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ATE272123T1 - Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren - Google Patents

Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren

Info

Publication number
ATE272123T1
ATE272123T1 AT95900505T AT95900505T ATE272123T1 AT E272123 T1 ATE272123 T1 AT E272123T1 AT 95900505 T AT95900505 T AT 95900505T AT 95900505 T AT95900505 T AT 95900505T AT E272123 T1 ATE272123 T1 AT E272123T1
Authority
AT
Austria
Prior art keywords
pct
aav
adeno
express
cell line
Prior art date
Application number
AT95900505T
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
Application granted granted Critical
Publication of ATE272123T1 publication Critical patent/ATE272123T1/de

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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • 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)
AT95900505T 1993-11-09 1994-11-03 Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren ATE272123T1 (de)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US15106593A 1993-11-09 1993-11-09
US27356394A 1994-07-11 1994-07-11
PCT/US1994/012582 WO1995013392A1 (en) 1993-11-09 1994-11-03 Stable cell lines capable of expressing the adeno-associated virus replication gene

Publications (1)

Publication Number Publication Date
ATE272123T1 true ATE272123T1 (de) 2004-08-15

Family

ID=26848297

Family Applications (1)

Application Number Title Priority Date Filing Date
AT95900505T ATE272123T1 (de) 1993-11-09 1994-11-03 Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren

Country Status (11)

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

Families Citing this family (293)

