JP2021521850A5 - - Google Patents

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Publication number
JP2021521850A5
JP2021521850A5 JP2020560340A JP2020560340A JP2021521850A5 JP 2021521850 A5 JP2021521850 A5 JP 2021521850A5 JP 2020560340 A JP2020560340 A JP 2020560340A JP 2020560340 A JP2020560340 A JP 2020560340A JP 2021521850 A5 JP2021521850 A5 JP 2021521850A5
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JP
Japan
Prior art keywords
nuclease
cells
nucleotide sequence
vector
seq
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2020560340A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021521850A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/028858 external-priority patent/WO2019209912A2/en
Publication of JP2021521850A publication Critical patent/JP2021521850A/ja
Publication of JP2021521850A5 publication Critical patent/JP2021521850A5/ja
Pending legal-status Critical Current

Links

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

Publications (2)

Publication Number Publication Date
JP2021521850A JP2021521850A (ja) 2021-08-30
JP2021521850A5 true JP2021521850A5 (enExample) 2023-03-30

Family

ID=68295746

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020560340A Pending JP2021521850A (ja) 2018-04-27 2019-04-24 X連鎖性高IgM症候群のゲノム編集治療

Country Status (8)

Country Link
US (1) US20210324381A1 (enExample)
EP (1) EP3784292A4 (enExample)
JP (1) JP2021521850A (enExample)
KR (1) KR20210005178A (enExample)
CN (1) CN112312931A (enExample)
AU (1) AU2019261385A1 (enExample)
CA (1) CA3098489A1 (enExample)
WO (1) WO2019209912A2 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
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
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
EP3801640A4 (en) 2018-05-30 2022-03-23 The Regents Of The University Of California GENE EDIT OF MONOGENIC DISEASES IN HUMAN HEMATOPOIETIC STEM CELLS - CORRECTION OF X-LINKED AGAMMAGLOBULINEMIA (XLA)
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
WO2026024636A1 (en) * 2024-07-24 2026-01-29 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Methods and compositions for editing a cd40lg gene

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4102099A (en) * 1998-05-29 1999-12-13 Heska Corporation Canine Interleukin -4 Proteins, Nucleic Acid Molecules, and Uses Thereof
EP3135765A1 (en) * 2012-12-06 2017-03-01 Sigma-Aldrich Co. LLC Crispr-based genome modification and regulation
US20190275168A1 (en) * 2015-04-30 2019-09-12 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
EP3294343A4 (en) * 2015-05-13 2018-09-26 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
US20180245065A1 (en) * 2016-11-01 2018-08-30 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
EP3622056A1 (en) * 2017-05-10 2020-03-18 Aarhus Universitet Interferon primed plasmacytoid dendritic cells
WO2019079527A1 (en) * 2017-10-17 2019-04-25 Casebia Therapeutics Limited Liability Partnership COMPOSITIONS AND METHODS FOR GENETIC EDITION FOR HEMOPHILIA A
ES2998052T3 (en) * 2017-10-24 2025-02-18 Editas Medicine Inc Systems and methods for treating hyper-igm syndrome
US20200263206A1 (en) * 2017-11-07 2020-08-20 Editas Medicine, Inc. Targeted integration systems and methods for the treatment of hemoglobinopathies
EP3710016A4 (en) * 2017-11-17 2021-12-01 Memorial Sloan-Kettering Cancer Center METHODS AND COMPOSITIONS FOR RELIEVING CYTOCINE RELEASE SYNDROME

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