AU2018254529B2 - 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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AU2018254529B2
AU2018254529B2 AU2018254529A AU2018254529A AU2018254529B2 AU 2018254529 B2 AU2018254529 B2 AU 2018254529B2 AU 2018254529 A AU2018254529 A AU 2018254529A AU 2018254529 A AU2018254529 A AU 2018254529A AU 2018254529 B2 AU2018254529 B2 AU 2018254529B2
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aav
nuclease
rule
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AU2018254529A1 (en
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Iram Khan
David J. Rawlings
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Seattle Childrens Hospital
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Seattle Childrens Hospital
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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)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
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  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
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  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
AU2018254529A 2017-04-21 2018-04-19 Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia Active AU2018254529B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024204421A AU2024204421A1 (en) 2017-04-21 2024-06-27 Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia

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

Related Child Applications (1)

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AU2024204421A Division AU2024204421A1 (en) 2017-04-21 2024-06-27 Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia

Publications (2)

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AU2018254529A1 AU2018254529A1 (en) 2019-11-07
AU2018254529B2 true AU2018254529B2 (en) 2024-03-28

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AU2018254529A Active AU2018254529B2 (en) 2017-04-21 2018-04-19 Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia
AU2024204421A Pending AU2024204421A1 (en) 2017-04-21 2024-06-27 Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia

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AU2024204421A Pending AU2024204421A1 (en) 2017-04-21 2024-06-27 Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia

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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)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018254529B2 (en) * 2017-04-21 2024-03-28 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
EP4034641A4 (en) * 2019-09-26 2024-01-03 NantBio, Inc. PRIMARY T-CELL EXPANSION
US20220387528A1 (en) * 2019-11-11 2022-12-08 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
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)

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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
DK3441468T3 (da) 2013-10-17 2021-07-26 Sangamo Therapeutics Inc Afgivelsesfremgangsmåder og sammensætninger til nukleasemedieret genom-manipulation
AU2015330699B2 (en) * 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
US10968253B2 (en) * 2015-10-20 2021-04-06 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
EP3516058A1 (en) * 2016-09-23 2019-07-31 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
AU2018254529B2 (en) * 2017-04-21 2024-03-28 Seattle Children's Hospital (dba Seattle Children's Research Institute) Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia
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
CA3098435A1 (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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
WANG ET AL., NUCLEIC ACID RESEARCH, vol. 45, no. 5, 29 November 2016 (2016-11-29), pages 1 - 12, XP055546039 *

Also Published As

Publication number Publication date
AU2024204421A1 (en) 2024-07-18
EP3612194A1 (en) 2020-02-26
JP2020517270A (ja) 2020-06-18
US20240141389A1 (en) 2024-05-02
WO2018195360A1 (en) 2018-10-25
CN110785179A (zh) 2020-02-11
US20200325494A1 (en) 2020-10-15
US11643671B2 (en) 2023-05-09
AU2018254529A1 (en) 2019-11-07
EP3612194A4 (en) 2020-12-30
WO2018195360A8 (en) 2019-06-13
CA3060570A1 (en) 2018-10-25

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