745416B2 (en) * 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
IL122206A0 (en) * 1995-06-07 1998-04-05 Univ North Carolina Adeno-associated virus vectors for gene expression
US20030206891A1 (en) * 1995-06-07 2003-11-06 Ariad Gene Therapeutics, Inc. Rapamycin-based biological regulation
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5837681A (en) * 1996-02-23 1998-11-17 Amgen Inc. Method for treating sensorineural hearing loss using glial cell line-derived neurotrophic factor (GDNF) protein product
US5929041A (en) * 1996-02-23 1999-07-27 Amgen Inc. Method for preventing and treating sensorineural hearing loss and vestibular disorders using glial cell line-derived neurotrophic factor(GDNF) protein product
US6455277B1 (en) 1996-04-22 2002-09-24 Amgen Inc. Polynucleotides encoding human glial cell line-derived neurotrophic factor receptor polypeptides
US7138251B1 (en) 1996-04-22 2006-11-21 Amgen Inc. Polynucleotides encoding a neurotrophic factor receptor
US6294370B1 (en) 1997-06-24 2001-09-25 Medigene Ag System for the production of AAV vectors
US6541012B2 (en) 1996-06-24 2003-04-01 Christoph Bogedain System for the production of AAV vectors
DE19625188A1 (de) * 1996-06-24 1998-01-08 Medigene Gmbh System zur Herstellung von AAV-Vektoren
IL128779A0 (en) * 1996-09-06 2000-01-31 Univ Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
WO1998009524A1 (en) * 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
GB9622500D0 (en) * 1996-10-29 1997-01-08 Oxford Biomedica Ltd Therapeutic gene
GB2333527B (en) * 1996-10-29 2001-05-02 Oxford Biomedica Ltd Therapeutic gene
US6924123B2 (en) 1996-10-29 2005-08-02 Oxford Biomedica (Uk) Limited Lentiviral LTR-deleted vector
WO1998046737A2 (en) * 1997-04-15 1998-10-22 University Of Medicine And Dentistry Of New Jersey cDNA FOR HUMAN GDNF AND PROMOTER THEREFOR WHICH ALLOWS REGULATED AND CONSTITUTIVE EXPRESSION
NZ333334A (en) * 1997-04-17 2001-06-29 Frank L Sorgi Delivery system for gene therapy to the brain
US6242426B1 (en) * 1997-07-25 2001-06-05 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6043221A (en) 1997-07-30 2000-03-28 Amgen Inc. Method for preventing and treating hearing loss using a neuturin protein product
DE69838584T2 (de) 1997-08-04 2008-06-26 Cell Genesys, Inc., Foster City Enhancer des menschlichen glandulären kallikreingens, vektoren die ihn enthalten und methoden für seine verwendung
US20030125278A1 (en) * 1997-08-13 2003-07-03 Tang De-Chu C. Immunization of animals by topical applications of a salmonella-based vector
US6706693B1 (en) 1997-08-13 2004-03-16 The Uab Research Foundation Vaccination by topical application of genetic vectors
US20030045492A1 (en) * 1997-08-13 2003-03-06 Tang De-Chu C. Vaccination by topical application of recombinant vectors
US6348450B1 (en) 1997-08-13 2002-02-19 The Uab Research Foundation Noninvasive genetic immunization, expression products therefrom and uses thereof
US6716823B1 (en) 1997-08-13 2004-04-06 The Uab Research Foundation Noninvasive genetic immunization, expression products therefrom, and uses thereof
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
EP1015619A1 (de) * 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methoden und zelllinien für die produktion von rekombinanten adeno-assoziierten viren
WO1999018227A1 (en) * 1997-10-08 1999-04-15 Advanced Research And Technology Institute Chimeric parvovirus-based recombinant vector system that specifically targets the erythroid lineage
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant 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
EP1071806B1 (de) * 1998-04-24 2004-06-16 University of Florida Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie
EP1080202B1 (de) 1998-05-27 2006-01-25 Avigen, Inc. Konvektion-erhöhte verabreichung aadc-kodierende aav vektoren
WO2000022152A1 (en) * 1998-10-13 2000-04-20 Avigen, Inc. Compositions and methods for producing recombinant adeno-associated virus
