JP2021534794A5 - - Google Patents

Info

Publication number
JP2021534794A5
JP2021534794A5 JP2021510957A JP2021510957A JP2021534794A5 JP 2021534794 A5 JP2021534794 A5 JP 2021534794A5 JP 2021510957 A JP2021510957 A JP 2021510957A JP 2021510957 A JP2021510957 A JP 2021510957A JP 2021534794 A5 JP2021534794 A5 JP 2021534794A5
Authority
JP
Japan
Prior art keywords
aav
composition according
viral vector
gars
nucleic acid
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
JP2021510957A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021534794A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/048832 external-priority patent/WO2020047268A1/en
Publication of JP2021534794A publication Critical patent/JP2021534794A/ja
Publication of JP2021534794A5 publication Critical patent/JP2021534794A5/ja
Priority to JP2024107253A priority Critical patent/JP2024133570A/ja
Pending legal-status Critical Current

Links

JP2021510957A 2018-08-29 2019-08-29 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法 Pending JP2021534794A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024107253A JP2024133570A (ja) 2018-08-29 2024-07-03 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862724604P 2018-08-29 2018-08-29
US62/724,604 2018-08-29
PCT/US2019/048832 WO2020047268A1 (en) 2018-08-29 2019-08-29 Products and methods for inhibition of expression of mutant gars protein

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024107253A Division JP2024133570A (ja) 2018-08-29 2024-07-03 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法

Publications (2)

Publication Number Publication Date
JP2021534794A JP2021534794A (ja) 2021-12-16
JP2021534794A5 true JP2021534794A5 (https=) 2022-08-31

Family

ID=67953864

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021510957A Pending JP2021534794A (ja) 2018-08-29 2019-08-29 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法
JP2024107253A Pending JP2024133570A (ja) 2018-08-29 2024-07-03 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024107253A Pending JP2024133570A (ja) 2018-08-29 2024-07-03 変異型ｇａｒｓタンパク質の発現を阻害するための生成物および方法

Country Status (6)

Country Link
US (1) US20210324417A1 (https=)
EP (1) EP3844284A1 (https=)
JP (2) JP2021534794A (https=)
AU (1) AU2019328270B2 (https=)
CA (1) CA3110665A1 (https=)
WO (1) WO2020047268A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL299762A (en) * 2020-07-13 2023-03-01 Univ Pennsylvania Useful preparations for the treatment of Charcot-Marietot disease
US20240318172A1 (en) * 2020-12-01 2024-09-26 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of peripheral myelin protein-22
EP4413131A1 (en) * 2021-10-07 2024-08-14 Research Institute at Nationwide Children's Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
IT202200009395A1 (it) * 2022-05-09 2022-08-09 Giam Pharma Int Sarl Attività antitumorale del meso-(p-acetamidophenyl)-calix[4]pirrolo mediante l’Inibizione dell’attività enzimatica della proteina Glicil-tRNA Sintasi 1 (GARS1)

Family Cites Families (26)

* 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
EP2152874A2 (en) * 2007-04-26 2010-02-17 University of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
DK2164967T3 (en) * 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
AU2011289831C1 (en) * 2010-07-12 2017-06-15 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glycyl-tRNA synthetases
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
EP3426775A1 (en) * 2016-03-10 2019-01-16 Novartis AG Chemically modified messenger rna's

Similar Documents

Publication Publication Date Title
US20220331409A1 (en) Factor ix gene therapy
JP2020019772A5 (https=)
JP2021534794A5 (https=)
JP2023553005A5 (https=)
JP2022518504A5 (https=)
JP2020532981A5 (https=)
JP2020522269A5 (https=)
WO2016077687A4 (en) Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP2022500066A5 (https=)
RU2018140499A (ru) Способы и композиции для лечения хореи гентингтона
JP2017510298A5 (https=)
JP2021512649A5 (https=)
JP2021534755A5 (https=)
JP2017535266A5 (https=)
JP2021500049A5 (https=)
EP2524037A1 (en) Restrictive inverted terminal repeats for viral vectors
JP7558535B2 (ja) アルデヒドデヒドロゲナーゼ欠損症の治療のための遺伝子治療
JP2017529395A5 (https=)
JP2023537980A5 (https=)
JP2024508696A5 (https=)
WO2020142714A1 (en) Aav expression cassette and aav vectors comprising the same
CN116096904A (zh) 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途
JP2021525531A5 (https=)
JP2024543211A5 (https=)
JP2021526834A (ja) 組み換えａａｖベクター及びその使用方法