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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cell
alternatives
vector
nuclease
nucleic acid
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CA3060570A
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English (en)
French (fr)
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David J. Rawlings
Iram Khan
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Seattle Childrens Hospital
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Seattle Childrens Hospital
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [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 (enExample)
EP (1) EP3612194A4 (enExample)
JP (1) JP2020517270A (enExample)
CN (1) CN110785179A (enExample)
AU (2) AU2018254529B2 (enExample)
CA (1) CA3060570A1 (enExample)
WO (1) WO2018195360A1 (enExample)

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
JP2020517270A (ja) * 2017-04-21 2020-06-18 シアトル チルドレンズ ホスピタル, ディービーエー シアトル チルドレンズ リサーチ インスティテュート 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
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
WO2021062267A1 (en) * 2019-09-26 2021-04-01 Nantbio, Inc. Primary t-cell expansion
EP4058587A4 (en) * 2019-11-11 2024-03-20 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat wiskott-aldrich syndrome (was)
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
CN110713995B (zh) * 2013-10-17 2023-08-01 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
EP3204496A1 (en) * 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
JP7059179B2 (ja) * 2015-10-20 2022-04-25 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作のための方法及び製品
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
JP2020517270A (ja) * 2017-04-21 2020-06-18 シアトル チルドレンズ ホスピタル, ディービーエー シアトル チルドレンズ リサーチ インスティテュート 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
AU2019261385A1 (en) * 2018-04-27 2020-11-19 Seattle Children's Hospital (dba Seattle Children's Research Institute) Therapeutic genome editing in X-linked hyper IgM syndrome
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
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
AU2018254529A1 (en) 2019-11-07
US20200325494A1 (en) 2020-10-15
US11643671B2 (en) 2023-05-09
US20240141389A1 (en) 2024-05-02
CN110785179A (zh) 2020-02-11
WO2018195360A8 (en) 2019-06-13
JP2020517270A (ja) 2020-06-18
WO2018195360A1 (en) 2018-10-25
EP3612194A4 (en) 2020-12-30
AU2024204421A1 (en) 2024-07-18
EP3612194A1 (en) 2020-02-26
AU2018254529B2 (en) 2024-03-28

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