CN116194578A - 通过靶向dux4基因来治疗面肩肱型肌营养不良症(fshd)的方法 - Google Patents

通过靶向dux4基因来治疗面肩肱型肌营养不良症(fshd)的方法 Download PDF

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CN116194578A
CN116194578A CN202180050873.1A CN202180050873A CN116194578A CN 116194578 A CN116194578 A CN 116194578A CN 202180050873 A CN202180050873 A CN 202180050873A CN 116194578 A CN116194578 A CN 116194578A
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山形哲也
秦园博
丽贝卡·温德姆勒
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Morris Medical Co ltd
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CN202180050873.1A 2020-08-31 2021-08-31 通过靶向dux4基因来治疗面肩肱型肌营养不良症(fshd)的方法 Pending CN116194578A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063072327P 2020-08-31 2020-08-31
US63/072,327 2020-08-31
US202163235359P 2021-08-20 2021-08-20
US63/235,359 2021-08-20
PCT/JP2021/031973 WO2022045366A1 (en) 2020-08-31 2021-08-31 Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene

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CN116194578A true CN116194578A (zh) 2023-05-30

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CN202180050873.1A Pending CN116194578A (zh) 2020-08-31 2021-08-31 通过靶向dux4基因来治疗面肩肱型肌营养不良症(fshd)的方法

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Country Link
US (1) US20230323456A1 (cg-RX-API-DMAC7.html)
EP (1) EP4204556A4 (cg-RX-API-DMAC7.html)
JP (1) JP7736329B2 (cg-RX-API-DMAC7.html)
CN (1) CN116194578A (cg-RX-API-DMAC7.html)
WO (1) WO2022045366A1 (cg-RX-API-DMAC7.html)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116096890A (zh) 2020-04-02 2023-05-09 米雷楚莱股份有限公司 使用工程化寡核苷酸的靶向抑制
AU2021342155A1 (en) 2020-09-11 2023-04-13 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of DUX4, compositions thereof, and methods of use
CN121127598A (zh) * 2023-03-16 2025-12-12 埃皮克里斯皮尔生物技术公司 用于调控异常基因表达的系统和方法

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3515506B1 (en) * 2016-09-23 2023-09-13 University of Massachusetts Silencing of dux4 by recombinant gene editing complexes
US11160823B2 (en) * 2016-11-07 2021-11-02 University Of Massachusetts Therapeutic targets for facioscapulohumeral muscular dystrophy
JP7412001B2 (ja) * 2018-06-08 2024-01-12 株式会社モダリス 改変されたＣａｓ９タンパク質及びその用途
MX2021004714A (es) * 2018-10-24 2021-06-04 Modalis Therapeutics Corp Proteina cas9 modificada, y uso de la misma.
AU2021257213A1 (en) * 2020-04-17 2022-11-03 Nevada Research & Innovation Corporation CRISPR-inhibition for facioscapulohumeral muscular dystrophy

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US20230323456A1 (en) 2023-10-12
EP4204556A4 (en) 2024-10-02
EP4204556A1 (en) 2023-07-05
JP7736329B2 (ja) 2025-09-09
JP2023539631A (ja) 2023-09-15
WO2022045366A1 (en) 2022-03-03

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