JPWO2023060215A5 - - Google Patents

Info

Publication number
JPWO2023060215A5
JPWO2023060215A5 JP2024520851A JP2024520851A JPWO2023060215A5 JP WO2023060215 A5 JPWO2023060215 A5 JP WO2023060215A5 JP 2024520851 A JP2024520851 A JP 2024520851A JP 2024520851 A JP2024520851 A JP 2024520851A JP WO2023060215 A5 JPWO2023060215 A5 JP WO2023060215A5
Authority
JP
Japan
Prior art keywords
mpz
aav
pharmaceutical combination
polynucleotide sequence
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
JP2024520851A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024537167A (ja
JP2024537167A5 (https=
Publication date
Application filed filed Critical
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

Links

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 JP2024537167A5 (https=) 2025-10-14
JPWO2023060215A5 true 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)

* 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
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

Similar Documents

Publication Publication Date Title
JP2021512649A5 (https=)
US12037588B2 (en) Compositions and methods comprising engineered short nuclear RNA (snRNA)
JP2021500049A (ja) 神経変性疾患の遺伝子治療
US20250269065A1 (en) B-cell lymphoma 2-associated anthanogene 3 (bag3) gene therapy using aav vector
KR20210096168A (ko) 신경퇴행성 질환을 위한 유전자 요법
JP7785389B2 (ja) 新規なイントロン断片
IL300294A (en) Gene therapy using nucleic acid constructs containing methyl CPG-binding protein 2 (MECP2) promoter sequences
JPWO2019153009A5 (https=)
JP2021534794A5 (https=)
US11753655B2 (en) Compositions and methods for treatment of neurological disorders
JP2025540074A (ja) 繰り返しエレメントの適切なパッケージングのためのアデノ随伴ウイルスベクター
JP2021534766A5 (https=)
JP2025536927A (ja) RNA編集のためのプログラム可能なsnRNAを含む組成物及び方法
AU2022282057B2 (en) Novel dual helper plasmid
US20200283800A1 (en) Gene therapies for neurodegenerative diseases
JPWO2023060215A5 (https=)
CA3219287C (en) Dual helper plasmid
JPWO2022119826A5 (https=)
JPWO2022028472A5 (https=)
JPWO2020047268A5 (https=)
JPWO2021076941A5 (https=)
JPWO2023213831A5 (https=)
JPWO2020210724A5 (https=)
RU2022111695A (ru) Выделенная нуклеиновая кислота, которая кодирует слитый белок на основе FVIII-BDD и гетерологичного сигнального пептида, и ее применение
JP2024504422A (ja) Aavに基づく遺伝子発現の調節