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
US6759237B1 (en) * 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
EP1135468B1 (de) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6790657B1 (en) * 1999-01-07 2004-09-14 The United States Of America As Represented By The Department Of Health And Human Services Lentivirus vector system
EP1146898A1 (de) * 1999-01-22 2001-10-24 Matthew John During Impfstoff-vermittelte behandlung neurologischer krankheiten
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US20040043488A1 (en) * 1999-02-17 2004-03-04 University Of Pittsburgh Of The Commonwealth System Of Higher Education Adeno-associated viral gene-transfer vector system
WO2000054595A1 (en) * 1999-03-15 2000-09-21 John Guy Recombinant adeno-associated virus transfer of genes affecting demyelination
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
WO2000058479A1 (en) 1999-03-26 2000-10-05 Amgen Inc. Beta secretase genes and polypeptides
US6893865B1 (en) * 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
US20040009936A1 (en) * 1999-05-03 2004-01-15 Tang De-Chu C. Vaccine and drug delivery by topical application of vectors and vector extracts
US6793926B1 (en) * 1999-05-27 2004-09-21 Genovo, Inc. Methods for production of a recombinant adeno-associated virus
EP1939300A1 (de) 1999-05-28 2008-07-02 Targeted Genetics Corporation Verfahren und Zusammensetzung zur Senkung des Spiegels des Tumornekrosefaktors (TNF) bei TNF-assoziierten Leiden
EP1180159B1 (de) 1999-05-28 2008-09-03 Targeted Genetics Corporation Methoden und zusammensetzungen zur reduktion des tumor-nekrosis-faktor-spiegels (tnf) bei tnf-assoziierten erkrankungen
EP1916258B1 (de) 1999-08-09 2014-04-23 Targeted Genetics Corporation Erweiterte Expression einer einstämmigen, heterologen Nukleotidsequenz von rekombinanten viralen Vektoren durch Modellierung der Sequenz mit stammübergreifenden Basispaaren
EP2369002A1 (de) * 1999-08-09 2011-09-28 Targeted Genetics Corporation Erweiterte Expression einer einstämmigen, heterologen Nukleotidsequenz von rekombinanten viralen Vektoren durch Modellierung der Sequenz mit stammübergreifenden Basispaaren
CA2381351C (en) * 1999-08-31 2009-11-17 Asahi Glass Company, Limited Process for producing a vic-dichloro acid fluoride
US7459540B1 (en) 1999-09-07 2008-12-02 Amgen Inc. Fibroblast growth factor-like polypeptides
US7408047B1 (en) 1999-09-07 2008-08-05 Amgen Inc. Fibroblast growth factor-like polypeptides
US7115391B1 (en) * 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
WO2001025253A2 (en) 1999-10-01 2001-04-12 The University Of North Carolina At Chapel Hill Temperature-sensitive regulation of viral vector production
CA2387484A1 (en) * 1999-10-12 2001-04-19 Haim Burstein Adeno-associated virus vectors encoding factor viii and methods of using the same
FR2804028B1 (fr) * 2000-01-21 2004-06-04 Merial Sas Vaccins adn ameliores pour animaux de rente
CA2397532A1 (en) * 2000-01-26 2001-08-02 Chiron Corporation Recombinant aav packaging systems
US6821775B1 (en) * 2000-02-11 2004-11-23 Genvec, Inc. Viral vector encoding pigment epithelium-derived factor
WO2002072754A2 (en) * 2001-03-08 2002-09-19 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Mva expressing modified hiv envelope, gag, and pol genes
US8623379B2 (en) 2000-03-02 2014-01-07 Emory University Compositions and methods for generating an immune response
US20040105871A1 (en) * 2000-03-02 2004-06-03 Robinson Harriet L. Compositions and methods for generating an immune response
CA2401974C (en) * 2000-03-02 2013-07-02 Emory University Dna expression vectors and methods of use
US7514239B2 (en) 2000-03-28 2009-04-07 Amgen Inc. Nucleic acid molecules encoding beta-like glycoprotein hormone polypeptides and heterodimers thereof
ES2327609T3 (es) * 2000-06-01 2009-11-02 University Of North Carolina At Chapel Hill Procedimientos y compuestos para controlar la liberacion de vectores de parvovirus reconbinantes.
