JP2021521850A - Ｘ連鎖性高ＩｇＭ症候群のゲノム編集治療 - Google Patents

Ｘ連鎖性高ＩｇＭ症候群のゲノム編集治療 Download PDF

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JP2021521850A
JP2021521850A JP2020560340A JP2020560340A JP2021521850A JP 2021521850 A JP2021521850 A JP 2021521850A JP 2020560340 A JP2020560340 A JP 2020560340A JP 2020560340 A JP2020560340 A JP 2020560340A JP 2021521850 A JP2021521850 A JP 2021521850A
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cells
cell
nucleic acid
nuclease
vector
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ジェイ． ローリングス，デイヴィッド
ジェイ． ローリングス，デイヴィッド
トムソン，ダニエル
エフ． ハン，イラム
エフ． ハン，イラム
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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）
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）
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JP2020560340A 2018-04-27 2019-04-24 Ｘ連鎖性高ＩｇＭ症候群のゲノム編集治療 Pending JP2021521850A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862663485P 2018-04-27 2018-04-27
US62/663,485 2018-04-27
PCT/US2019/028858 WO2019209912A2 (en) 2018-04-27 2019-04-24 Therapeutic genome editing in x-linked hyper igm syndrome

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JP2021521850A true JP2021521850A (ja) 2021-08-30
JP2021521850A5 JP2021521850A5 (https=) 2023-03-30

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US (1) US20210324381A1 (https=)
EP (1) EP3784292A4 (https=)
JP (1) JP2021521850A (https=)
KR (1) KR20210005178A (https=)
CN (1) CN112312931A (https=)
AU (1) AU2019261385A1 (https=)
CA (1) CA3098489A1 (https=)
WO (1) WO2019209912A2 (https=)

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Publication number Priority date Publication date Assignee Title
CA3060570A1 (en) 2017-04-21 2018-10-25 Seattle Children's Hospital (Dba Seattle Children's Research Institute Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US20230158110A1 (en) * 2018-05-30 2023-05-25 The Regents Of The University Of California Gene editing of monogenic disorders in human hematopoietic stem cells -- correction of x-linked hyper-igm syndrome (xhim)
WO2025073293A1 (en) * 2023-10-02 2025-04-10 Consorcio Centro De Investigación Biomédica En Red Gene editing methods for cd34+ cells to achieve clinically relevant efficacies

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PL3360964T3 (pl) * 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
EP3289080B1 (en) * 2015-04-30 2021-08-25 The Trustees of Columbia University in the City of New York Gene therapy for autosomal dominant diseases
US10179918B2 (en) * 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
CA2985650A1 (en) * 2015-05-13 2016-11-17 Seattle Children's Hospital (dba Seattle Children's Research Institute) Enhancing endonuclease based gene editing in primary cells
WO2018035387A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018073393A2 (en) * 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
EP3535396A1 (en) * 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
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WO2018206577A1 (en) * 2017-05-10 2018-11-15 Aarhus Universitet Interferon primed plasmacytoid dendritic cells
MA50833A (fr) * 2017-10-17 2020-08-26 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique pour l'hémophilie a
DK3701028T3 (da) * 2017-10-24 2024-11-11 Editas Medicine Inc Systemer og fremgangsmåder til behandling af hyper-igm-syndrom
US20200263206A1 (en) * 2017-11-07 2020-08-20 Editas Medicine, Inc. Targeted integration systems and methods for the treatment of hemoglobinopathies
AU2018370217A1 (en) * 2017-11-17 2020-05-28 Memorial Sloan-Kettering Cancer Center Methods and compositions for alleviating Cytokine Release Syndrome

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Title
CURINGA, GABRIELLE ET AL.: ""Successful Editing of the CD40LG Locus in Human Hematopoietic Stem Cells"", MOLECULAR THERAPY, vol. Vol. 25, No. 5S1, 635, JPN6023050855, May 2017 (2017-05-01), ISSN: 0005416115 *
HUBBARD, NICHOLAS ET AL.: ""Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome"", BLOOD, vol. 127, no. 21, JPN6023050854, 26 May 2016 (2016-05-26), pages 2513 - 2522, XP055397792, ISSN: 0005416116, DOI: 10.1182/blood-2015-11-683235 *

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EP3784292A2 (en) 2021-03-03
US20210324381A1 (en) 2021-10-21
CA3098489A1 (en) 2019-10-31
KR20210005178A (ko) 2021-01-13
EP3784292A4 (en) 2022-01-19
WO2019209912A3 (en) 2020-01-16
AU2019261385A1 (en) 2020-11-19
WO2019209912A2 (en) 2019-10-31
CN112312931A (zh) 2021-02-02

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