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
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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 (enExample) 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 (enExample)
EP (1) EP4186919A1 (enExample)
JP (1) JP2023081369A (enExample)

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5187063A (en) * 1990-05-23 1993-02-16 University Of Iowa Research Foundation Measuring non-dystrophin proteins and diagnosing muscular dystrophy
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
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
WO1996017947A1 (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
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびaavの精製
JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたaavベクター
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
EP1696036B1 (en) 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
CA2995542A1 (en) 1997-09-05 1999-03-11 Genzyme 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
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
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたaav5キャプシドおよびaav5ベクターを含む組換えaavベクター
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
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
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
MX2020004035A (es) * 2017-10-20 2020-11-09 Res Inst Nationwide Childrens Hospital Métodos y materiales para terapia génica con nt-3.
AU2019216257B2 (en) * 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
EP3953484A4 (en) * 2019-04-12 2023-04-12 University of Massachusetts Aav-mediated gene therapy for maple syrup urine disease (msud)
CA3150964A1 (en) * 2019-08-21 2021-02-25 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
US20230040544A1 (en) * 2019-12-16 2023-02-09 Research Institute At Nationwide Children's Hospital Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc)

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