PT728214E - Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado - Google Patents

Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado

Info

Publication number
PT728214E
PT728214E PT95900505T PT95900505T PT728214E PT 728214 E PT728214 E PT 728214E PT 95900505 T PT95900505 T PT 95900505T PT 95900505 T PT95900505 T PT 95900505T PT 728214 E PT728214 E PT 728214E
Authority
PT
Portugal
Prior art keywords
pct
aav
cell lines
express
additional
Prior art date
Application number
PT95900505T
Other languages
English (en)
Inventor
James P Trempe
Qicheng Yang
Original Assignee
Ohio Med College
Targeted Genetics Corp
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 Ohio Med College, Targeted Genetics Corp filed Critical Ohio Med College
Publication of PT728214E publication Critical patent/PT728214E/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
    • 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

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PT95900505T 1993-11-09 1994-11-03 Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado PT728214E (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US15106593A 1993-11-09 1993-11-09
US27356394A 1994-07-11 1994-07-11

Publications (1)

Publication Number Publication Date
PT728214E true PT728214E (pt) 2004-11-30

Family

ID=26848297

Family Applications (1)

Application Number Title Priority Date Filing Date
PT95900505T PT728214E (pt) 1993-11-09 1994-11-03 Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado

Country Status (11)

Country Link
US (1) US5837484A (pt)
EP (1) EP0728214B1 (pt)
JP (1) JP3952312B2 (pt)
AT (1) ATE272123T1 (pt)
AU (1) AU678867B2 (pt)
CA (1) CA2176215C (pt)
DE (1) DE69433922T2 (pt)
DK (1) DK0728214T3 (pt)
ES (1) ES2220923T3 (pt)
PT (1) PT728214E (pt)
WO (1) WO1995013392A1 (pt)