ATE419351T1 (de) 2000-06-28 2009-01-15 Amgen Inc Thymus-stroma lymphopoietin rezeptor-moleküle und deren verwendung
JP2005500835A (ja) * 2001-06-29 2005-01-13 ノバルティス アクチエンゲゼルシャフト Ｐｅｒｖスクリーニング法およびその使用
EP1279740A1 (de) * 2001-07-26 2003-01-29 Vrije Universiteit Brussel Von Adeno-assoziertem Virus abstammender Vektor zur Gentherapy
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
US20030129164A1 (en) * 2001-12-03 2003-07-10 Flannery John G. Expression of glial-derived neurotrophic factor for treatment of diseases of the eye
EP1453547B1 (de) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-assoziierte virus (aav) serotyp 8 sequenzen, diese enthaltende vektoren und ihre verwendung
CA2469053C (en) 2001-12-17 2011-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
JP2005516949A (ja) * 2001-12-19 2005-06-09 リジュン・ワン Ｇｄｎｆの骨格筋へのアデノ随伴ウイルス媒介性のデリバリー
US7662924B2 (en) 2002-02-22 2010-02-16 The Board Of Trustees Of The University Of Illinois Beta chain-associated regulator of apoptosis
EP1575481A4 (de) * 2002-03-01 2010-01-06 Celltech R & D Inc Verfahren zur erhöhung oder verringerung der knochendichte
US20030219733A1 (en) * 2002-04-09 2003-11-27 Clark Kelly Reed Antibody gene transfer and recombinant AAV therefor
JP2006506072A (ja) 2002-11-12 2006-02-23 デイッセロース，アルバート，ビー アデノウイルスベクターワクチン
EP1587923B1 (de) 2003-01-22 2011-08-24 Duke University Verbesserte konstrukte zur expression lysosomaler polypeptide
US7510872B2 (en) * 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
US20040248826A1 (en) * 2003-06-03 2004-12-09 Alberto Auricchio Treatment of cancer by in vivo gene-transfer induced TIMP-3 expression
US20050019927A1 (en) * 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
JP2007535304A (ja) 2003-11-24 2007-12-06 シドニー キンメル キャンサー センター ムチン抗原ワクチン
US8828957B2 (en) * 2003-12-11 2014-09-09 Microvax, Llc Methods for generating immunity to antigen
CA2555675A1 (en) 2004-02-10 2005-08-25 Trustees Of Dartmouth College Nicotinamide riboside kinase compositions and methods for using the same
DE602005026811D1 (de) 2004-06-22 2011-04-21 Univ Illinois Verfahren zur inhibierung von tumorzellwachstum mit foxm1 sirns
US7517870B2 (en) 2004-12-03 2009-04-14 Fondazione Telethon Use of compounds that interfere with the hedgehog signaling pathway for the manufacture of a medicament for preventing, inhibiting, and/or reversing ocular diseases related with ocular neovascularization
CN101124328A (zh) * 2004-12-15 2008-02-13 北卡罗来纳查佩尔山大学 嵌合载体
US20070041943A1 (en) * 2004-12-29 2007-02-22 Children's Hospital Research Expression of virus entry inhibitors and recombinant AAV thereof
AU2006336468B2 (en) 2005-02-11 2012-04-12 University Of Southern California Method of expressing proteins with disulfide bridges
AU2006216671A1 (en) * 2005-02-23 2006-08-31 The Uab Research Foundation Alkyl-glycoside enhanced vaccination
US7629315B2 (en) 2005-03-09 2009-12-08 University Of Pittsburgh Of The Commonwealth System Of Higher Education Compositions for blocking the inhibitory effect of human CRP on human leptin
EP2567976B1 (de) 2005-03-23 2017-07-19 Genmab A/S Antikörper gegen CD38 zur Behandling von multiplem Myelom
CN104293835B (zh) 2005-04-07 2017-07-04 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
WO2006130525A2 (en) * 2005-05-31 2006-12-07 Sidney Kimmel Cancer Center Methods for immunotherapy of cancer
SG163572A1 (en) 2005-07-11 2010-08-30 Globeimmune Inc Compositions and methods for eliciting an immune response to escape mutants of targeted therapies
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
EP1951862B1 (de) * 2005-11-07 2013-07-24 MicroVAX, LLC Cd40-ligand-fusionsproteinimpfstoff
EP2018421B1 (de) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Skalierbares herstellungsverfahren für aav
US8008256B2 (en) * 2006-05-01 2011-08-30 University Of Southern California Combination therapy for treatment of cancer
CA2652858A1 (en) * 2006-06-15 2007-12-27 Targeted Genetics Corporation Methods for treating target joints in inflammatory arthritis using aav vectors encoding a tnf antagonist
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
EP2044199B1 (de) * 2006-07-25 2012-11-14 Celladon Corporation Erweiterte antegrade epikardiale koronare infusion adeno-assoziierter viraler vektoren welcher serca2a beinhaltet für gentherapie
