JP2019522461A5 - - Google Patents

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JP2019522461A5
JP2019522461A5 JP2018558287A JP2018558287A JP2019522461A5 JP 2019522461 A5 JP2019522461 A5 JP 2019522461A5 JP 2018558287 A JP2018558287 A JP 2018558287A JP 2018558287 A JP2018558287 A JP 2018558287A JP 2019522461 A5 JP2019522461 A5 JP 2019522461A5
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JP
Japan
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seq
grna molecule
set forth
nucleotide sequence
sequence set
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JP2018558287A
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Japanese (ja)
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JP7075597B2 (ja
JP2019522461A (ja
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Priority claimed from PCT/US2017/031351 external-priority patent/WO2017193029A2/en
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JP2018558287A 2016-05-05 2017-05-05 デュシェンヌ型筋ジストロフィーを治療するためのcrispr/cas関連の方法および組成物 Active JP7075597B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662332297P 2016-05-05 2016-05-05
US62/332,297 2016-05-05
PCT/US2017/031351 WO2017193029A2 (en) 2016-05-05 2017-05-05 Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy

Publications (3)

Publication Number Publication Date
JP2019522461A JP2019522461A (ja) 2019-08-15
JP2019522461A5 true JP2019522461A5 (enExample) 2020-06-25
JP7075597B2 JP7075597B2 (ja) 2022-05-26

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JP2018558287A Active JP7075597B2 (ja) 2016-05-05 2017-05-05 デュシェンヌ型筋ジストロフィーを治療するためのcrispr/cas関連の方法および組成物

Country Status (5)

Country Link
US (1) US20190134221A1 (enExample)
EP (1) EP3452498B1 (enExample)
JP (1) JP7075597B2 (enExample)
ES (1) ES2957660T3 (enExample)
WO (1) WO2017193029A2 (enExample)

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US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
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WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
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WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
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WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
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US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
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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
US20240366604A1 (en) 2021-04-28 2024-11-07 Graviton Bioscience Bv Selective inhibitors of rock2 for the treatment of muscular dystrophy
WO2023039444A2 (en) * 2021-09-08 2023-03-16 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exon 51 for treatment of duchenne muscular dystrophy

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