JP7231226B2 - 顔面肩甲上腕型筋ジストロフィーのための治療標的 - Google Patents

顔面肩甲上腕型筋ジストロフィーのための治療標的 Download PDF

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JP7231226B2
JP7231226B2 JP2019523836A JP2019523836A JP7231226B2 JP 7231226 B2 JP7231226 B2 JP 7231226B2 JP 2019523836 A JP2019523836 A JP 2019523836A JP 2019523836 A JP2019523836 A JP 2019523836A JP 7231226 B2 JP7231226 B2 JP 7231226B2
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dux4
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ジョーンズ,ペーター,エル.
グリーン,マイケル,アール.
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ユニバーシティ オブ マサチューセッツ
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JP2019523836A 2016-11-07 2017-11-07 顔面肩甲上腕型筋ジストロフィーのための治療標的 Active JP7231226B2 (ja)

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US201662418694P 2016-11-07 2016-11-07
US62/418,694 2016-11-07
US201762490783P 2017-04-27 2017-04-27
US62/490,783 2017-04-27
PCT/US2017/060410 WO2018085842A1 (en) 2016-11-07 2017-11-07 Therapeutic targets for facioscapulohumeral muscular dystrophy

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US11160823B2 (en) 2016-11-07 2021-11-02 University Of Massachusetts Therapeutic targets for facioscapulohumeral muscular dystrophy
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019060432A2 (en) * 2017-09-19 2019-03-28 Children's National Medical Center GAPMERS AND METHODS OF USING THE SAME FOR THE TREATMENT OF MUSCLE DYSTROPHY
KR102922694B1 (ko) 2018-04-19 2026-02-03 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 유전자 편집을 위한 조성물 및 방법
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
EP3906308A1 (en) * 2018-12-31 2021-11-10 Research Institute at Nationwide Children's Hospital Dux4 rna silencing using rna targeting crispr-cas13b
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020252455A1 (en) * 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
AU2021257213A1 (en) * 2020-04-17 2022-11-03 Nevada Research & Innovation Corporation CRISPR-inhibition for facioscapulohumeral muscular dystrophy
CA3189601A1 (en) 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
WO2022045366A1 (en) * 2020-08-31 2022-03-03 Modalis Therapeutics Corporation Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene
JP2022055361A (ja) * 2020-09-28 2022-04-07 田辺三菱製薬株式会社 Dux4の発現を調節するための医薬組成物
CN120092093A (zh) * 2022-08-25 2025-06-03 富士胶片株式会社 靶病毒的制造方法及培养组合物
CN115896223A (zh) * 2022-10-19 2023-04-04 中国医学科学院基础医学研究所 一种表观调节因子dot1l在肌肉分化中的应用

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EP3896451B1 (en) 2024-06-26
JP2019534298A (ja) 2019-11-28
CA3042700A1 (en) 2018-05-11
EP3535588A1 (en) 2019-09-11
EP3896451A1 (en) 2021-10-20
US20220152083A1 (en) 2022-05-19
US20220226364A1 (en) 2022-07-21
AU2017355712A1 (en) 2019-05-16
WO2018085842A1 (en) 2018-05-11
AU2017355712B2 (en) 2024-01-11
JP2023071691A (ja) 2023-05-23
US11963975B2 (en) 2024-04-23
EP3535588A4 (en) 2020-09-16
US20190343865A1 (en) 2019-11-14
US11160823B2 (en) 2021-11-02

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