WO2008067480A2 (en) 2006-11-29 2008-06-05 Nationwide Children's Hospital Myostatin inhibition for enhancing muscle and/or improving muscle function
CA2678508A1 (en) * 2007-02-17 2008-08-21 Bradley University Universal learning system
WO2008115610A1 (en) * 2007-03-19 2008-09-25 Globeimmune, Inc. Compositions and methods for targeted ablation of mutational escape of targeted therapies for cancer
EP2612868B1 (de) 2007-11-01 2018-08-15 Astellas Pharma Inc. Immunsuppressive Polypeptide und Nucleinsäuren
KR20100129295A (ko) * 2008-02-19 2010-12-08 셀라돈 코포레이션 심근 내 바이러스 벡터의 흡수를 개선시키기 위한 조성물
US7943379B2 (en) * 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
JOP20190083A1 (ar) 2008-06-04 2017-06-16 Amgen Inc بولي ببتيدات اندماجية طافرة لـfgf21 واستخداماتها
UA105016C2 (uk) 2008-10-10 2014-04-10 Амген Інк. Fgf21 мутанти і їх застосування
WO2010056901A2 (en) 2008-11-13 2010-05-20 University Of Southern California Method of expressing proteins with disulfide bridges with enhanced yields and activity
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
ES2634118T3 (es) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
EP2412387B1 (de) 2009-03-27 2014-11-19 Proyecto de Biomedicina Cima, S.L. Verfahren und zusammensetzungen zur behandlung von zirrhose und leberfibrose
CN102439157B (zh) 2009-04-30 2015-09-16 宾夕法尼亚大学托管会 包含腺伴随病毒构建体的靶向传导气道细胞组合物
LT3421603T (lt) 2009-05-02 2022-01-10 Genzyme Corporation Genų terapija, skirta neurodegeneraciniams sutrikimams
BRPI1011404B1 (pt) 2009-05-05 2022-05-03 Amgen Inc Polipeptídeos mutantes fgf21, polipeptídeo de fusão, multímero, composição farmacêutica, ácido nucleico isolado, vetor e célula hospedeira
AU2010246038A1 (en) 2009-05-05 2011-12-01 Amgen Inc. FGF21 mutants and uses thereof
JP5955771B2 (ja) 2009-10-22 2016-07-20 トーマス・ジェファーソン・ユニバーシティＴｈｏｍａｓ Ｊｅｆｆｅｒｓｏｎ Ｕｎｉｖｅｒｓｉｔｙ 細胞ベースの抗癌組成物ならびに当該物の製造方法および使用方法
CN102712933A (zh) 2009-11-05 2012-10-03 西马生物医学计划公司 调控的表达系统
UA109888C2 (uk) 2009-12-07 2015-10-26 ІЗОЛЬОВАНЕ АНТИТІЛО АБО ЙОГО ФРАГМЕНТ, ЩО ЗВ'ЯЗУЄТЬСЯ З β-КЛОТО, РЕЦЕПТОРАМИ FGF І ЇХНІМИ КОМПЛЕКСАМИ
US9517264B2 (en) 2010-04-15 2016-12-13 Amgen Inc. Human FGF receptor and β-Klotho binding proteins
AU2011255203B2 (en) 2010-05-21 2016-01-21 Peptimed, Inc. Reagents and methods for treating cancer
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
CN103476399A (zh) 2011-04-18 2013-12-25 独立行政法人国立精神·神经医疗研究中心 药剂递送粒子及其制造方法
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
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
US10196636B2 (en) 2011-04-21 2019-02-05 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
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
WO2013016352A1 (en) 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
EP2797613B1 (de) 2011-10-27 2019-12-04 Wellstat Ophthalmics Corporation Für rod-derived cone viability-faktor codierende vektoren
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
CA2880653C (en) 2012-08-01 2022-05-17 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
JP2015529685A (ja) 2012-09-17 2015-10-08 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 Ａｔｎａｔｉｏｎｗｉｄｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ 筋萎縮性側索硬化症の処置のための組成物および方法
EP3738974A1 (de) 2012-09-28 2020-11-18 The University of North Carolina at Chapel Hill Gegen oligodendrozyten gerichtete aav-vektoren
US11274305B2 (en) 2013-04-04 2022-03-15 Trustees Of Dartmouth College Compositions and methods for in vivo excision of HIV-1 proviral DNA
AU2014253730B2 (en) 2013-04-20 2018-09-13 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted U7snRNA polynucleotide constructs
EP4219727A3 (de) 2013-08-27 2023-09-06 Research Institute at Nationwide Children's Hospital Produkte und verfahren zur behandlung von amyotropher lateralsklerose
EP3065784A4 (de) 2013-11-05 2017-05-10 The Research Institute at Nationwide Children's Hospital Zusammensetzungen und verfahren zur hemmung von nf-kb und sod-1 zur behandlung von amyotropher lateralsklerose
WO2015077717A1 (en) 2013-11-25 2015-05-28 The Broad Institute Inc. Compositions and methods for diagnosing, evaluating and treating cancer by means of the dna methylation status
WO2015085147A1 (en) 2013-12-05 2015-06-11 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
NZ721908A (en) 2013-12-20 2022-12-23 Massachusetts Gen Hospital Combination therapy with neoantigen vaccine