* 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
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
EP0755454B1 (de) 1994-04-13 2008-02-13 The Rockefeller University Aav-vermittelte überbringung von dna in zellen des nervensystems
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
JPH10507352A (ja) * 1994-10-13 1998-07-21 ドイチェス クレブスフォルシュンクスツェントルム スチフトゥング デス エッフェントリヒェン レヒツ ｒｅｐ陰性ＡＡＶ変異体の調製およびそれに使用可能な細胞
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US6342390B1 (en) * 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
US6326356B1 (en) 1996-10-18 2001-12-04 Board Of Regents, The University Of Texas System Suppression of neu overexpression using a mini-E1A gene
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
EP0796339A1 (de) * 1994-12-06 1997-09-24 Targeted Genetics Corporation Verpackungszelllinien zur bereitstellung rekombinanter aav vektoren mit einem hohen titer
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
US20020103147A1 (en) 1997-09-05 2002-08-01 Hammond H. Kirk Gene therapy for congestive heart failure
CA2188575A1 (en) 1995-02-28 1996-09-06 H. Kirk Hammond Gene transfer-mediated angiogenesis therapy
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
US5622856A (en) * 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
WO1997006272A2 (en) * 1995-08-03 1997-02-20 Avigen, Inc. 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
DE19625188A1 (de) * 1996-06-24 1998-01-08 Medigene Gmbh System zur Herstellung von AAV-Vektoren
US6541012B2 (en) 1996-06-24 2003-04-01 Christoph Bogedain System for the production of AAV vectors
US6294370B1 (en) 1997-06-24 2001-09-25 Medigene Ag System for the production of AAV vectors
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
JP2001500015A (ja) * 1996-09-06 2001-01-09 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 Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
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
US6060247A (en) * 1996-11-18 2000-05-09 Mcgill University Post-mitotic neurons containing adenovirus vectors that modulate apoptosis and growth
CA2270285A1 (en) * 1996-12-18 1998-06-25 Targeted Genetics Corporation Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
AU728220B2 (en) * 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
CA2682108C (en) 1997-09-05 2013-12-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) * 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released 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
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
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
EP2942393A1 (de) 1998-09-04 2015-11-11 Genzyme Corporation Verfahren zur erzeugung helferfreier präparate mit hohem titer aus freigesetzten rekombinanten aav-vektoren
DE69839740D1 (de) 1998-10-22 2008-08-28 Univ Montana Vakzine enthaltend Omp85 Proteine von Neisseria gonorrhoeae und Neisseria meningitidis
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
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
DK1180159T3 (da) * 1999-05-28 2008-11-17 Targeted Genetics Corp Fremgangsmåder og sammensætninger til at sænke niveauet af Tumor-Nekrose-Faktor (TNF) i TNF-associerede lidelser
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
JP4827353B2 (ja) * 1999-08-09 2011-11-30 ターゲティッド ジェネティクス コーポレイション 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大
WO2001025253A2 (en) 1999-10-01 2001-04-12 The 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
WO2001027303A1 (en) * 1999-10-12 2001-04-19 The University Of North Carolina At Chapel Hill Adeno-associated virus vectors encoding factor viii and methods of using the same
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 (de) 2001-09-18 2004-12-01 Clontech Lab Inc Auf stellenspezifischer rekombinase beruhendes verfahren zur herstellung von adenovirusvektoren
US7399753B2 (en) * 2002-02-25 2008-07-15 Virxsys Corporation Trans-splicing mediated photodynamic therapy
WO2003073991A2 (en) * 2002-03-01 2003-09-12 Celltech R & D, Inc. Methods to increase or decrease bone density
AU2003302216A1 (en) * 2002-05-08 2004-06-23 Virxsys Corporation Use of spliceosome mediated rna trans-splicing to confer cell selective replication to adenoviruses
