JP2022535412A5 - - Google Patents

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Publication number
JP2022535412A5
JP2022535412A5 JP2021571874A JP2021571874A JP2022535412A5 JP 2022535412 A5 JP2022535412 A5 JP 2022535412A5 JP 2021571874 A JP2021571874 A JP 2021571874A JP 2021571874 A JP2021571874 A JP 2021571874A JP 2022535412 A5 JP2022535412 A5 JP 2022535412A5
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JP
Japan
Prior art keywords
vector
seq
promoter
sequence
aav9
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JP2021571874A
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English (en)
Japanese (ja)
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JP2022535412A (ja
JP7692369B2 (ja
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Priority claimed from GBGB1907882.3A external-priority patent/GB201907882D0/en
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Publication of JP2022535412A publication Critical patent/JP2022535412A/ja
Publication of JP2022535412A5 publication Critical patent/JP2022535412A5/ja
Application granted granted Critical
Publication of JP7692369B2 publication Critical patent/JP7692369B2/ja
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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
GBGB1907882.3A GB201907882D0 (en) 2019-06-03 2019-06-03 Methods
GB1907882.3 2019-06-03
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 (3)

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

Family

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
WO2022140521A1 (en) * 2020-12-22 2022-06-30 Wisconsin Alumni Research Foundation Regulatory elements for schwann cell-specific gene expression
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
WO2023136624A1 (ko) 2022-01-13 2023-07-20 주식회사 툴젠 슈반 세포 특이적 프로모터
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
KR20250126990A (ko) * 2024-02-16 2025-08-26 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
WO2008073303A2 (en) 2006-12-07 2008-06-19 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
AU2009271524A1 (en) 2008-07-16 2010-01-21 The Board Of Trustees Of The Leland Stanford Junior University Modulation of GM98 (MRF) in remyelination
US20120233713A1 (en) 2009-09-02 2012-09-13 Brian Popko Methods and systems for inducible ablation of neural cells
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
EP2713724A4 (en) 2011-05-26 2015-03-11 Biogen Idec Inc METHODS OF TREATING MULTIPLE SCLEROSIS AND PRESERVING AND / OR INCREASING MYELINE CONTENT
CN107551269A (zh) 2012-02-29 2018-01-09 百深公司 周围神经的IgG刺激髓鞘再生
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics 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
CN114657141B (zh) 2014-09-12 2025-02-25 京都府公立大学法人 雪旺氏细胞及其制备方法
US20180305689A1 (en) 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use
JP6981973B2 (ja) 2015-10-01 2021-12-17 ヒート バイオロジクス,インコーポレイテッド I型及びii型細胞外ドメインを異種キメラタンパク質として連結する組成物及び方法
WO2018056412A1 (ja) 2016-09-23 2018-03-29 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
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
KR102437228B1 (ko) 2016-11-14 2022-08-30 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
CN118581154A (zh) 2017-06-02 2024-09-03 国家健康与医学研究院 用于遗传障碍的基因疗法的基因组编辑手段和结合病毒载体的基因疗法
AU2018345772B2 (en) 2017-10-02 2025-04-17 Research Institute At Nationwide Children's Hospital MiRNA detargeting system for tissue specific interference
US11926653B2 (en) 2017-10-20 2024-03-12 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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