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3572516A1 (de) 2014-08-09 2019-11-27 The Research Institute at Nationwide Children's Hospital Verfahren und materialien zur aktivierung einer internen ribosomeintrittsstelle in exon 5 des dmd-gens
WO2016057975A2 (en) 2014-10-10 2016-04-14 Research Institute At Nationwide Children's Hospital Guided injections for aav gene transfer to muscle
WO2016073693A2 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
CA2966884A1 (en) 2014-11-05 2016-05-12 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
JP6863891B2 (ja) 2014-11-14 2021-04-21 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 調節性ポリヌクレオチド
EP4344741A2 (de) 2014-11-21 2024-04-03 The University of North Carolina at Chapel Hill Auf das zentrale nervensystem gerichtete aav-vektoren
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2016100975A1 (en) 2014-12-19 2016-06-23 Massachsetts Institute Ot Technology Molecular biomarkers for cancer immunotherapy
US10993997B2 (en) 2014-12-19 2021-05-04 The Broad Institute, Inc. Methods for profiling the t cell repertoire
MA41451A (fr) 2015-02-04 2017-12-12 Univ Washington Constructions anti-tau
KR102554850B1 (ko) 2015-02-06 2023-07-13 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
CN107532168A (zh) 2015-02-23 2018-01-02 克里斯珀医疗股份公司 治疗血红蛋白病的材料和方法
WO2016145150A2 (en) 2015-03-11 2016-09-15 The Broad Institute Inc. Selective treatment of prmt5 dependent cancer
PL3291843T3 (pl) * 2015-05-07 2023-07-17 University Of South Florida Zmodyfikowany gen ube3a w terapii genowej zespołu angelmana
IL294183B2 (en) 2015-05-20 2023-10-01 Dana Farber Cancer Inst Inc shared neoantigens
TW202241500A (zh) 2015-06-09 2022-11-01 美商博德研究所有限公司 用於贅瘤疫苗之調配物及其製備方法
US10017832B2 (en) 2015-08-25 2018-07-10 Washington University Compositions and methods for site specific recombination at asymmetric sites
JP7338970B2 (ja) 2015-09-17 2023-09-05 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 Ｇａｌｇｔ２遺伝子治療のための方法および物質
WO2017064546A1 (en) 2015-09-24 2017-04-20 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
EP3368063B1 (de) 2015-10-28 2023-09-06 Vertex Pharmaceuticals Inc. Materialien und verfahren zur behandlung von duchenne-muskeldystrophie
CA2999649A1 (en) 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
IL259441B2 (en) 2015-11-16 2024-01-01 Res Inst Nationwide Childrens Hospital Materials and methods for the treatment of titin-based myopathies and other titinopathy
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
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
JP6947729B2 (ja) 2015-12-23 2021-10-13 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 筋萎縮性側索硬化症及び／または前頭側頭葉変性症の治療のための材料及び方法
WO2017120589A1 (en) 2016-01-08 2017-07-13 Washington University Compositions comprising chemerin and methods of use thereof
US20170198263A1 (en) 2016-01-13 2017-07-13 Merial Inc. Recombinant AAV Vectors Expressing Osteoprotective genes, including HAS2 and Lubricin, useful in the Treatment of Osteoarthritis and Related Joint Conditions in Mammals
WO2017134529A1 (en) 2016-02-02 2017-08-10 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
EP3416689B1 (de) 2016-02-18 2023-01-18 CRISPR Therapeutics AG Materialien und verfahren zur behandlung von schwerer kombinierter immundefizienz (scid) oder omenn syndrome
WO2017147509A1 (en) 2016-02-25 2017-08-31 Marco Colonna Compositions comprising trem2 and methods of use thereof
EP3420109A4 (de) 2016-02-26 2019-11-06 Research Institute at Nationwide Children's Hospital Rekombinante virusprodukte und verfahren zur induktion von dux4-exon-skipping
EP3429632B1 (de) 2016-03-16 2023-01-04 CRISPR Therapeutics AG Materialien und verfahren zur behandlung von erblicher haemochromatose
WO2017172772A1 (en) 2016-03-28 2017-10-05 Dimension Therapeutics Methods of heat inactivation of adenovirus
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
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
IL262262B2 (en) 2016-04-15 2023-10-01 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of B-sarcoglycan and microRNA-29 and treatment of muscular dystrophy
WO2017184463A1 (en) 2016-04-17 2017-10-26 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
MX2018012729A (es) 2016-04-18 2019-07-04 Crispr Therapeutics Ag Materiales y metodos para el tratamiento de hemoglobinopatias.