Families Citing this family (267)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5436146A (en) * 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
PT733103E (pt) * 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
JPH10501686A (ja) 1994-04-13 1998-02-17 ザ ロックフェラー ユニヴァーシティ 神経系の細胞へのdnaのaav仲介送達
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
EP0785991A1 (de) * 1994-10-13 1997-07-30 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
WO1996017947A1 (en) * 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5843742A (en) * 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
US6752987B1 (en) 1995-02-28 2004-06-22 The Regents Of The University Of California Adenovirus encoding human adenylylcyclase (AC) VI
JP3961019B2 (ja) 1995-02-28 2007-08-15 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 遺伝子転移媒介の血管形成療法
US5756283A (en) * 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US6093570A (en) * 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
EP0842287B1 (en) * 1995-08-03 2004-02-04 Avigen, Inc. High efficiency helper system for aav vector production
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
US6162796A (en) * 1995-09-27 2000-12-19 The Rockefeller University Method for transferring genes to the heart using AAV vectors
US6086913A (en) * 1995-11-01 2000-07-11 University Of British Columbia Liposomal delivery of AAV vectors
US6004797A (en) * 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
US20020193580A1 (en) * 1995-12-15 2002-12-19 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20060088938A1 (en) * 1995-12-15 2006-04-27 Mitchell Lloyd G Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants
US20030027250A1 (en) * 1995-12-15 2003-02-06 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US5952221A (en) * 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
DE19608751B4 (de) 1996-03-06 2006-05-18 Medigene Ag Verwendung eines Adeno-assoziierten Virus-Vektors zur Steigerung der Immunogenität von Zellen
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
CA2264499A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
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
AU722624B2 (en) * 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
AU742696B2 (en) * 1996-11-18 2002-01-10 Mcgill University Post-mitotic neurons containing adenovirus vectors that modulate apoptosis and growth
US6541258B2 (en) 1996-12-18 2003-04-01 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
ES2224375T3 (es) * 1997-04-14 2005-03-01 Cell Genesys, Inc. Metodos para aumentar la eficacia del producto de aav recombinante.
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU758708B2 (en) 1997-09-05 2003-03-27 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released 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
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
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
DK1930418T3 (da) 1998-09-04 2015-07-13 Genzyme Corp Fremgangsmåder til frembringelse af hjælperfri præparater med høj titer af frigjorte rekombinante AAV-vektorer
EP1939215A1 (en) 1998-10-22 2008-07-02 The University of Montana Omp85 proteins of neisseria gonorrhoeae and neisseria meningitidis, compositions containing same and methods of use thereof
US6303362B1 (en) * 1998-11-19 2001-10-16 The Board Of Trustees Of The Leland Stanford Junior University Adenoviral vector and methods for making and using the same
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6893865B1 (en) * 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
US6793926B1 (en) * 1999-05-27 2004-09-21 Genovo, Inc. Methods for production of a recombinant adeno-associated virus
JP2003501043A (ja) * 1999-05-28 2003-01-14 ターゲティッド ジェネティクス コーポレイション 腫瘍壊死因子(tnf)関連障害において、tnfのレベルを低下させるための方法および組成物
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
AU781958C (en) 1999-08-09 2006-03-30 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
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
US6627617B1 (en) 1999-10-01 2003-09-30 University Of North Carolina At Chapel Hill Temperature-sensitive regulation of viral vector production
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
AU7879000A (en) * 1999-10-12 2001-04-23 University Of North Carolina At Chapel Hill, The Adeno-associated virus vectors encoding factor viii and methods of using the same
AU2460001A (en) 1999-12-27 2001-07-09 Regents Of The University Of California, The Gene therapy for congestive heart failure
US6916635B2 (en) * 2000-10-02 2005-07-12 The Research Foundation Of State University Of New York Hybrid adenovirus/adeno-associated virus vectors and methods of use thereof
US20040126774A1 (en) * 2001-01-08 2004-07-01 Mitchell Lioyd G. Correction of factor VIII genetic defects using spliceosome mediated RNA trans splicing
