CN110785179A - 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 - Google Patents

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 Download PDF

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CN110785179A
CN110785179A CN201880041482.1A CN201880041482A CN110785179A CN 110785179 A CN110785179 A CN 110785179A CN 201880041482 A CN201880041482 A CN 201880041482A CN 110785179 A CN110785179 A CN 110785179A
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cell
cells
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vector
nuclease
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戴维德·J·罗林斯
伊拉姆·卡恩
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Seattle Childrens Hospital
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]

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CN201880041482.1A 2017-04-21 2018-04-19 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 Pending CN110785179A (zh)

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

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CN110785179A true CN110785179A (zh) 2020-02-11

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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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111808859A (zh) * 2020-07-13 2020-10-23 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

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* 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
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

Citations (8)

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WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
WO2016057961A1 (en) * 2014-10-10 2016-04-14 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
CN105899665A (zh) * 2013-10-17 2016-08-24 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
WO2018058064A1 (en) * 2016-09-23 2018-03-29 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing
US20190055288A1 (en) * 2015-10-20 2019-02-21 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
US20200206322A1 (en) * 2017-03-21 2020-07-02 Stitch Bio, Llc Treating cancer with cas endonuclease complexes
CN112312931A (zh) * 2018-04-27 2021-02-02 西雅图儿童医院（Dba西雅图儿童研究所） X连锁高IgM综合征的治疗性基因组编辑
CN112469823A (zh) * 2018-04-27 2021-03-09 西雅图儿童医院d/b/a西雅图儿童研究所 布鲁顿氏酪氨酸激酶的基于talen和基于crispr/cas的基因编辑

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US7582738B2 (en) * 2004-12-17 2009-09-01 Children's Hospital Medical Center Diagnostic assay for Wiskott-Aldrich syndrome and genetically related disorders
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
US11521964B2 (en) 2018-06-29 2022-12-06 Intel Corporation Schottky diode structures and integration with III-V transistors

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WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
CN105899665A (zh) * 2013-10-17 2016-08-24 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
WO2016057961A1 (en) * 2014-10-10 2016-04-14 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
US20190055288A1 (en) * 2015-10-20 2019-02-21 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
WO2018058064A1 (en) * 2016-09-23 2018-03-29 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
CN112312931A (zh) * 2018-04-27 2021-02-02 西雅图儿童医院（Dba西雅图儿童研究所） X连锁高IgM综合征的治疗性基因组编辑
CN112469823A (zh) * 2018-04-27 2021-03-09 西雅图儿童医院d/b/a西雅图儿童研究所 布鲁顿氏酪氨酸激酶的基于talen和基于crispr/cas的基因编辑

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Title
NCBI: "Homo sapiens WASP actin nucleation promoting factor (WAS), RefSeqGene (LGR_125) onchromosome X，NG_007877.1", 《NCBI》 *
PANKAJ K MANDA, ET AL: "Transient Manipulation of DNA Damage Repair Pathway Choice Improves Homology-Directed Repair During CRISPR/Cas9-Mediated Genome Editing", 《MOLECULAR THERAPY》 *
SOWMYA PATTBHI ET AL: "In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods", 《MOLECULAR THERAPY: NUCLEIC ACIDS》 *
YEBO WANG ET AL: "Integration-defective lentiviral vector mediates efficient gene editing through homology-directed repair in human embryonic stem cells", 《NUCLEIC ACID RESEARCH》 *
ZHU Q ET AL: "Wiskott-Aldrich syndrome/X-linked thrombocytopenia : WASP gene mutations, protein expression, and phenotype", 《BLOOD》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111808859A (zh) * 2020-07-13 2020-10-23 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
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

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WO2018195360A8 (en) 2019-06-13
JP2020517270A (ja) 2020-06-18
US20240141389A1 (en) 2024-05-02
US20200325494A1 (en) 2020-10-15
WO2018195360A1 (en) 2018-10-25
AU2024204421A1 (en) 2024-07-18
AU2018254529A1 (en) 2019-11-07
EP3612194A1 (en) 2020-02-26
CA3060570A1 (en) 2018-10-25
AU2018254529B2 (en) 2024-03-28
EP3612194A4 (en) 2020-12-30
US11643671B2 (en) 2023-05-09

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