US20190346442A1 (en) 2016-04-18 2019-11-14 The Broad Institute, Inc. Improved hla epitope prediction
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3458589A4 (de) 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von morbus huntington
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc modulatory polynucleotides
EP3478829A1 (de) 2016-06-29 2019-05-08 Crispr Therapeutics AG Materialien und verfahren zur behandlung von myotoner dystrophie typ 1 (dm1) und anderen erkrankungen
EP3478313B1 (de) 2016-06-29 2022-05-04 CRISPR Therapeutics AG Materialien und verfahren zur behandlung von amyotropher lateralsklerose (als) und anderen verwandten erkrankungen
AU2017290614C1 (en) 2016-06-29 2024-01-18 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
US11801313B2 (en) 2016-07-06 2023-10-31 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
CN109843914B (zh) 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
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
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
EP3526321A4 (de) 2016-10-14 2020-05-13 Ultragenyx Pharmaceutical Inc. Verwendung von tonifizierenden mitteln zur verbesserung der ausbeute von rekombinantem adeno-assoziiertem virus
AU2017362491B2 (en) 2016-11-17 2023-02-02 Nationwide Children's Hospital Inc. Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2
EP3555296A4 (de) 2016-12-13 2020-07-29 Seattle Children's Hospital (DBA Seattle Children's Research Institute) Verfahren zur exogenen wirkstoffaktivierung von chemisch induzierten signalisierungskomplexen, die in vitro und in vivo in manipulierten zellen exprimiert werden
US11549149B2 (en) 2017-01-24 2023-01-10 The Broad Institute, Inc. Compositions and methods for detecting a mutant variant of a polynucleotide
ES2965475T3 (es) 2017-02-12 2024-04-15 Biontech Us Inc Métodos y composiciones basados en HLA y usos de los mismos
EP3585898A1 (de) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materialien und verfahren zur behandlung von spinozerebellärer ataxie typ 1 (sca1) und anderen mit dem gen des proteins von spinozerebellärer ataxie typ 1 (atxn1) assoziierten erkrankungen oder störungen
WO2018154418A1 (en) 2017-02-22 2018-08-30 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
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
CA3054031A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Compositions and methods for gene editing
BR112019019248A2 (pt) 2017-03-17 2020-04-28 Research Institute At Nationwide Children's Hospital entrega de vetor de vírus adeno-associado de microdistrofina específica muscular para tratamento de distrofia muscular
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
US11898170B2 (en) 2017-03-22 2024-02-13 Ultragenyx Pharmaceutical Inc. Cell culture methods involving HDAC inhibitors or rep proteins
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
AU2018367896B2 (en) 2017-05-12 2023-06-01 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019011817A1 (en) 2017-07-08 2019-01-17 Genethon TREATMENT OF SPINAL AMYOTROPHY
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CA3077426A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
MA50833A (fr) 2017-10-17 2020-08-26 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique pour l'hémophilie 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
US11926653B2 (en) 2017-10-20 2024-03-12 Research Institute At Nationwide Children's Hospital Methods and materials for NT-3 gene therapy
MA50849A (fr) 2017-10-26 2020-09-02 Vertex Pharma Substances et procédés pour le traitement d'hémoglobinopathies
CA3082136A1 (en) 2017-11-08 2019-05-16 Avexis, Inc. Means and method for preparing viral vectors and uses of same
US20210363521A1 (en) 2017-11-09 2021-11-25 Vertex Pharmaceuticals Incorporated 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
CA3084825A1 (en) 2017-12-14 2019-06-20 Crispr Therapeutics Ag Novel rna-programmable endonuclease systems and their use in genome editing and other applications
WO2019126186A1 (en) 2017-12-18 2019-06-27 Neon Therapeutics, Inc. Neoantigens and uses thereof
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
CA3084633A1 (en) 2017-12-21 2019-06-27 Crispr Therapeutics Ag Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
MA51637A (fr) 2018-01-12 2020-11-18 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique par ciblage de la transferrine
EP3749767A1 (de) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materialien und verfahren zur behandlung von hämoglobinopathien
MA51788A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes pour traiter des hémoglobinopathies
US20210130824A1 (en) 2018-02-16 2021-05-06 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting fibrinogen-alpha
AU2019239957A1 (en) 2018-03-19 2020-09-10 Bayer Healthcare Llc Novel RNA-programmable endonuclease systems and uses thereof
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
US20210255185A1 (en) 2018-06-08 2021-08-19 Novartis Ag Cell-based assay for measuring drug product potency
GB201809588D0 (en) 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
AU2019288291A1 (en) 2018-06-18 2021-01-21 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
CA3103485A1 (en) 2018-06-18 2019-12-26 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
BR112020026853A2 (pt) 2018-06-29 2021-04-20 Research Institute At Nationwide Children's Hospital produtos de vírus adeno-associados recombinantes e métodos para tratar a distrofia muscular de cinturas tipo 2a
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
WO2020072700A1 (en) 2018-10-02 2020-04-09 Dana-Farber Cancer Institute, Inc. Hla single allele lines
CA3115524A1 (en) * 2018-10-15 2020-04-23 Win Den Cheung Methods for measuring the infectivity of replication defective viral vectors and viruses
US20210348159A1 (en) 2018-10-17 2021-11-11 Crispr Therapeutics Ag Compositions and methods for delivering transgenes
TW202039859A (zh) 2018-11-30 2020-11-01 美商艾夫西斯公司 Aav病毒載體及其用途
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
CA3122319A1 (en) 2018-12-21 2020-06-25 Genethon Expression cassettes for gene therapy vectors
CN113474840A (zh) 2018-12-21 2021-10-01 百欧恩泰美国公司 用于预测hla ii类特异性表位及表征cd4+ t细胞的方法和系统
AU2019419494A1 (en) 2018-12-31 2021-07-15 Research Institute At Nationwide Children's Hospital DUX4 RNA silencing using RNA targeting CRISPR-Cas13b
EA202192160A1 (ru) 2019-02-04 2021-11-17 Рисерч Инститьют Эт Нэшнуайд Чилдрен'С Хоспитал Доставка полинуклеотида cln3 с помощью аденоассоциированного вируса
MX2021009404A (es) 2019-02-04 2021-11-12 Res Inst Nationwide Childrens Hospital Administracion del virus adenoasociado de polinucleotido de cln6.
