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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JP2019522461A (ja
JP7075597B2 (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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US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
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JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Rnaガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
MX2018005377A (es) 2015-11-30 2018-11-09 Univ Duke Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
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AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
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BR112020015617A2 (pt) * 2018-01-31 2021-01-05 The Board Of Regents Of The University Of Texas System Composições e métodos para correção de mutações da distrofina em cardiomiócitos humanos
US12152242B2 (en) 2018-04-23 2024-11-26 The Curators Of The University Of Missouri CRISPR therapy
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WO2022229351A1 (en) 2021-04-28 2022-11-03 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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JP7085716B2 (ja) * 2013-06-05 2022-06-17 デューク ユニバーシティ Rnaガイド遺伝子編集及び遺伝子調節
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MX2018005377A (es) * 2015-11-30 2018-11-09 Univ Duke Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.

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