US6844192B2 (en) 2001-06-29 2005-01-18 Wake Forest University Adenovirus E4 protein variants for virus production
EP1427816A4 (en) 2001-09-18 2004-12-01 Clontech Lab Inc METHOD FOR PRODUCING ADENOVIRAL VECTORS BASED ON A SITE SPECIFIC RECOMBINASE
US7399753B2 (en) * 2002-02-25 2008-07-15 Virxsys Corporation Trans-splicing mediated photodynamic therapy
US7332276B2 (en) 2002-03-01 2008-02-19 Celltech R&D, Inc. Methods to increase or decrease bone density
EP1504016A4 (en) * 2002-05-08 2006-11-22 Intronn Inc USE OF SPLICEOSOM-MEDIATED RNA-TRANS-SPLICING TO GIVE ADENOVIRES A CELLSELECTIVE REPLICATION
AU2003285009B2 (en) * 2002-10-23 2010-10-07 Virxsys Corporation Screening methods for identification of efficient pre-trans-splicing molecules
ATE521701T1 (de) 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
WO2004075861A2 (en) * 2003-02-26 2004-09-10 Children's Hospital, Inc. Recombinant adeno-associated virus production
US20060177933A1 (en) * 2004-01-23 2006-08-10 Madaiah Puttaraju Expression of apoA-1 and variants thereof using spliceosome mediated RNA trans-splicing
US8053232B2 (en) * 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
US7968334B2 (en) * 2004-01-23 2011-06-28 Virxsys Corporation Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
EP1723227A4 (en) 2004-02-10 2007-09-19 Dartmouth College NICOTINAMIDRIBOSIDE KINASE COMPOSITIONS AND METHOD FOR THEIR USE
US20060094110A1 (en) * 2004-07-30 2006-05-04 Mcgarrity Gerard J Use of spliceosome mediated RNA trans-splicing for immunotherapy
US20060134658A1 (en) * 2004-08-09 2006-06-22 Garcia-Blanco Mariano A Use of RNA trans-splicing for generation of interfering RNA molecules
US7166963B2 (en) * 2004-09-10 2007-01-23 Axcelis Technologies, Inc. Electrodeless lamp for emitting ultraviolet and/or vacuum ultraviolet radiation
DE102004047492B4 (de) * 2004-09-23 2006-07-20 Jost-Werke Gmbh & Co. Kg Verfahren zum Übertragen von elektrischer, pneumatischer oder hydraulischer Energie sowie ein Energieübertragungssystem
US7871795B2 (en) 2004-10-08 2011-01-18 Virxsys Corporation Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
AU2005326784B2 (en) * 2004-10-08 2012-03-15 Virxsys Corporation Use of RNA trans-splicing for antibody gene transfer and antibody polypeptide production
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
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
JP2012521750A (ja) 2009-03-27 2012-09-20 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 肝硬変及び肝線維症の治療のための方法及び組成物
SI3421603T1 (sl) 2009-05-02 2022-02-28 Genzyme Corporation Genska terapija za nevrodegenerativne motnje
AU2010316996A1 (en) 2009-11-05 2012-06-14 Proyecto De Biomedicina Cima, S.L. Regulated expression systems
JP2013544600A (ja) 2010-11-23 2013-12-19 プレサージュ バイオサイエンシズ,インコーポレイテッド 固体デリバリーのための治療方法および組成物
US20140155469A1 (en) 2011-04-19 2014-06-05 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors and viruses
WO2012145597A2 (en) 2011-04-21 2012-10-26 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
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
CA2842798C (en) 2011-07-25 2021-02-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
US9434928B2 (en) 2011-11-23 2016-09-06 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
BR112015002168A2 (pt) 2012-08-01 2017-11-07 Nationwide Childrens Hospital liberação intratecal de vírus 9 adeno-associado recombinante
US9539307B2 (en) 2012-09-17 2017-01-10 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
JP6576904B2 (ja) 2013-04-04 2019-09-18 トラスティーズ・オブ・ダートマス・カレッジ HIV−1プロウイルスDNAのinvivo切除のための組成物及び方法
CA2909085C (en) 2013-04-08 2023-08-29 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
EA032706B1 (ru) 2013-04-20 2019-07-31 Рисёрч Инститъют Эт Нэйшнвайд Чилдрен'С Хоспитал ДОСТАВКА НАЦЕЛЕННЫХ НА ЭКЗОН 2 ПОЛИНУКЛЕОТИДНЫХ КОНСТРУКЦИЙ U7snRNA ПРИ ПОМОЩИ РЕКОМБИНАНТНОГО АДЕНОАССОЦИИРОВАННОГО ВИРУСА
SG11201509419QA (en) 2013-05-15 2015-12-30 Univ Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
EP3039146B1 (en) 2013-08-27 2020-05-27 Research Institute at Nationwide Children's Hospital Products and methods for treatment of amyotrophic lateral sclerosis
WO2015069647A1 (en) 2013-11-05 2015-05-14 The Research Institute At Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS
US10047130B2 (en) 2014-03-18 2018-08-14 Washington University Methods and compositions for red-shifted chromophore substitution for optogenetic applications
JP6986444B2 (ja) 2014-08-09 2021-12-22 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遺伝子のエクソン5内の内部リボソーム進入部位を活性化するための方法及び材料
WO2016057975A2 (en) 2014-10-10 2016-04-14 Research Institute At Nationwide Children's Hospital Guided injections for aav gene transfer to muscle
AU2015342997B2 (en) 2014-11-05 2021-11-18 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
WO2016122791A1 (en) 2015-01-30 2016-08-04 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