MA54951A (fr) 2019-02-15 2021-12-22 Bayer Healthcare Llc Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée
JP2022520875A (ja) 2019-02-25 2022-04-01 ノバルティス アーゲー Ｂｉｅｔｔｉクリスタリン網膜症を治療するための組成物及び方法
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
EP3930765A1 (de) 2019-02-26 2022-01-05 Research Institute at Nationwide Children's Hospital Adenoassoziierte virusvektorverabreichung von b-sarcoglycan und behandlung von muskeldystrophie
MA55297A (fr) 2019-03-12 2022-01-19 Bayer Healthcare Llc Nouveaux systèmes d'endonucléase à arn programmable haute fidélité et leurs utilisations
US20220193259A1 (en) 2019-04-15 2022-06-23 Sanford Research Gene therapy for treating or preventing visual effects in batten disease
EP3966327A1 (de) 2019-05-08 2022-03-16 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two-vektorsystemen zur behandlung von dmd
AU2020278960A1 (en) 2019-05-17 2021-12-23 Research Institute At Nationwide Children's Hospital Optimized gene therapy targeting retinal cells
US20200407729A1 (en) 2019-06-28 2020-12-31 Crispr Therapeutics Ag Materials and methods for controlling gene editing
AU2020319168B2 (en) 2019-07-25 2024-02-29 Novartis Ag Regulatable expression systems
MX2022002132A (es) 2019-08-21 2022-05-18 Res Inst Nationwide Childrens Hospital Administracion de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular.
JP2022552014A (ja) 2019-10-18 2022-12-14 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 Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法
US20220378945A1 (en) 2019-10-18 2022-12-01 Research Institute At Nationwide Children's Hospital Gene therapy targeting cochlear cells
AU2020385387A1 (en) 2019-11-22 2022-06-02 Fondazione Irccs Ca' Granda Ospedale Maggiore Foliclinico Materials and methods for treatment of disorders associated with the IGHMBP2 gene
CN115516100A (zh) 2019-12-20 2022-12-23 全国儿童医院研究所 靶向肌肉疾病中肌肉的优化基因疗法
EP4107266A1 (de) 2020-02-18 2022-12-28 Research Institute at Nationwide Children's Hospital Aav-vermitteltes targeting von mirna bei der behandlung von x-assoziierten erkrankungen
WO2021209521A1 (en) 2020-04-14 2021-10-21 Genethon Vectors for the treatment of acid ceramidase deficiency
CN115867650A (zh) 2020-04-20 2023-03-28 克里斯蒂安娜基因编辑研究公司 用于肺癌治疗的aav递送系统
CA3187353A1 (en) 2020-06-15 2021-12-23 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies
WO2022018638A1 (en) 2020-07-21 2022-01-27 Crispr Therapeutics Ag Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
CA3188420A1 (en) 2020-08-07 2022-02-10 Joseph Bauman Vesicle targeting proteins and uses of same
TW202227634A (zh) 2020-09-08 2022-07-16 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
EP4214317A2 (de) 2020-09-15 2023-07-26 Research Institute at Nationwide Children's Hospital Aav-vermittelte homologieunabhängige gezielte integrationsgeneditierung zur korrektur verschiedener dmd-mutationen bei patienten mit muskeldystrophie
AU2021349277A1 (en) 2020-09-28 2023-05-11 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
WO2022070107A1 (en) 2020-09-30 2022-04-07 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis
WO2022115745A1 (en) 2020-11-30 2022-06-02 Research Institute At Nationwide Children's Hospital Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
CN117083081A (zh) 2020-12-14 2023-11-17 百欧恩泰美国公司 用于癌症免疫疗法的组织特异性抗原
US20220228142A1 (en) 2020-12-17 2022-07-21 Vertex Pharmaceuticals Incorporated Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
WO2022164860A1 (en) 2021-01-27 2022-08-04 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
US20240093191A1 (en) 2021-02-03 2024-03-21 Research Institute At Nationwide Children's Hospital Compositions and methods for treating disease associated with dux4 overexpression
WO2022170038A1 (en) 2021-02-05 2022-08-11 Amicus Therapeutics, Inc. Adeno-associated virus delivery of cln3 polynucleotide
JP2024508324A (ja) 2021-03-04 2024-02-26 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 Ｄｍｄエクソンの重複を修正するためのｃｒｉｓｐｒ－ｃａｓ９を使用したジストロフィンベースのミオパチーの治療のための生成物及び方法
EP4305157A1 (de) 2021-03-09 2024-01-17 Huidagene Therapeutics (Singapore) Pte. Ltd. Manipuliertes crispr/cas13-system und verwendungen davon
CA3216711A1 (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