ATE471387T1 (de) * 2002-10-23 2010-07-15 Virxsys Corp Screening-verfahren zur identifizierung wirksamer prä-trans-spleissmoleküle
WO2004064750A2 (en) 2003-01-22 2004-08-05 Duke University Improved constructs for expressing lysosomal polypeptides
WO2004075861A2 (en) * 2003-02-26 2004-09-10 Children's Hospital, Inc. Recombinant adeno-associated virus production
US7968334B2 (en) * 2004-01-23 2011-06-28 Virxsys Corporation Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
JP2007518423A (ja) * 2004-01-23 2007-07-12 イントロン、インコーポレイテッド スプライセオソーム仲介型ｒｎａトランススプライシングを使用するアポａ−１及びその変異体の発現
US8053232B2 (en) * 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
EP1723227A4 (de) 2004-02-10 2007-09-19 Dartmouth College Nicotinamidribosid-kinase-zusammensetzungen und verfahren zu deren verwendung
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
CA2583306A1 (en) * 2004-10-08 2006-08-10 Intronn, Inc. Use of rna trans-splicing for antibody gene transfer and antibody polypeptide production
CA2693178C (en) 2006-11-29 2018-12-04 Nationwide Children's Hospital, Inc. Myostatin inhibition for enhancing muscle and/or improving muscle function
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US20120093775A1 (en) 2009-03-27 2012-04-19 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
EP3988660B1 (de) 2009-05-02 2025-05-14 Genzyme Corporation Gentherapie für neurodegenerative erkrankungen
EP2497830A1 (de) 2009-11-05 2012-09-12 Proyecto de Biomedicina Cima, S.L. Regulierte expressionssysteme
AU2011332815A1 (en) 2010-11-23 2013-05-30 Presage Biosciences, Inc. Therapeutic methods and compositions for solid delivery
WO2012145509A2 (en) 2011-04-19 2012-10-26 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors, and viruses
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
CA3106285A1 (en) 2011-07-25 2013-01-31 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
PT2879719T (pt) 2012-08-01 2018-10-08 Ohio State Innovation Foundation Administração intratecal do vírus adeno-associado 9 recombinante
AU2013315007A1 (en) 2012-09-17 2015-04-09 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
WO2014165349A1 (en) 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
AU2014251099B2 (en) 2013-04-08 2019-01-17 The University Of Kansas Chimeric adeno-associated virus/ bocavirus parvovirus vector
CN109652385A (zh) 2013-04-20 2019-04-19 全国儿童医院研究所 外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
CA2912678C (en) 2013-05-15 2023-10-10 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
HRP20200862T1 (hr) 2013-08-27 2020-08-21 Research Institute At Nationwide Children's Hospital Proizvodi i metode liječenja amiotrofične lateralne skleroze
JP2016538276A (ja) 2013-11-05 2016-12-08 ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルＴｈｅ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ Ａｔ Ｎａｔｉｏｎｗｉｄｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ 筋萎縮性側索硬化症の処置のためのＮＦ−κＢおよびＳＯＤ−１を阻害する組成物および方法
WO2015142984A1 (en) 2014-03-18 2015-09-24 Washington University Methods and compositions for red-shifted chromophore substitution for optogenetic applications
EP3572516B1 (de) 2014-08-09 2024-11-20 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
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
US20180008727A1 (en) 2015-01-30 2018-01-11 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
AU2016225179C1 (en) 2015-02-23 2022-11-03 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
SI3294323T1 (sl) 2015-05-15 2022-06-30 Regenxbio Inc. Adenoasociacijski virus za terapevtsko dostavo v centralni živčni sistem
US10017832B2 (en) 2015-08-25 2018-07-10 Washington University Compositions and methods for site specific recombination at asymmetric sites
US10980897B2 (en) 2015-09-17 2021-04-20 Research Institute At Nationwide Children's Hospital Methods and materials for GALGT2 gene therapy
JP2018532402A (ja) 2015-09-24 2018-11-08 クリスパー セラピューティクス アーゲー Ｒｎａプログラム可能エンドヌクレアーゼの新規のファミリーならびにゲノム編集および他の適用におけるそれらの使用
CN108513546A (zh) 2015-10-28 2018-09-07 克里斯珀医疗股份公司 用于治疗杜氏肌营养不良症的材料和方法
BR112018008971A2 (pt) 2015-11-06 2018-11-27 Crispr Therapeutics Ag materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
HK1257955A1 (zh) 2015-11-16 2019-11-01 The Research Institute At Nationwide Children's Hospital 用於治疗肌联蛋白类肌病和其他肌联蛋白病变的材料和方法
WO2017093804A2 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
