JP2021527418A5 - - Google Patents

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Publication number
JP2021527418A5
JP2021527418A5 JP2020570457A JP2020570457A JP2021527418A5 JP 2021527418 A5 JP2021527418 A5 JP 2021527418A5 JP 2020570457 A JP2020570457 A JP 2020570457A JP 2020570457 A JP2020570457 A JP 2020570457A JP 2021527418 A5 JP2021527418 A5 JP 2021527418A5
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JP
Japan
Prior art keywords
seq
domain
polynucleotide
sequence
raav
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JP2020570457A
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English (en)
Japanese (ja)
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JP2021527418A (ja
JP7534961B2 (ja
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Priority claimed from PCT/US2019/037769 external-priority patent/WO2019246125A1/en
Publication of JP2021527418A publication Critical patent/JP2021527418A/ja
Publication of JP2021527418A5 publication Critical patent/JP2021527418A5/ja
Priority to JP2024127744A priority Critical patent/JP7879196B2/ja
Application granted granted Critical
Publication of JP7534961B2 publication Critical patent/JP7534961B2/ja
Active legal-status Critical Current
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JP2020570457A 2018-06-18 2019-06-18 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 Active JP7534961B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024127744A JP7879196B2 (ja) 2018-06-18 2024-08-02 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862686522P 2018-06-18 2018-06-18
US62/686,522 2018-06-18
PCT/US2019/037769 WO2019246125A1 (en) 2018-06-18 2019-06-18 Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024127744A Division JP7879196B2 (ja) 2018-06-18 2024-08-02 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法

Publications (3)

Publication Number Publication Date
JP2021527418A JP2021527418A (ja) 2021-10-14
JP2021527418A5 true JP2021527418A5 (enExample) 2022-06-28
JP7534961B2 JP7534961B2 (ja) 2024-08-15

Family

ID=67263074

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020570457A Active JP7534961B2 (ja) 2018-06-18 2019-06-18 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法
JP2024127744A Active JP7879196B2 (ja) 2018-06-18 2024-08-02 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024127744A Active JP7879196B2 (ja) 2018-06-18 2024-08-02 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法

Country Status (6)

Country Link
US (1) US12668617B2 (enExample)
EP (1) EP3807309A1 (enExample)
JP (2) JP7534961B2 (enExample)
AU (2) AU2019288291B2 (enExample)
CA (1) CA3104471A1 (enExample)
WO (1) WO2019246125A1 (enExample)

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH05506435A (ja) * 1990-01-30 1993-09-22 ラ ホヤ キャンサー リサーチ ファウンデーション メロシン、それをコードする核酸、フラグメントおよびその使用
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
US20110166081A1 (en) 2009-12-03 2011-07-07 University Of Iowa Research Foundation Alpha-dystroglycan as a Protein Therapeutic
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
KR102799163B1 (ko) 2015-09-17 2025-04-21 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Galgt2 유전자 치료 방법 및 물질
WO2017181011A1 (en) 2016-04-15 2017-10-19 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy
WO2018151841A1 (en) * 2017-02-17 2018-08-23 Sanofi Multispecific binding molecules having specificity to dystroglycan and laminin-2

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