JP2023541444A - 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 - Google Patents

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 Download PDF

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JP2023541444A
JP2023541444A JP2023516690A JP2023516690A JP2023541444A JP 2023541444 A JP2023541444 A JP 2023541444A JP 2023516690 A JP2023516690 A JP 2023516690A JP 2023516690 A JP2023516690 A JP 2023516690A JP 2023541444 A JP2023541444 A JP 2023541444A
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nucleotide sequence
nucleic acid
promoter
dmd
seq
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ケビン フラニガン，
アンソニー アーロン スティーブンソン，
ジュリアン ヘイブンズ，
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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
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JP2023516690A 2020-09-15 2021-09-15 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 Pending JP2023541444A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063078428P 2020-09-15 2020-09-15
US63/078,428 2020-09-15
US202163180232P 2021-04-27 2021-04-27
US63/180,232 2021-04-27
PCT/US2021/050461 WO2022060841A2 (en) 2020-09-15 2021-09-15 Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy

Publications (2)

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JP2023541444A true JP2023541444A (ja) 2023-10-02
JP2023541444A5 JP2023541444A5 (https=) 2024-09-20

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JP2023516690A Pending JP2023541444A (ja) 2020-09-15 2021-09-15 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

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US (1) US20230357795A1 (https=)
EP (1) EP4214317A2 (https=)
JP (1) JP2023541444A (https=)
AU (1) AU2021345112A1 (https=)
CA (1) CA3195233A1 (https=)
WO (1) WO2022060841A2 (https=)

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Publication number Priority date Publication date Assignee Title
WO2023240157A2 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
CN115806989B (zh) * 2022-11-25 2023-08-08 昆明理工大学 针对DMD基因5号外显子突变的sgRNA及载体和应用

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WO2022060841A3 (en) 2022-04-28
EP4214317A2 (en) 2023-07-26
WO2022060841A2 (en) 2022-03-24
AU2021345112A1 (en) 2023-04-27
US20230357795A1 (en) 2023-11-09
CA3195233A1 (en) 2022-03-24

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