EP3394260B1 (de) 2015-12-23 2021-02-17 CRISPR Therapeutics AG Materialien und verfahren zur behandlung von amyothrophischer lateraler sklerose und/oder frontotemporaler demenz
WO2017120589A1 (en) 2016-01-08 2017-07-13 Washington University Compositions comprising chemerin and methods of use thereof
WO2017134529A1 (en) 2016-02-02 2017-08-10 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
WO2017141109A1 (en) 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
WO2017147509A1 (en) 2016-02-25 2017-08-31 Marco Colonna Compositions comprising trem2 and methods of use thereof
AU2017222653B2 (en) 2016-02-26 2023-06-01 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inducing DUX4 exon skipping
US11083799B2 (en) 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
AU2017241534A1 (en) 2016-03-28 2018-10-04 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability
US11345913B2 (en) 2016-04-02 2022-05-31 Research Institute At Nationwide Children's Hospital Modified U6 promoter system for tissue specific expression
CA3021057A1 (en) 2016-04-15 2017-10-19 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
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
MX2018012729A (es) 2016-04-18 2019-07-04 Crispr Therapeutics Ag Materiales y metodos para el tratamiento de hemoglobinopatias.
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
AU2017290614C1 (en) 2016-06-29 2024-01-18 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
AU2017292169B2 (en) 2016-07-06 2021-12-23 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
EP3481857A1 (de) 2016-07-06 2019-05-15 Crispr Therapeutics AG Materialien und verfahren zur behandlung von schmerbedingten erkrankungen
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
AU2017362969A1 (en) 2016-11-15 2019-06-20 Regents Of The University Of Minnesota Method for improving neurological function in MPSI and MPSII and other neurological disorders
EP3541429A4 (de) 2016-11-17 2020-08-12 Nationwide Children's Hospital, Inc. Intrathekale verabreichung von rekombinantem adeno-assoziiertem virus zur codierung von methyl-cpg-bindendem protein 2
IL319407A (en) 2016-11-23 2025-05-01 Gritstone Bio Inc Administration of neoantigens in the virus
JP7206214B2 (ja) 2016-12-13 2023-01-17 シアトル チルドレンズ ホスピタル （ディービーエイ シアトル チルドレンズ リサーチ インスティテュート） インビトロ及びインビボで操作された細胞において発現された化学誘導シグナル伝達複合体の外因性薬物活性化の方法
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
EP3583205A1 (de) 2017-02-17 2019-12-25 Lonza Ltd Säugetierzellen zur herstellung von adeno-assoziierten viren
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
US20200040061A1 (en) 2017-02-22 2020-02-06 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
JP2020508056A (ja) 2017-02-22 2020-03-19 クリスパー・セラピューティクス・アクチェンゲゼルシャフトＣＲＩＳＰＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＡＧ 遺伝子編集のための組成物および方法
US11407997B2 (en) 2017-02-22 2022-08-09 Crispr Therapeutics Ag Materials and methods for treatment of primary hyperoxaluria type 1 (PH1) and other alanine-glyoxylate aminotransferase (AGXT) gene related conditions or disorders
US11559588B2 (en) 2017-02-22 2023-01-24 Crispr Therapeutics Ag Materials and methods for treatment of Spinocerebellar Ataxia Type 1 (SCA1) and other Spinocerebellar Ataxia Type 1 Protein (ATXN1) gene related conditions or disorders
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
IL311713B2 (en) 2017-03-17 2025-10-01 Res Inst Nationwide Childrens Hospital Muscle-specific delivery of microdystrophin using an adeno-associated virus vector for the treatment of muscular dystrophy
JP2020518648A (ja) 2017-05-08 2020-06-25 グリットストーン オンコロジー インコーポレイテッド アルファウイルス新生抗原ベクター
JP7356354B2 (ja) 2017-05-12 2023-10-04 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 細胞の操作のための材料及び方法並びに免疫腫瘍学におけるその使用
CN111065741A (zh) 2017-07-08 2020-04-24 吉尼松公司 脊髓性肌萎缩症的治疗
JP7105866B2 (ja) 2017-07-25 2022-07-25 オックスフォード ジェネティクス リミテッド アデノウイルスベクター
US20200248179A1 (en) 2017-10-02 2020-08-06 Research Institute At Nationwide Children's Hospital MiRNA Detargeting System for Tissue Specific Interference
MA50833A (fr) 2017-10-17 2020-08-26 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique pour l'hémophilie a
JP7213238B2 (ja) 2017-10-18 2023-01-26 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達