WO2016135558A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
KR102406615B1 (ko) 2015-05-15 2022-06-08 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 치료적 전달
US10017832B2 (en) 2015-08-25 2018-07-10 Washington University Compositions and methods for site specific recombination at asymmetric sites
CN108136049A (zh) 2015-09-17 2018-06-08 全国儿童医院研究所 Galgt2基因治疗的方法和材料
US20190048340A1 (en) 2015-09-24 2019-02-14 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
AU2016344609B2 (en) 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
BR112018008971A2 (pt) 2015-11-06 2018-11-27 Crispr Therapeutics Ag materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
CA3005633C (en) 2015-11-16 2023-11-21 Research Institute Of Nationwide Children's Hospital Materials and methods for treatment of titin-based myopathies and other titinopathies
EP3384024B1 (en) 2015-12-01 2022-02-02 CRISPR Therapeutics AG Materials and methods for treatment of alpha-1 antitrypsin deficiency
CN109312339B (zh) 2015-12-23 2022-01-28 克里斯珀医疗股份公司 用于治疗肌萎缩侧索硬化症和/或额颞叶变性的材料和方法
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
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US20190112353A1 (en) 2016-02-18 2019-04-18 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
CA3015633A1 (en) 2016-02-26 2017-08-31 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inducing dux4 exon skipping
WO2017158422A1 (en) 2016-03-16 2017-09-21 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
EP3436427B1 (en) 2016-03-28 2021-12-08 The Regents of the University of California Method and composition for treating neuronal hyper-excitability
BR112018070249A2 (pt) 2016-04-02 2019-01-29 Research Institute At Nationwide Children's Hospital sistema promotor de u6 modificada para expressão específica de tecido
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
MA44681A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration de b-sarcoglycane et de microarn-29 à l'aide d'un vecteur de virus adéno-associé, et traitement de la dystrophie musculaire
JP6974349B2 (ja) 2016-04-18 2021-12-01 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 ヘモグロビン異常症の処置のための材料及び方法
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US11174469B2 (en) 2016-06-29 2021-11-16 Crispr Therapeutics Ag Materials and methods for treatment of Amyotrophic Lateral Sclerosis (ALS) and other related disorders
EP3478829A1 (en) 2016-06-29 2019-05-08 Crispr Therapeutics AG Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
AU2017290614C1 (en) 2016-06-29 2024-01-18 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
JP7305534B2 (ja) 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
WO2018007980A1 (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
JP7106534B2 (ja) 2016-11-17 2022-07-26 ネイションワイド チルドレンズ ホスピタル, インコーポレイテッド メチル-cpg結合タンパク質2をコードする組換えアデノ随伴ウイルスの髄腔内送達
RU2019119272A (ru) 2016-11-23 2020-12-24 Гритстоун Онколоджи, Инк. Вирусная доставка неоантигенов
CN118546256A (zh) 2016-12-13 2024-08-27 西雅图儿童医院(Dba西雅图儿童研究所) 在体外和体内对工程化的细胞中表达的化学物质诱导的信号传导复合物进行外源性药物激活的方法
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
WO2018150271A1 (en) 2017-02-17 2018-08-23 Lonza Ltd. Mammalian cells for producing adeno-associated viruses
EP3585899A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders
EP3585898A1 (en) 2017-02-22 2020-01-01 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
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
CA3054031A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Compositions and methods for gene editing
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
KR20240112964A (ko) 2017-03-17 2024-07-19 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 근이영양증을 치료하기 위한 근육 특이적 마이크로-디스트로핀의 아데노-관련 바이러스 벡터 전달
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
WO2018208856A1 (en) 2017-05-08 2018-11-15 Gritstone Oncology, Inc. Alphavirus neoantigen vectors
US11622977B2 (en) 2017-05-12 2023-04-11 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
CN111065741A (zh) 2017-07-08 2020-04-24 吉尼松公司 脊髓性肌萎缩症的治疗
CA3070654C (en) 2017-07-25 2021-07-27 Oxford Genetics Limited Adenoviral vectors
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
CN111433367A (zh) 2017-10-20 2020-07-17 全国儿童医院研究所 Nt-3基因疗法的方法和材料
US20210180091A1 (en) 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
WO2019094253A1 (en) 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
WO2019092505A1 (en) 2017-11-09 2019-05-16 Casebia Therapeutics Llp Self-inactivating (sin) crispr/cas or crispr/cpf1 systems and uses thereof
AU2018372763A1 (en) 2017-11-21 2020-05-28 Bayer Healthcare Llc Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa
AU2018386301A1 (en) 2017-12-14 2020-06-18 Bayer Healthcare Llc Novel RNA-programmable endonuclease systems and their use in genome editing and other applications
JP7402163B2 (ja) 2017-12-21 2023-12-20 クリスパー セラピューティクス アーゲー 2a型アッシャー症候群の処置のための材料および方法
