TW202100748A - 恢復肌縮蛋白功能之基於ｃｒｉｓｐｒ／ｃａｓ之基因組編輯組合物 - Google Patents

恢復肌縮蛋白功能之基於ｃｒｉｓｐｒ／ｃａｓ之基因組編輯組合物 Download PDF

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TW202100748A
TW202100748A TW109112554A TW109112554A TW202100748A TW 202100748 A TW202100748 A TW 202100748A TW 109112554 A TW109112554 A TW 109112554A TW 109112554 A TW109112554 A TW 109112554A TW 202100748 A TW202100748 A TW 202100748A
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查爾斯 Ａ 格斯巴赫
奧利弗 亞德里安 皮卡
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美國公爵大學
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TW109112554A 2019-04-14 2020-04-14 恢復肌縮蛋白功能之基於ｃｒｉｓｐｒ／ｃａｓ之基因組編輯組合物 TW202100748A (zh)

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US201962833759P 2019-04-14 2019-04-14
US62/833,759 2019-04-14

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US (1) US20220195406A1 (enExample)
EP (1) EP3930766A4 (enExample)
JP (1) JP7827288B2 (enExample)
AR (1) AR118668A1 (enExample)
TW (1) TW202100748A (enExample)
WO (1) WO2020214613A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115820642A (zh) * 2022-11-11 2023-03-21 昆明理工大学 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
KR20240132120A (ko) 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
JP2023507174A (ja) * 2019-12-16 2023-02-21 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｄｍｄ変異の修正のための方法及び組成物
JP2023522788A (ja) * 2020-04-27 2023-05-31 デューク ユニバーシティ 標的化されたゲノム組込みによってデュシェンヌ型筋ジストロフィーを矯正するためのｃｒｉｓｐｒ／ｃａｓ９療法
JP2023541444A (ja) * 2020-09-15 2023-10-02 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィー患者における多様なｄｍｄ変異の補正のためのａａｖ媒介性の相同性非依存的標的化組み込み遺伝子編集
WO2022221741A1 (en) * 2021-04-16 2022-10-20 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
WO2022251181A1 (en) * 2021-05-25 2022-12-01 The Board Of Regents Of The University Of Texas System Correction of duchenne muscular dystrophy mutations with all-in-one adeno-associated virus-delivered single-cut crispr
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
WO2016187717A1 (en) * 2015-05-26 2016-12-01 Exerkine Corporation Exosomes useful for genome editing
EP4279084B1 (en) 2015-10-28 2025-06-11 Vertex Pharmaceuticals Inc. Materials and methods for treatment of duchenne muscular dystrophy
EP3443081A4 (en) * 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
CA3030587A1 (en) 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
EP3746557A1 (en) * 2018-01-31 2020-12-09 The Board Of Regents Of The University Of Texas System Compositions and methods for correcting dystrophin mutations in human cardiomyocytes

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115820642A (zh) * 2022-11-11 2023-03-21 昆明理工大学 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统
CN115820642B (zh) * 2022-11-11 2023-10-10 昆明理工大学 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统

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EP3930766A4 (en) 2023-02-22
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WO2020214613A1 (en) 2020-10-22
US20220195406A1 (en) 2022-06-23
JP2022529424A (ja) 2022-06-22
JP7827288B2 (ja) 2026-03-10

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