WO2022226334A1 (en) 2021-04-23 2022-10-27 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
EP4330270A2 (de) 2021-04-30 2024-03-06 Takeda Pharmaceutical Company Limited Aav8-capsid-varianten mit verbessertem leber-targeting
WO2022229703A2 (en) 2021-04-30 2022-11-03 Takeda Pharmaceutical Company, Ltd. New aav8 based immune escaping variants
EP4334447A1 (de) 2021-05-07 2024-03-13 UCL Business Ltd Abca4-genomeditierung
BR112023024078A2 (pt) 2021-05-17 2024-01-30 Sarepta Therapeutics Inc Produção de vetores de aav recombinantes para o tratamento de distrofia muscular
EP4108263A3 (de) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Rekombinante adeno-assoziierte virusprodukte und verfahren zur behandlung von gliedergürtel-muskeldystrophie 2a
CA3222950A1 (en) 2021-06-11 2022-12-15 Bayer Aktiengesellschaft Type v rna programmable endonuclease systems
EP4101928A1 (de) 2021-06-11 2022-12-14 Bayer AG Programmierbare typ-v-rna-endonukleasesysteme
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
IL310725A (en) 2021-08-11 2024-04-01 Solid Biosciences Inc Treatment of muscular dystrophy
EP4144841A1 (de) 2021-09-07 2023-03-08 Bayer AG Neue programmierbare rna-endonuklease-systeme mit verbesserter pam-spezifität und deren verwendung
WO2023042104A1 (en) 2021-09-16 2023-03-23 Novartis Ag Novel transcription factors
WO2023060215A1 (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
WO2023060233A1 (en) 2021-10-08 2023-04-13 Amicus Therapeutics, Inc. Biomarkers for lysosomal storage diseases
US20230139985A1 (en) 2021-10-15 2023-05-04 Research Institute At Nationwide Children's Hospital Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
US20230279431A1 (en) 2021-11-30 2023-09-07 Research Institute At Nationwide Children's Hospital Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
EP4198048A1 (de) 2021-12-16 2023-06-21 Genethon Calpain-3-gentransfersteigerung unter verwendung von modifizierten itr-sequenzen
EP4198047A1 (de) 2021-12-16 2023-06-21 Genethon Erhöhung des gentransfers von fukutin-verwandtem protein unter verwendung modifizierter itr-sequenzen
EP4198134A1 (de) 2021-12-16 2023-06-21 Genethon Transfererhöhung des gamma-sarcoglycan-gens unter verwendung von modifizierten itr-sequenzen
EP4198046A1 (de) 2021-12-16 2023-06-21 Genethon Erhöhung des gentransfers von alpha-sarcoglykan unter verwendung von modifizierten itr-sequenzen
WO2023122669A1 (en) 2021-12-21 2023-06-29 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of limb girdle muscular dystrophy
WO2023118068A1 (en) 2021-12-23 2023-06-29 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2023168400A2 (en) 2022-03-03 2023-09-07 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
WO2023196818A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California Genetic complementation compositions and methods
WO2023214346A1 (en) 2022-05-06 2023-11-09 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
WO2023237587A1 (en) 2022-06-10 2023-12-14 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
WO2024015892A1 (en) 2022-07-13 2024-01-18 The Broad Institute, Inc. Hla-ii immunopeptidome methods and systems for antigen discovery
WO2024035782A1 (en) 2022-08-10 2024-02-15 Aav Gene Therapeutics, Inc. Aav-mediated intramuscular delivery of insulin
WO2024064913A1 (en) 2022-09-23 2024-03-28 Sarepta Therapeutics, Inc. Recombinant aav vectors for treating muscular dystrophy

Family Cites Families (8)

* 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
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
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) * 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
JPH09509564A (ja) * 1993-11-09 1997-09-30 ターゲテッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成
DK0728214T3 (da) * 1993-11-09 2004-11-29 Targeted Genetics Corp Stabile cellelinjer, der er i stand til at udtrykke det adenoassocierede virusreplikationsgen
FR2716682B1 (fr) * 1994-01-28 1996-04-26 Centre Nat Rech Scient Procédé de préparation de virus adéno-associés (AAV) recombinants et utilisations.
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods

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