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)
JP2021509823A (ja) 2018-01-04 2021-04-08 アイコニック セラピューティクス インコーポレイテッド 抗組織因子抗体、抗体薬物コンジュゲート、及び関連する方法
MA51637A (fr) 2018-01-12 2020-11-18 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique par ciblage de la transferrine
MA51788A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes pour traiter des hémoglobinopathies
WO2019150196A1 (en) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
MA51869A (fr) 2018-02-16 2020-12-23 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique par ciblage du fibrinogène-alpha
WO2019183150A1 (en) 2018-03-19 2019-09-26 Casebia Therapeutics Limited Liability Partnership 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
SG11202007874XA (en) 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Rapamycin resistant cells
WO2019236949A1 (en) 2018-06-08 2019-12-12 Avexis Inc. 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
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
AU2019288291B2 (en) 2018-06-18 2024-09-26 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
US20210277362A1 (en) 2018-06-29 2021-09-09 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
US20210324417A1 (en) 2018-08-29 2021-10-21 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of mutant gars protein
JP7320601B2 (ja) 2018-09-11 2023-08-03 上▲海▼市公共▲衛▼生▲臨▼床中心 広域スペクトルな抗インフルエンザワクチン免疫原及びその使用
CA3116885A1 (en) 2018-10-17 2020-04-23 Crispr Therapeutics Ag Compositions and methods for delivering transgenes
TWI844587B (zh) 2018-11-30 2024-06-11 瑞士商諾華公司 Aav病毒載體及其用途
US20220042045A1 (en) 2018-12-21 2022-02-10 Genethon Expression cassettes for gene therapy vectors
EP3906308A1 (en) 2018-12-31 2021-11-10 Research Institute at Nationwide Children's Hospital Dux4 rna silencing using rna targeting crispr-cas13b
EP3921430A1 (en) 2019-02-04 2021-12-15 Research Institute at Nationwide Children's Hospital Adeno-associated virus delivery of cln3 polynucleotide
MX2021009404A (es) 2019-02-04 2021-11-12 Res Inst Nationwide Childrens Hospital Administracion del virus adenoasociado de polinucleotido de cln6.
WO2020168362A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
JP2022521025A (ja) 2019-02-25 2022-04-04 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
CN113474461A (zh) 2019-02-25 2021-10-01 诺华股份有限公司 治疗bietti晶体营养不良的组合物和方法
BR112021016570A2 (pt) 2019-02-26 2021-11-03 Res Inst Nationwide Childrens Hospital Entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscular
SG11202109741VA (en) 2019-03-12 2021-10-28 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
AU2020253435A1 (en) 2019-04-01 2021-11-18 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
SG11202111353QA (en) 2019-04-15 2021-11-29 Univ Iowa Res Found Methods and compositions for transgene expression
SG11202111334SA (en) 2019-04-15 2021-11-29 Univ Iowa Res Found Compositions and methods for treatment of cystic fibrosis
US20220193259A1 (en) 2019-04-15 2022-06-23 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
US20220226507A1 (en) 2019-05-17 2022-07-21 Research Institute At Nationwide Children's Hospital Optimized gene therapy targeting retinal cells
SG11202113187WA (en) 2019-05-30 2021-12-30 Gritstone Bio Inc Modified adenoviruses
WO2020264254A1 (en) 2019-06-28 2020-12-30 Crispr Therapeutics Ag Materials and methods for controlling gene editing
WO2021014428A1 (en) 2019-07-25 2021-01-28 Novartis Ag Regulatable expression systems
PL4017871T3 (pl) 2019-08-21 2024-07-08 Research Institute At Nationwide Children's Hospital Dostarczanie alfa-sarkoglikanu za pomocą wektora wirusa związanego z adenowirusami i leczenie dystrofii mięśniowej
US20230416776A1 (en) 2019-10-08 2023-12-28 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
WO2021077115A1 (en) 2019-10-18 2021-04-22 Research Institute At Nationwide Children's Hospital Gene therapy targeting cochlear cells
EP4045662A1 (en) 2019-10-18 2022-08-24 Research Institute at Nationwide Children's Hospital Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
EP4061831A1 (en) 2019-11-22 2022-09-28 Research Institute at Nationwide Children's Hospital Materials and methods for treatment of disorders associated with the ighmbp2 gene
EP4077687A1 (en) 2019-12-20 2022-10-26 Research Institute at Nationwide Children's Hospital Optimized gene therapy for targeting muscle in muscle diseases
KR20220155999A (ko) 2020-02-18 2022-11-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 X-연관 장애의 치료에서의 miRNA의 AAV-매개 표적화
JP2023523573A (ja) 2020-04-14 2023-06-06 ジェネトン 酸性セラミダーゼ欠乏症を処置するためのベクター
BR112022025586A2 (pt) 2020-06-15 2023-03-07 Res Inst Nationwide Childrens Hospital Liberação de vetor de vírus adenoassociado para distrofias musculares
WO2022006253A2 (en) 2020-06-30 2022-01-06 University Of Iowa Research Foundation Methods and compositions for administering recombinant viral vectors
