JP2020517270A5 - - Google Patents

Download PDF

Info

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
Authority
JP
Japan
Prior art keywords
cells
nuclease
nucleic acid
cell
binding site
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
JP2019557380A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020517270A (ja
Filing date
Publication date
Application filed filed Critical
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

Links

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 (https=) 2021-06-10

Family

ID=63856121

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 (https=)
EP (1) EP3612194A4 (https=)
JP (1) JP2020517270A (https=)
CN (1) CN110785179A (https=)
AU (2) AU2018254529B2 (https=)
CA (1) CA3060570A1 (https=)
WO (1) WO2018195360A1 (https=)

Families Citing this family (6)

* 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
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
JP7847850B2 (ja) * 2019-11-11 2026-04-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア ウィスコットアルドリッチ症候群(was)を治療するための、造血幹細胞中のレンチウイルスベクター
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
WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
EP3441468B1 (en) 2013-10-17 2021-05-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
AU2015330699B2 (en) * 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2017068077A1 (en) * 2015-10-20 2017-04-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
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
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
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
KR20210005178A (ko) 2018-04-27 2021-01-13 시애틀 칠드런즈 호스피탈 디/비/에이 시애틀 칠드런즈 리서치 인스티튜트 X-연관 고 igm 증후군에서의 치료적 게놈 편집
US11521964B2 (en) 2018-06-29 2022-12-06 Intel Corporation Schottky diode structures and integration with III-V transistors

Similar Documents

Publication Publication Date Title
JP2020517270A5 (https=)
JP7623272B2 (ja) 持続的エピジェネティック遺伝子サイレンシング
Ahmadi et al. Viral vectors and extracellular vesicles: innate delivery systems utilized in CRISPR/Cas-mediated cancer therapy
Newby et al. In vivo somatic cell base editing and prime editing
JP2020518276A5 (https=)
Amini et al. Super-treg: toward a new era of adoptive treg therapy enabled by genetic modifications
AU2022259838A1 (en) CRISPR/CAS-related methods and compositions for improving transplantation
ES2942309T3 (es) Materiales y métodos para el tratamiento de hemoglobinopatías
TW202028466A (zh) 編輯RNAs的方法以及組成物
JP2021511038A5 (https=)
CN110612353A (zh) 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
JP2015533786A5 (https=)
JP2021097671A5 (https=)
IL295858B1 (en) gRNA molecules including TRACR and crRNA, compositions containing them and their use
JP2020518268A5 (https=)
CA2985615A1 (en) Crispr/cas-related methods and compositions for treating hiv infection and aids
JP2017504354A5 (https=)
JP2021526858A5 (https=)
JP2021526858A (ja) Rna標的化融合タンパク質組成物および使用方法
JP2021521855A5 (https=)
JP2016512691A5 (https=)
JP2017505117A5 (https=)
JP2019503198A (ja) Dna結合ドメインと切断ドメインとを連結するための組成物
Hollinger et al. Viral vector-mediated gene therapies
RU2022103641A (ru) Искусственная модификация генома для регуляции экспрессии гена