JP7736329B2 - Ｄｕｘ４遺伝子を標的とした顔面肩甲上腕型筋ジストロフィーの治療方法 - Google Patents

Ｄｕｘ４遺伝子を標的とした顔面肩甲上腕型筋ジストロフィーの治療方法

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JP7736329B2
JP7736329B2 JP2023513722A JP2023513722A JP7736329B2 JP 7736329 B2 JP7736329 B2 JP 7736329B2 JP 2023513722 A JP2023513722 A JP 2023513722A JP 2023513722 A JP2023513722 A JP 2023513722A JP 7736329 B2 JP7736329 B2 JP 7736329B2
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promoter
vector
polynucleotide
base sequence
sequence
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JP2023539631A5 (enExample
JP2023539631A (ja
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哲也 山形
ユアンバウ シン
レベッカ ウィンドミューラー
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Modalis Therapeutics Corp
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JP2023513722A 2020-08-31 2021-08-31 Ｄｕｘ４遺伝子を標的とした顔面肩甲上腕型筋ジストロフィーの治療方法 Active JP7736329B2 (ja)

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

Publications (3)

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JP2023539631A JP2023539631A (ja) 2023-09-15
JP2023539631A5 JP2023539631A5 (enExample) 2024-09-05
JP7736329B2 true JP7736329B2 (ja) 2025-09-09

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US (1) US20230323456A1 (enExample)
EP (1) EP4204556A4 (enExample)
JP (1) JP7736329B2 (enExample)
CN (1) CN116194578A (enExample)
WO (1) WO2022045366A1 (enExample)

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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018057863A1 (en) 2016-09-23 2018-03-29 University Of Massachusetts Silencing of dux4 by recombinant gene editing complexes

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018057863A1 (en) 2016-09-23 2018-03-29 University Of Massachusetts Silencing of dux4 by recombinant gene editing complexes

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
HIMEDA, C. L. et al.，CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy，Mol. Ther.，2016年，Vol. 24，pp. 527-535

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Publication number Publication date
US20230323456A1 (en) 2023-10-12
EP4204556A4 (en) 2024-10-02
EP4204556A1 (en) 2023-07-05
CN116194578A (zh) 2023-05-30
JP2023539631A (ja) 2023-09-15
WO2022045366A1 (en) 2022-03-03

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