US20230374483A1 (en) 2020-07-08 2023-11-23 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
WO2022018638A1 (en) 2020-07-21 2022-01-27 Crispr Therapeutics Ag Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
WO2022026927A1 (en) 2020-07-30 2022-02-03 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
JP2023541108A (ja) 2020-08-06 2023-09-28 グリットストーン バイオ インコーポレイテッド マルチエピトープワクチンカセット
US20230414787A1 (en) 2020-08-27 2023-12-28 University Of Iowa Research Foundation Gene knock-out for treatment of glaucoma
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
JP2023541444A (ja) 2020-09-15 2023-10-02 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィー患者における多様なdmd変異の補正のためのaav媒介性の相同性非依存的標的化組み込み遺伝子編集
EP4217375A1 (en) 2020-09-28 2023-08-02 Research Institute at Nationwide Children's Hospital Products and methods for treating muscular dystrophy
US20230392134A1 (en) 2020-09-30 2023-12-07 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis
AU2021385595A1 (en) 2020-11-30 2023-06-29 Research Institute At Nationwide Children's Hospital Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
WO2022133246A1 (en) 2020-12-17 2022-06-23 Vertex Pharmaceuticals Incorporated Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
CA3209471A1 (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
EP4288556A1 (en) 2021-02-05 2023-12-13 Regents of the University of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses
EP4301462A1 (en) 2021-03-04 2024-01-10 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
US20240189452A1 (en) 2021-04-13 2024-06-13 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
EP4323011A1 (en) 2021-04-15 2024-02-21 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis
AU2022262420A1 (en) 2021-04-23 2023-11-16 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
EP4334447A1 (en) 2021-05-07 2024-03-13 UCL Business Ltd Abca4 genome editing
WO2022245675A1 (en) 2021-05-17 2022-11-24 Sarepta Therapeutics, Inc. Production of recombinant aav vectors for treating muscular dystrophy
EP4108263A3 (en) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
EP4352214A1 (en) 2021-06-11 2024-04-17 Bayer AG Type v rna programmable endonuclease systems
EP4101928A1 (en) 2021-06-11 2022-12-14 Bayer AG 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
IL310725A (en) 2021-08-11 2024-04-01 Solid Biosciences Inc Treatment of muscular dystrophy
EP4144841A1 (en) 2021-09-07 2023-03-08 Bayer AG Novel small rna programmable endonuclease systems with impoved pam specificity and uses thereof
WO2023042104A1 (en) 2021-09-16 2023-03-23 Novartis Ag Novel transcription factors
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
EP4413376A1 (en) 2021-10-08 2024-08-14 Amicus Therapeutics, Inc. Biomarkers for lysosomal storage diseases
JP2023059858A (ja) 2021-10-15 2023-04-27 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
JP2023081369A (ja) 2021-11-30 2023-06-09 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
EP4198047A1 (en) 2021-12-16 2023-06-21 Genethon Fukutin related protein gene transfer increase using modified itr sequences
EP4198048A1 (en) 2021-12-16 2023-06-21 Genethon Calpain-3 gene transfer increase using modified itr sequences
EP4198046A1 (en) 2021-12-16 2023-06-21 Genethon Alpha-sarcoglycan gene transfer increase using modified itr sequences
EP4198134A1 (en) 2021-12-16 2023-06-21 Genethon Gamma-sarcoglycan gene transfer increase using modified itr sequences
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
WO2023166425A1 (en) 2022-03-01 2023-09-07 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions
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
TW202404993A (zh) 2022-04-11 2024-02-01 美商特納亞治療股份有限公司 具經工程化蛋白殼之腺相關病毒
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
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
WO2024081706A1 (en) 2022-10-11 2024-04-18 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)
WO2024086747A1 (en) 2022-10-19 2024-04-25 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2024092171A1 (en) 2022-10-26 2024-05-02 University Of Iowa Research Foundation Method to deliver large genes using virus and a dna recombination system
WO2024092126A1 (en) 2022-10-27 2024-05-02 Cargo Therapeutics, Inc. Compositions and methods for improved immunotherapies
WO2024129743A2 (en) 2022-12-13 2024-06-20 Bluerock Therapeutics Lp 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
WO2024163560A1 (en) 2023-02-01 2024-08-08 Sarepta Therapeutics, Inc. Raav production methods
WO2024168276A2 (en) 2023-02-09 2024-08-15 Cargo Therapeutics, Inc. Compositions and methods for immunotherapies
WO2024220592A2 (en) 2023-04-18 2024-10-24 Research Institute At Nationwide Children's Hospital, Inc. Gene therapy for treating limb girdle muscular dystrophy r9 and congenital muscular dystrophy 1c

