JP2018534950A5 - - Google Patents

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JP2018534950A5
JP2018534950A5 JP2018547872A JP2018547872A JP2018534950A5 JP 2018534950 A5 JP2018534950 A5 JP 2018534950A5 JP 2018547872 A JP2018547872 A JP 2018547872A JP 2018547872 A JP2018547872 A JP 2018547872A JP 2018534950 A5 JP2018534950 A5 JP 2018534950A5
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seq
grna molecule
nucleotide sequence
targeting domain
grna
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JP2018547872A
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JP7108307B2 (ja
JP2018534950A (ja
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Priority claimed from PCT/US2016/064285 external-priority patent/WO2017095967A2/en
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Priority to JP2022110179A priority Critical patent/JP7517724B2/ja
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Priority to JP2024104063A priority patent/JP2024153629A/ja
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JP2018547872A 2015-11-30 2016-11-30 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法 Active JP7108307B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022110179A JP7517724B2 (ja) 2015-11-30 2022-07-08 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP2024104063A JP2024153629A (ja) 2015-11-30 2024-06-27 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562260712P 2015-11-30 2015-11-30
US62/260,712 2015-11-30
US201662330336P 2016-05-02 2016-05-02
US62/330,336 2016-05-02
PCT/US2016/064285 WO2017095967A2 (en) 2015-11-30 2016-11-30 Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use

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JP2022110179A Division JP7517724B2 (ja) 2015-11-30 2022-07-08 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

Publications (3)

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JP2018534950A JP2018534950A (ja) 2018-11-29
JP2018534950A5 true JP2018534950A5 (enExample) 2020-02-27
JP7108307B2 JP7108307B2 (ja) 2022-07-28

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JP2018547872A Active JP7108307B2 (ja) 2015-11-30 2016-11-30 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP2022110179A Active JP7517724B2 (ja) 2015-11-30 2022-07-08 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP2024104063A Pending JP2024153629A (ja) 2015-11-30 2024-06-27 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

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JP2022110179A Active JP7517724B2 (ja) 2015-11-30 2022-07-08 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP2024104063A Pending JP2024153629A (ja) 2015-11-30 2024-06-27 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

Country Status (11)

Country Link
US (1) US12214054B2 (enExample)
EP (2) EP4644567A2 (enExample)
JP (3) JP7108307B2 (enExample)
KR (2) KR102787119B1 (enExample)
CN (2) CN118147141A (enExample)
AU (1) AU2016362282B2 (enExample)
CA (1) CA3001623A1 (enExample)
EA (1) EA201891317A3 (enExample)
IL (1) IL259100B2 (enExample)
MX (3) MX2018005377A (enExample)
WO (1) WO2017095967A2 (enExample)

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SG11202105873SA (en) * 2018-12-12 2021-07-29 Solid Biosciences Inc Combination therapy for treating muscular dystrophy
EP3952884A4 (en) * 2019-04-12 2023-03-22 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
AR118670A1 (es) * 2019-04-14 2021-10-20 Univ Duke Eliminación mediada por vectores aav de grandes puntos de mutación para el tratamiento de la distrofia muscular de duchenne
US20210047649A1 (en) * 2019-05-08 2021-02-18 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
CN110499333A (zh) * 2019-08-01 2019-11-26 广州德赫生物科技有限公司 用于修复dmd基因突变的核酸序列及系统
WO2021072276A1 (en) * 2019-10-11 2021-04-15 Yale University Compositions and methods for upregulating isoforms of dystrophin as therapy for duchenne muscular dystrophy (dmd)
US20230287389A1 (en) * 2019-11-07 2023-09-14 Qingdao Kingagroot Chemical Compound Co., Ltd. A method for generating new mutation in organism and use thereof
EP4125349A4 (en) * 2020-04-27 2024-07-10 Duke University GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
EP4126224A4 (en) * 2020-04-27 2024-07-03 Duke University HIGH-THROUGHPUT SCREENING METHODS TO DISCOVER OPTIMAL GRNA PAIRS FOR CRISPR-MEDIATED EXON DELETION
CN112522256B (zh) * 2020-08-19 2023-08-22 南京启真基因工程有限公司 CRISPR/Cas9系统及其在构建抗肌萎缩蛋白基因缺陷的猪源重组细胞中的应用
US20230383270A1 (en) * 2020-10-12 2023-11-30 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
EP4232152A4 (en) * 2020-10-21 2025-04-23 Duke University Dual AAV vector-mediated deletion of a large mutation hotspot for the treatment of Duchenne muscular dystrophy
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