CN119193705A (zh) 2017-10-20 2024-12-27 全国儿童医院研究所 Nt-3基因疗法的方法和材料
WO2019081982A1 (en) 2017-10-26 2019-05-02 Crispr Therapeutics Ag SUBSTANCES AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
MX2020004830A (es) 2017-11-08 2020-11-11 Avexis Inc Medios y metodo para preparar vectores virales y usos de los mismos.
EP3707155A2 (de) 2017-11-09 2020-09-16 Vertex Pharmaceuticals Incorporated Crispr/cas-systeme für die behandlung von dmd
US10662425B2 (en) 2017-11-21 2020-05-26 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant retinitis pigmentosa
CN111801417B (zh) 2017-12-14 2024-10-29 克里斯珀医疗股份公司 新的rna-可编程的内切核酸酶系统及其在基因组编辑和其他应用中的用途
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
EP3728595A1 (de) 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materialien und verfahren zur behandlung des usher-syndroms des typs 2a und/oder von nichtsyndromaler autosomaler rezessiver retinitis pigmentosa (arrp)
BR112020013679A2 (pt) 2018-01-04 2020-12-01 Iconic Therapeutics, Inc. anticorpos de fator antitecidual, conjugados anticorpo-fármaco e métodos relacionados
CA3088180A1 (en) 2018-01-12 2019-07-18 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting transferrin
KR102824021B1 (ko) 2018-01-31 2025-06-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 2c형 지대근이영양증에 대한 유전자 치료
WO2019150203A1 (en) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3749767A1 (de) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materialien und verfahren zur behandlung von hämoglobinopathien
WO2019161310A1 (en) 2018-02-16 2019-08-22 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing by targeting fibrinogen-alpha
EP3768834B1 (de) 2018-03-19 2025-08-13 CRISPR Therapeutics AG Neuartige rna-programmierbare endonukleasesysteme und deren verwendung in der genombearbeitung und in anderen anwendungen
WO2019204668A1 (en) 2018-04-18 2019-10-24 Casebia Therapeutics Limited Liability Partnership Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
CA3091490A1 (en) 2018-04-27 2019-10-31 Seattle Children's Hospital (dba Seattle Children's Research Institute) Rapamycin resistant cells
CN120718958A (zh) 2018-06-08 2025-09-30 诺华股份有限公司 用于测量药物产品效力的基于细胞的测定
GB201809588D0 (en) 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
US12491265B2 (en) 2018-06-18 2025-12-09 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
US20240254181A1 (en) 2018-06-18 2024-08-01 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
CA3110466A1 (en) 2018-08-22 2020-02-27 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene
EP3844284A1 (de) 2018-08-29 2021-07-07 Research Institute at Nationwide Children's Hospital Produkte und verfahren zur hemmung der expression von mutierten gars-proteinen
WO2020051766A1 (zh) 2018-09-11 2020-03-19 上海市公共卫生临床中心 一种广谱抗流感疫苗免疫原及其应用
BR112021007403A2 (pt) 2018-10-17 2021-12-07 Bayer Healthcare Llc Composições e métodos para entrega de transgenes
CN113710799B (zh) 2018-11-28 2024-11-12 克里斯珀医疗股份公司 用于在LNP中使用的编码CAS9的优化mRNA
EP3886919A1 (de) 2018-11-30 2021-10-06 Novartis AG Aav-virusvektoren und verwendungen davon
JP2022516010A (ja) 2018-12-21 2022-02-24 ジェネトン 遺伝子療法ベクターのための発現カセット
KR20210110345A (ko) 2018-12-31 2021-09-07 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 RNA 표적화 CRISPR-Cas13b를 사용한 DUX4 RNA 침묵화
KR20210124300A (ko) 2019-02-04 2021-10-14 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Cln3 폴리뉴클레오티드의 아데노-관련 바이러스 전달
MX2021009404A (es) 2019-02-04 2021-11-12 Res Inst Nationwide Childrens Hospital Administracion del virus adenoasociado de polinucleotido de cln6.
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc Gene editing for hemophilia A with improved Factor VIII expression
EP3931334A1 (de) 2019-02-25 2022-01-05 Novartis AG Zusammensetzungen und verfahren zur behandlung von bietti-kristall-dystrophie
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
MX2021010356A (es) 2019-02-26 2022-03-04 Res Inst Nationwide Childrens Hospital SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR.
WO2020186059A2 (en) 2019-03-12 2020-09-17 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
CA3135609A1 (en) 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
CA3137078A1 (en) 2019-04-15 2020-10-22 University Of Iowa Research Foundation Methods and compositions for transgene expression
