JP2023081369A5 - - Google Patents

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Publication number
JP2023081369A5
JP2023081369A5 JP2022191138A JP2022191138A JP2023081369A5 JP 2023081369 A5 JP2023081369 A5 JP 2023081369A5 JP 2022191138 A JP2022191138 A JP 2022191138A JP 2022191138 A JP2022191138 A JP 2022191138A JP 2023081369 A5 JP2023081369 A5 JP 2023081369A5
Authority
JP
Japan
Prior art keywords
recombinant aav
cell
sequence
complementary
subject
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022191138A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023081369A (ja
Filing date
Publication date
Application filed filed Critical
Publication of JP2023081369A publication Critical patent/JP2023081369A/ja
Publication of JP2023081369A5 publication Critical patent/JP2023081369A5/ja
Pending legal-status Critical Current

Links

JP2022191138A 2021-11-30 2022-11-30 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 Pending JP2023081369A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163284418P 2021-11-30 2021-11-30
US63/284,418 2021-11-30

Publications (2)

Publication Number Publication Date
JP2023081369A JP2023081369A (ja) 2023-06-09
JP2023081369A5 true JP2023081369A5 (cg-RX-API-DMAC7.html) 2025-10-27

Family

ID=85278650

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022191138A Pending JP2023081369A (ja) 2021-11-30 2022-11-30 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

Country Status (3)

Country Link
US (1) US20230279431A1 (cg-RX-API-DMAC7.html)
EP (1) EP4186919A1 (cg-RX-API-DMAC7.html)
JP (1) JP2023081369A (cg-RX-API-DMAC7.html)

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
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
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP2001500497A (ja) 1996-09-06 2001-01-16 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に指図される遺伝子治療の方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
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
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP2736539B1 (en) 2011-07-25 2017-08-23 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
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
AR114350A1 (es) * 2018-01-31 2020-08-26 Res Institute At Nationwide Children’S Hospital Terapia genética para la distrofia muscular de cinturas del tipo 2c

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