CA3060570A1 - Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia - Google Patents

Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia Download PDF

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Publication number
CA3060570A1
CA3060570A1 CA3060570A CA3060570A CA3060570A1 CA 3060570 A1 CA3060570 A1 CA 3060570A1 CA 3060570 A CA3060570 A CA 3060570A CA 3060570 A CA3060570 A CA 3060570A CA 3060570 A1 CA3060570 A1 CA 3060570A1
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Prior art keywords
cell
alternatives
vector
nuclease
nucleic acid
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Pending
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CA3060570A
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English (en)
French (fr)
Inventor
David J. Rawlings
Iram Khan
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Seattle Childrens Hospital
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Seattle Childrens Hospital
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Application filed by Seattle Childrens Hospital filed Critical Seattle Childrens Hospital
Publication of CA3060570A1 publication Critical patent/CA3060570A1/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3060570A 2017-04-21 2018-04-19 Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia Pending CA3060570A1 (en)

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 (1)

Publication Number Publication Date
CA3060570A1 true CA3060570A1 (en) 2018-10-25

Family

ID=63856121

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3060570A Pending CA3060570A1 (en) 2017-04-21 2018-04-19 Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia

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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115927585A (zh) * 2022-08-29 2023-04-07 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

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
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

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

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115927585A (zh) * 2022-08-29 2023-04-07 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
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

Also Published As

Publication number Publication date
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
CN110785179A (zh) 2020-02-11
EP3612194A1 (en) 2020-02-26
AU2018254529B2 (en) 2024-03-28
EP3612194A4 (en) 2020-12-30
US11643671B2 (en) 2023-05-09

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