JP7731801B2 (ja) 2019-04-15 2025-09-01 ユニヴァーシティ オブ アイオワ リサーチ ファウンデーション 嚢胞性線維症の治療のための組換えアデノ随伴ウイルス
WO2020214737A1 (en) 2019-04-15 2020-10-22 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
US20220233655A1 (en) 2019-05-17 2022-07-28 Research Institute At Nationwide Children's Hospital Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme
AU2020282369A1 (en) 2019-05-30 2022-01-20 Seattle Project Corp. Modified adenoviruses
US12173290B2 (en) 2019-06-28 2024-12-24 Crispr Therapeutics Ag Materials and methods for controlling gene editing
US20220307053A1 (en) 2019-07-25 2022-09-29 Novartis Ag Regulatable expression systems
CN120400170A (zh) 2019-08-21 2025-08-01 全国儿童医院研究所 Α-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
WO2021072115A1 (en) 2019-10-08 2021-04-15 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
EP4045662A1 (de) 2019-10-18 2022-08-24 Research Institute at Nationwide Children's Hospital Materialien und verfahren zur behandlung von krankheiten, die mit mutationen im irf2bpl-gen einhergehen
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
AU2020408225A1 (en) 2019-12-20 2022-07-14 Research Institute At Nationwide Children's Hospital Optimized gene therapy for targeting muscle in muscle diseases
JP2023513932A (ja) 2020-02-18 2023-04-04 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 Ｘ連鎖障害の治療におけるｍｉＲＮＡのＡＡＶ媒介標的化
CN115379863A (zh) 2020-04-14 2022-11-22 吉尼松公司 用于治疗酸性神经酰胺酶缺乏症的载体
KR20230035043A (ko) 2020-06-15 2023-03-10 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 근이영양증을 위한 아데노-연관 바이러스 벡터 전달
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
CN116323953A (zh) 2020-07-30 2023-06-23 沙普治疗股份有限公司 诱导产生raav病毒粒子的稳定细胞系
WO2022032196A2 (en) 2020-08-06 2022-02-10 Gritstone Bio, Inc. Multiepitope vaccine cassettes
WO2022047201A1 (en) 2020-08-27 2022-03-03 University Of Iowa Research Foundation Gene knock-out for treatment of glaucoma
TWI887479B (zh) 2020-09-08 2025-06-21 美商薩羅塔治療公司 表現γ-肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療
CA3195233A1 (en) 2020-09-15 2022-03-24 Research Institute At Nationwide Children's Hospital Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
CA3194122A1 (en) 2020-09-28 2022-03-31 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
US20240026356A1 (en) 2020-11-30 2024-01-25 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
AU2022212922A1 (en) 2021-01-27 2023-08-17 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
EP4288539A1 (de) 2021-02-03 2023-12-13 Research Institute at Nationwide Children's Hospital Zusammensetzungen und verfahren zur behandlung von erkrankungen im zusammenhang mit dux4-überexpression
WO2022170038A1 (en) 2021-02-05 2022-08-11 Amicus Therapeutics, Inc. Adeno-associated virus delivery of cln3 polynucleotide
US20240316217A1 (en) 2021-02-05 2024-09-26 Regents Of The University Of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses
WO2022187571A1 (en) 2021-03-04 2022-09-09 Research Institute At Nationwide Children's Hospital Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
EP4305157A1 (de) 2021-03-09 2024-01-17 Huidagene Therapeutics (Singapore) Pte. Ltd. Manipuliertes crispr/cas13-system und verwendungen davon
EP4323010A1 (de) 2021-04-13 2024-02-21 Research Institute at Nationwide Children's Hospital Rekombinantes adeno-assoziiertes virus, das für methyl-cpg-bindendes protein 2 codiert, zur behandlung des pitt-hopkins-syndroms durch intrathekale verabreichung
CA3216495A1 (en) 2021-04-15 2022-10-20 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis
US12275941B2 (en) 2021-04-15 2025-04-15 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of dynamin-1 variants
EP4326752A1 (de) 2021-04-23 2024-02-28 Research Institute at Nationwide Children's Hospital Produkte und verfahren zur behandlung von muskeldystrophie
WO2022234295A1 (en) 2021-05-07 2022-11-10 Ucl Business Ltd Abca4 genome editing
EP4341413A1 (de) 2021-05-17 2024-03-27 Sarepta Therapeutics, Inc. Herstellung rekombinanter aav-vektoren zur behandlung von muskeldystrophie
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
BR112023023768A2 (pt) 2021-06-11 2024-02-27 Bayer Ag Sistemas de endonucleases programáveis por rna tipo v
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
EP4402265A1 (de) 2021-09-16 2024-07-24 Novartis AG Neue transkriptionsfaktoren
