TWI887234B - 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 - Google Patents

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 Download PDF

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TWI887234B
TWI887234B TW109112555A TW109112555A TWI887234B TW I887234 B TWI887234 B TW I887234B TW 109112555 A TW109112555 A TW 109112555A TW 109112555 A TW109112555 A TW 109112555A TW I887234 B TWI887234 B TW I887234B
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cas9
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TW202104594A (zh
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查爾斯 Ａ 格斯巴赫
凱倫 布拉克萊克
漢姆 賈桂琳 Ｎ 羅賓森
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美國公爵大學
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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TW109112555A 2019-04-14 2020-04-14 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 TWI887234B (zh)

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US201962833760P 2019-04-14 2019-04-14
US62/833,760 2019-04-14

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TW202104594A TW202104594A (zh) 2021-02-01
TWI887234B true TWI887234B (zh) 2025-06-21

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TW109112555A TWI887234B (zh) 2019-04-14 2020-04-14 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
TW114119381A TW202536186A (zh) 2019-04-14 2020-04-14 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

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US (1) US20220184229A1 (https=)
EP (1) EP3931331A4 (https=)
JP (2) JP2022529631A (https=)
KR (1) KR20210151916A (https=)
CN (1) CN113785063A (https=)
AR (1) AR118670A1 (https=)
AU (1) AU2020257898B2 (https=)
BR (1) BR112021020515A2 (https=)
CA (1) CA3137248A1 (https=)
CL (1) CL2021002680A1 (https=)
CO (1) CO2021013692A2 (https=)
EA (1) EA202192815A1 (https=)
IL (1) IL287163A (https=)
MX (1) MX2021012511A (https=)
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WO2013163628A2 (en) 2012-04-27 2013-10-31 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
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods 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
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
US20230272428A1 (en) * 2019-12-16 2023-08-31 President And Fellows Of Harvard College Methods and compositions for correction of dmd mutations
US20230346978A1 (en) * 2020-04-14 2023-11-02 University Of Massachusetts Dcas13-mediated therapeutic rna base editing for in vivo gene therapy
WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
MX2024002988A (es) * 2021-09-08 2024-06-11 Flagship Pioneering Innovations Vi Llc Composiciones y metodos moduladores de la serpina.
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
WO2024193704A1 (en) * 2023-03-22 2024-09-26 Huidagene Therapeutics Co., Ltd. Guide nucleic acids targeting dmd and uses thereof
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TW202104594A (zh) 2021-02-01
IL287163A (en) 2021-12-01
AR118670A1 (es) 2021-10-20
EP3931331A1 (en) 2022-01-05
CL2021002680A1 (es) 2022-07-15
EA202192815A1 (ru) 2022-01-24
CN113785063A (zh) 2021-12-10
US20220184229A1 (en) 2022-06-16
AU2020257898B2 (en) 2026-03-05
WO2020214609A1 (en) 2020-10-22
JP2022529631A (ja) 2022-06-23
AU2020257898A1 (en) 2021-11-04
BR112021020515A2 (pt) 2022-01-04
SG11202111172QA (en) 2021-11-29
KR20210151916A (ko) 2021-12-14
CA3137248A1 (en) 2020-10-22
CO2021013692A2 (es) 2022-01-17
TW202536186A (zh) 2025-09-16
JP2025087680A (ja) 2025-06-10
MX2021012511A (es) 2022-01-06
EP3931331A4 (en) 2022-11-30

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