JPWO2020245169A5 - - Google Patents

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JPWO2020245169A5
JPWO2020245169A5 JP2021571874A JP2021571874A JPWO2020245169A5 JP WO2020245169 A5 JPWO2020245169 A5 JP WO2020245169A5 JP 2021571874 A JP2021571874 A JP 2021571874A JP 2021571874 A JP2021571874 A JP 2021571874A JP WO2020245169 A5 JPWO2020245169 A5 JP WO2020245169A5
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JP
Japan
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vector
seq
promoter
aav9
mpz
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JP2021571874A
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English (en)
Japanese (ja)
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JP7692369B2 (ja
JP2022535412A5 (https=
JP2022535412A (ja
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Priority claimed from GBGB1907882.3A external-priority patent/GB201907882D0/en
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JP2021571874A 2019-06-03 2020-06-03 シャルコー・マリー・トゥース病のようなシュワン細胞関連疾患を治療するためのミエリンタンパク質ゼロのプロモーターを含むaavベクター及びその使用 Active JP7692369B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1907882.3 2019-06-03
GBGB1907882.3A GB201907882D0 (en) 2019-06-03 2019-06-03 Methods
PCT/EP2020/065312 WO2020245169A1 (en) 2019-06-03 2020-06-03 Aav vectors with myelin protein zero promoter and uses thereof for treating schwann cell-associated diseases like charcot-marie-tooth disease

Publications (4)

Publication Number Publication Date
JP2022535412A JP2022535412A (ja) 2022-08-08
JPWO2020245169A5 true JPWO2020245169A5 (https=) 2023-06-02
JP2022535412A5 JP2022535412A5 (https=) 2023-06-02
JP7692369B2 JP7692369B2 (ja) 2025-06-13

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ID=67385825

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021571874A Active JP7692369B2 (ja) 2019-06-03 2020-06-03 シャルコー・マリー・トゥース病のようなシュワン細胞関連疾患を治療するためのミエリンタンパク質ゼロのプロモーターを含むaavベクター及びその使用

Country Status (16)

Country Link
US (1) US12582725B2 (https=)
EP (1) EP3976800A1 (https=)
JP (1) JP7692369B2 (https=)
KR (1) KR20220016485A (https=)
CN (1) CN114026242A (https=)
AU (1) AU2020288605A1 (https=)
BR (1) BR112021024285A2 (https=)
CA (1) CA3142211A1 (https=)
CL (1) CL2021003201A1 (https=)
CO (1) CO2021016487A2 (https=)
EA (1) EA202290001A1 (https=)
GB (1) GB201907882D0 (https=)
IL (1) IL288462A (https=)
MX (1) MX2021014789A (https=)
SG (1) SG11202112889VA (https=)
WO (1) WO2020245169A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240076665A1 (en) * 2020-12-22 2024-03-07 Wisconsin Alumni Research Foundation Regulatory elements for schwann cell-specific gene expression
CA3234702A1 (en) 2021-10-07 2023-04-13 Research Institute At Nationwide Children's Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
JP2025502269A (ja) * 2022-01-13 2025-01-24 ツールゲン インコーポレイテッド シュワン細胞特異的プロモーター
GB202303495D0 (en) * 2023-03-09 2023-04-26 Ucl Business Ltd Therapy for charcot-marie-tooth disease
WO2025105734A1 (ko) * 2023-11-15 2025-05-22 주식회사 카인사이언스 탈수초 질환 치료용 펩타이드 및 이의 용도
WO2025174135A1 (ko) * 2024-02-16 2025-08-21 주식회사 엣진 샤르코-마리-투스 질환 치료를 위한 징크 핑거 단백질 및 이를 발현시키는 방법
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
KR20260001543A (ko) * 2024-06-25 2026-01-06 연세대학교 산학협력단 바이러스 벡터를 포함한 난청 치료용 조성물

Family Cites Families (24)

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PL210751B1 (pl) 2006-09-12 2012-02-29 Anna Jurewicz Izolowany fragment białka Nogo-A, jego zastosowanie oraz sposób diagnozowania oraz leczenia stwardnienia rozsianego i polineuropatii zapalno-demielinizacyjnej
US20090018031A1 (en) 2006-12-07 2009-01-15 Switchgear Genomics Transcriptional regulatory elements of biological pathways tools, and methods
US20090215178A1 (en) 2008-02-22 2009-08-27 Zequn Tang Methods to enhance the stability and homogeneity of transgene expression in clonal cell lines
CA2730785A1 (en) 2008-07-16 2010-01-21 The Board Of Trustees Of The Leland Stanford Junior University Modulation of gm98 (mrf) in remyelination
AU2010289419A1 (en) 2009-09-02 2012-03-29 The University Of Chicago Methods and systems for inducible ablation of neural cells
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
AU2012258558A1 (en) 2011-05-26 2013-05-02 Biogen Ma Inc. Methods of treating multiple sclerosis and preserving and/or increasing myelin content
DK2820042T3 (da) 2012-02-29 2019-10-28 Baxalta GmbH Igg-stimuleret remyelinisering af perifere nerver
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9644215B2 (en) * 2013-04-12 2017-05-09 The General Hospital Corporation AAV1-caspase gene therapy induced pyroptosis for the treatment of tumors
WO2015140351A1 (en) 2014-03-21 2015-09-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for enhancing myelination
EP2933335A1 (en) * 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
US10675382B2 (en) 2014-09-12 2020-06-09 Kyoto Prefectural Public University Corporation Schwann cells and method for preparing same
EP3286318A2 (en) 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
RU2766200C1 (ru) 2015-10-01 2022-02-09 Хит Байолоджикс, Инк. Композиции и способы для соединения внеклеточных доменов типа i и типа ii в качестве гетерологичных химерных белков
JP7064197B2 (ja) 2016-09-23 2022-05-10 国立大学法人大阪大学 シュワン細胞分化促進剤及び末梢神経再生促進剤
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018088694A2 (ko) 2016-11-14 2018-05-17 주식회사 툴젠 인위적으로 조작된 sc 기능 조절 시스템
WO2018106753A1 (en) * 2016-12-07 2018-06-14 The General Hospital Corporation Methods and compositions relating the treatment of tumors
EP3635121A1 (en) 2017-06-02 2020-04-15 Institut National de la Sante et de la Recherche Medicale (INSERM) Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
AU2018345772B2 (en) * 2017-10-02 2025-04-17 Research Institute At Nationwide Children's Hospital MiRNA detargeting system for tissue specific interference
AU2018351528B2 (en) * 2017-10-20 2025-02-27 Research Institute At Nationwide Children's Hospital Methods and materials for NT-3 gene therapy
IL299762A (en) 2020-07-13 2023-03-01 Univ Pennsylvania Useful preparations for the treatment of Charcot-Marietot disease

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