CA3234702A1 (en) 2021-10-07 2023-04-13 Research Institute At Nationwide Children's Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
WO2023060233A1 (en) 2021-10-08 2023-04-13 Amicus Therapeutics, Inc. Biomarkers for lysosomal storage diseases
EP4219726A1 (de) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Selbstkomplementärer adeno-assoziierter virusvektor und dessen verwendung bei der behandlung von muskeldystrophie
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 (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
EP4198048A1 (de) 2021-12-16 2023-06-21 Genethon Calpain-3-gentransfersteigerung unter verwendung von modifizierten itr-sequenzen
CA3242989A1 (en) 2021-12-21 2023-06-29 Research Institute At Nationwide Children's Hospital MATERIALS AND METHODS FOR THE TREATMENT OF MUSCULAR BELLY DYSTROPHY
EP4453191A1 (de) 2021-12-23 2024-10-30 Bayer Aktiengesellschaft Neuartige programmierbare endonukleasesysteme für kleine v-rna
EP4486890A1 (de) 2022-03-01 2025-01-08 CRISPR Therapeutics AG Verfahren und zusammensetzungen zur behandlung von angiopoietin-like 3 (angptl3)-bedingten erkrankungen
US20250186621A1 (en) 2022-03-03 2025-06-12 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
AU2023250649A1 (en) 2022-04-04 2024-11-14 The Regents Of The University Of California Genetic complementation compositions and methods
EP4508062A1 (de) 2022-04-11 2025-02-19 Tenaya Therapeutics, Inc. Adeno-assoziiertes virus mit manipuliertem kapsid
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
JP2025523399A (ja) 2022-06-08 2025-07-23 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 変異型又は病原性ｋｃｎｑ３発現に関連する疾患又は状態を治療するための生成物及び方法
IL316824A (en) 2022-06-10 2025-01-01 Bayer Ag Novel small, programmable endonuclease systems for type V RNA
JP2025524569A (ja) 2022-07-06 2025-07-30 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 Ｃｌｎ１ポリヌクレオチドのアデノ随伴ウイルス送達
WO2024035782A1 (en) 2022-08-10 2024-02-15 Aav Gene Therapeutics, Inc. Aav-mediated intramuscular delivery of insulin
JP2025532137A (ja) 2022-09-23 2025-09-29 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための組換えａａｖベクター
AU2023360998A1 (en) 2022-10-11 2025-05-22 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)
AU2023364350A1 (en) 2022-10-19 2025-06-05 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2024092171A1 (en) 2022-10-26 2024-05-02 University Of Iowa Research Foundation Method to deliver large genes using virus and a dna recombination system
EP4608856A1 (de) 2022-10-27 2025-09-03 CARGO Therapeutics, Inc. Zusammensetzungen und verfahren für verbesserte immuntherapien
AU2023398007A1 (en) 2022-12-13 2025-06-19 Bayer Aktiengesellschaft Engineered type v rna programmable endonucleases and their uses
WO2024151982A1 (en) 2023-01-13 2024-07-18 Amicus Therapeutics, Inc. Gene therapy constructs for the treatment of pompe disease
CN120615127A (zh) 2023-02-01 2025-09-09 萨勒普塔医疗公司 Raav产生方法
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
WO2024229259A1 (en) 2023-05-02 2024-11-07 Research Institute At Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases
AU2024266487A1 (en) 2023-05-02 2025-11-27 Research Institute At Nationwide Children's Hospital Treatment of multiple sclerosis using nt-3 gene therapy
AU2024265701A1 (en) 2023-05-02 2025-12-04 Research Institute At Nationwide Children's Hospital A modular system to convert therapeutic microrna expression cassettes from polymerase iii-based to polymerase ii-based promoters
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2024254319A1 (en) 2023-06-07 2024-12-12 Research Institute At Nationwide Children's Hospital Gene therapy for lysosomal acid lipase deficiency (lal-d)
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WO2025240690A2 (en) 2024-05-15 2025-11-20 Research Institute At Nationwide Children's Hospital Products and methods for treating diseases or conditions associated with progerin expression from an aberrant lmna gene
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav

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

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DE69433922T2 (de) 2005-07-28
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WO1995013392A1 (en) 1995-05-18
EP0728214B1 (de) 2004-07-28
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