CN112312931A - X连锁高IgM综合征的治疗性基因组编辑 - Google Patents

X连锁高IgM综合征的治疗性基因组编辑 Download PDF

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CN112312931A
CN112312931A CN201980042296.4A CN201980042296A CN112312931A CN 112312931 A CN112312931 A CN 112312931A CN 201980042296 A CN201980042296 A CN 201980042296A CN 112312931 A CN112312931 A CN 112312931A
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cell
cells
nucleic acid
nuclease
seq
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戴维德·J·罗林斯
丹尼尔·汤姆森
伊拉姆·F·可汗
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Seattle Childrens Hospital
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CN201980042296.4A 2018-04-27 2019-04-24 X连锁高IgM综合征的治疗性基因组编辑 Pending CN112312931A (zh)

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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CN112312931A true CN112312931A (zh) 2021-02-02

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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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110785179A (zh) * 2017-04-21 2020-02-11 西雅图儿童医院（Dba西雅图儿童研究所） Wiskott-Aldrich综合征和X连锁血小板减少症中的治疗性基因组编辑

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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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AU4102099A (en) * 1998-05-29 1999-12-13 Heska Corporation Canine Interleukin -4 Proteins, Nucleic Acid Molecules, and Uses Thereof
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
GB201618414D0 (en) * 2016-11-01 2016-12-14 Patterson James Regulated cell lines and methods of use thereof
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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110785179A (zh) * 2017-04-21 2020-02-11 西雅图儿童医院（Dba西雅图儿童研究所） Wiskott-Aldrich综合征和X连锁血小板减少症中的治疗性基因组编辑

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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
JP2021521850A (ja) 2021-08-30
AU2019261385A1 (en) 2020-11-19
WO2019209912A2 (en) 2019-10-31

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