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
US5141742A (en) * 1986-02-07 1992-08-25 Oncogen Vaccines against melanoma
AU7906691A (en) * 1990-05-23 1991-12-10 United States of America, as represented by the Secretary, U.S. Department of Commerce, The 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
DE4436664A1 (de) * 1994-10-13 1996-07-04 Max Planck Gesellschaft Bereitstellung von rep-negativen AAV-Mutanten und hierfür verwendbare Zellen

Also Published As

Publication number Publication date
EP0728214B1 (en) 2004-07-28
US5837484A (en) 1998-11-17
ES2220923T3 (es) 2004-12-16
JP3952312B2 (ja) 2007-08-01
CA2176215C (en) 2007-06-26
EP0728214A1 (en) 1996-08-28
JPH09510602A (ja) 1997-10-28
AU8130994A (en) 1995-05-29
AU678867B2 (en) 1997-06-12
ATE272123T1 (de) 2004-08-15
DE69433922T2 (de) 2005-07-28
EP0728214A4 (en) 1997-02-12
DE69433922D1 (de) 2004-09-02
WO1995013392A1 (en) 1995-05-18
DK0728214T3 (da) 2004-11-29
CA2176215A1 (en) 1995-05-18

Similar Documents

Publication Publication Date Title
PT728214E (pt) Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
MX9709549A (es) Adenovirus recombinantes, su uso para preparar aav, linea celular complementaria y composiciones farmaceuticas que los contienen.
AU7497494A (en) Recombinant vector containing a sequence of a lipoprotein gene for the expression of nucleotide sequences
AU1539595A (en) Process for preparing recombinant adeno-associated viruses (AAVs), and uses
EP1164195A3 (en) Adeno-associated virus with inverted terminal repeat sequences as promoter
AU1189195A (en) Recombinant protein production in bovine adenovirus expression vector system
ES2103266T3 (es) Procedimiento de dna recombinante y celulas huesped.
WO1994008002A3 (en) Methods and compositions for controlling translation of hcv proteins
HU9202458D0 (en) Expressing systems
CA2304105A1 (en) Control pod dehiscence or shatter
PT988391E (pt) Vectores adenovirais recombinantes compreendendo uma sequencia de splicing
WO1996001317A3 (en) Mammalian peroxisome proliferator-activated receptors and uses thereof
WO1994013812A3 (en) Novel entomopoxvirus genes, proteins and methods of use thereof
AU6724794A (en) GRB3-3 gene, its variants and their uses
AU4835696A (en) Amplifying sequences, vectors comprising these sequences and their uses in compositions for the expression of nucleotide sequences in transfected cells, therapeutic and vaccine applications.
DE69632201D1 (de) Regulierte expression von proteinen in stabil transformierten säugetierzellen
CA2033069A1 (en) Saf polypeptide, gene, promoter and use for expression in streptomyces
HUP9802587A2 (hu) Glükózzal indukálható rekombináns virális vektor, ezt tartalmazó gyógyszerkészítmény és ezzel transzformált sejtek
CA2139430A1 (fr) Polypeptides ayant une activite de recepteur serotoninergique (5ht5a), acides nucleiques codant pour ces polypeptides et utilisations
GB2318793A (en) Mpt83 gene from mycobacterium tuberculosis
WO1992014818A3 (en) Entomopoxvirus expression system comprising spheroidin or thymidine-kinase sequences
DK0528925T3 (da) Klonings- og/eller ekspressionsvektorer, deres fremstilling og anvendelse