JP2024537167A5 - - Google Patents

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Publication number
JP2024537167A5
JP2024537167A5 JP2024520851A JP2024520851A JP2024537167A5 JP 2024537167 A5 JP2024537167 A5 JP 2024537167A5 JP 2024520851 A JP2024520851 A JP 2024520851A JP 2024520851 A JP2024520851 A JP 2024520851A JP 2024537167 A5 JP2024537167 A5 JP 2024537167A5
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JP
Japan
Application number
JP2024520851A
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Japanese (ja)
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JP2024537167A (ja
JPWO2023060215A5 (https=
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Publication date
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Priority claimed from PCT/US2022/077726 external-priority patent/WO2023060215A1/en
Publication of JP2024537167A publication Critical patent/JP2024537167A/ja
Publication of JP2024537167A5 publication Critical patent/JP2024537167A5/ja
Publication of JPWO2023060215A5 publication Critical patent/JPWO2023060215A5/ja
Pending legal-status Critical Current

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JP2024520851A 2021-10-07 2022-10-07 ミエリンタンパク質ゼロサイレンシング及びcmt1b疾患を治療するための生成物及び方法 Pending JP2024537167A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163253357P 2021-10-07 2021-10-07
US63/253,357 2021-10-07
PCT/US2022/077726 WO2023060215A1 (en) 2021-10-07 2022-10-07 Products and methods for myelin protein zero silencing and treating cmt1b disease

Publications (3)

Publication Number Publication Date
JP2024537167A JP2024537167A (ja) 2024-10-10
JP2024537167A5 true JP2024537167A5 (https=) 2025-10-14
JPWO2023060215A5 JPWO2023060215A5 (https=) 2025-10-14

Family

ID=83995553

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024520851A Pending JP2024537167A (ja) 2021-10-07 2022-10-07 ミエリンタンパク質ゼロサイレンシング及びcmt1b疾患を治療するための生成物及び方法

Country Status (6)

Country Link
US (1) US20240401046A1 (https=)
EP (1) EP4413131A1 (https=)
JP (1) JP2024537167A (https=)
AU (1) AU2022360382A1 (https=)
CA (1) CA3234702A1 (https=)
WO (1) WO2023060215A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118291543A (zh) * 2024-02-29 2024-07-05 神拓生物技术(杭州)有限公司 一种二型神经纤维瘤基因治疗的慢病毒载体及其应用

Family Cites Families (25)

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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
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
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
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
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
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
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
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
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
KR102066663B1 (ko) * 2017-02-21 2020-01-15 사회복지법인 삼성생명공익재단 유전성 말초 신경질환 진단용 마커 및 그의 용도
WO2020028533A1 (en) * 2018-08-01 2020-02-06 Yale University Compositions and methods for identification of membrane targets for enhancement of t cell activity against cancer
JP2021534794A (ja) * 2018-08-29 2021-12-16 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 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
GB201907882D0 (en) 2019-06-03 2019-07-17 Cyprus Foundation For Muscular Dystrophy Res Methods

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