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
Prior art date
Application number
JP2020570457A
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English (en)
Japanese (ja)
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JP2021527418A (ja
JP7534961B2 (ja
JPWO2019246125A5 (https=
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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 JPWO2019246125A5 publication Critical patent/JPWO2019246125A5/ja
Publication of JP2021527418A5 publication Critical patent/JP2021527418A5/ja
Priority to JP2024127744A priority Critical patent/JP2024156872A/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 JP2024156872A (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 JP2024156872A (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 (4)

Publication Number Publication Date
JP2021527418A JP2021527418A (ja) 2021-10-14
JPWO2019246125A5 JPWO2019246125A5 (https=) 2022-06-28
JP2021527418A5 true JP2021527418A5 (https=) 2022-06-28
JP7534961B2 JP7534961B2 (ja) 2024-08-15

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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 Pending JP2024156872A (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

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JP2024127744A Pending JP2024156872A (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) US20240254181A1 (https=)
EP (1) EP3807309A1 (https=)
JP (2) JP7534961B2 (https=)
AU (2) AU2019288291B2 (https=)
CA (1) CA3104471A1 (https=)
WO (1) WO2019246125A1 (https=)

Family Cites Families (26)

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ATE192458T1 (de) * 1990-01-30 2000-05-15 Jolla Cancer Res Found Merosin, dafür kodierende nukleinsäuren, fragmente und deren verwendung
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
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
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
AU707866B2 (en) 1994-12-06 1999-07-22 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
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved 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
EP0932418B1 (en) 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法
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
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
AU2002248297A1 (en) 2001-01-05 2002-07-16 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
JP6202701B2 (ja) 2014-03-21 2017-09-27 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プログラム
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
EA201890543A1 (ru) 2015-09-17 2018-08-31 Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл Способы и материалы для генной терапии galgt2
AU2017250791B2 (en) 2016-04-15 2022-11-24 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of micro-dystrophin 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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