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
Priority to JP2026072273A priority patent/JP2026120817A/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 (2)

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
JP2026072273A JP2026120817A (ja) 2018-08-29 2026-04-24 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 (enExample) 2022-08-31

Family

ID=67953864

Family Applications (3)

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JP2024107253A Withdrawn JP2024133570A (ja) 2018-08-29 2024-07-03 変異型garsタンパク質の発現を阻害するための生成物および方法
JP2026072273A Pending JP2026120817A (ja) 2018-08-29 2026-04-24 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 (2)

Application Number Title Priority Date Filing Date
JP2024107253A Withdrawn 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
JP2026072273A Pending JP2026120817A (ja) 2018-08-29 2026-04-24 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 (enExample)
EP (1) EP3844284A1 (enExample)
JP (3) JP2021534794A (enExample)
AU (1) AU2019328270B2 (enExample)
CA (1) CA3110665A1 (enExample)
WO (1) WO2020047268A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022015715A1 (en) * 2020-07-13 2022-01-20 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
WO2022119826A1 (en) * 2020-12-01 2022-06-09 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of peripheral myelin protein-22
US20240401046A1 (en) * 2021-10-07 2024-12-05 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)

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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
WO2008134646A2 (en) * 2007-04-26 2008-11-06 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
JP6116479B2 (ja) * 2010-07-12 2017-04-19 エータイアー ファーマ, インコーポレイテッド グリシルtRNA合成酵素のタンパク質フラグメントに関連した治療用、診断用および抗体組成物の革新的発見
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
WO2017153936A1 (en) * 2016-03-10 2017-09-14 Novartis Ag Chemically modified messenger rna's

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