JP2020517270A5 - - Google Patents

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Publication number
JP2020517270A5
JP2020517270A5 JP2019557380A JP2019557380A JP2020517270A5 JP 2020517270 A5 JP2020517270 A5 JP 2020517270A5 JP 2019557380 A JP2019557380 A JP 2019557380A JP 2019557380 A JP2019557380 A JP 2019557380A JP 2020517270 A5 JP2020517270 A5 JP 2020517270A5
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JP
Japan
Prior art keywords
cells
nuclease
nucleic acid
cell
binding site
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Pending
Application number
JP2019557380A
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English (en)
Japanese (ja)
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JP2020517270A (ja
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Priority claimed from PCT/US2018/028442 external-priority patent/WO2018195360A1/en
Publication of JP2020517270A publication Critical patent/JP2020517270A/ja
Publication of JP2020517270A5 publication Critical patent/JP2020517270A5/ja
Pending legal-status Critical Current

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JP2019557380A 2017-04-21 2018-04-19 ウィスコット・アルドリッチ症候群およびｘ連鎖性血小板減少症の治療的ゲノム編集 Pending JP2020517270A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762488249P 2017-04-21 2017-04-21
US62/488,249 2017-04-21
PCT/US2018/028442 WO2018195360A1 (en) 2017-04-21 2018-04-19 Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia

Publications (2)

Publication Number Publication Date
JP2020517270A JP2020517270A (ja) 2020-06-18
JP2020517270A5 true JP2020517270A5 (enExample) 2021-06-10

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019557380A Pending JP2020517270A (ja) 2017-04-21 2018-04-19 ウィスコット・アルドリッチ症候群およびｘ連鎖性血小板減少症の治療的ゲノム編集

Country Status (7)

Country Link
US (2) US11643671B2 (enExample)
EP (1) EP3612194A4 (enExample)
JP (1) JP2020517270A (enExample)
CN (1) CN110785179A (enExample)
AU (2) AU2018254529B2 (enExample)
CA (1) CA3060570A1 (enExample)
WO (1) WO2018195360A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11643671B2 (en) * 2017-04-21 2023-05-09 Seattle Children's Hospital Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia
WO2020150499A1 (en) * 2019-01-16 2020-07-23 Fred Hutchinson Cancer Research Center Methods to tag and isolate cells infected with the human immunodeficiency virus
US12016883B2 (en) * 2019-09-26 2024-06-25 Nantbio, Inc. Primary T-cell expansion
CN114945674A (zh) * 2019-11-11 2022-08-26 加利福尼亚大学董事会 造血干细胞中治疗wiskott-aldrich综合征(was)的慢病毒载体
CN111808859B (zh) * 2020-07-13 2022-09-13 中国科学院广州生物医药与健康研究院 WAS基因的gRNA及其应用
CN115927585B (zh) * 2022-08-29 2024-04-23 湖南家辉生物技术有限公司 WAS致病突变基因及在制备Wiskott-Aldrich综合征诊断试剂盒中的应用

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7582738B2 (en) * 2004-12-17 2009-09-01 Children's Hospital Medical Center Diagnostic assay for Wiskott-Aldrich syndrome and genetically related disorders
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
CN110713995B (zh) * 2013-10-17 2023-08-01 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
AU2015330699B2 (en) * 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
JP7059179B2 (ja) * 2015-10-20 2022-04-25 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作のための方法及び製品
US20180127786A1 (en) * 2016-09-23 2018-05-10 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing
US20200206322A1 (en) * 2017-03-21 2020-07-02 Stitch Bio, Llc Treating cancer with cas endonuclease complexes
US11643671B2 (en) * 2017-04-21 2023-05-09 Seattle Children's Hospital Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia
WO2019210216A2 (en) 2018-04-27 2019-10-31 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Talen-based and crispr/cas-based gene editing for bruton's tyrosine kinase
JP2021521850A (ja) 2018-04-27 2021-08-30 シアトル チルドレンズ ホスピタル （ディービーエイ シアトル チルドレンズ リサーチ インスティテュート） Ｘ連鎖性高ＩｇＭ症候群のゲノム編集治療
US11521964B2 (en) 2018-06-29 2022-12-06 Intel Corporation Schottky diode structures and integration with III-V transistors

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