KR20210005178A - X-연관 고 igm 증후군에서의 치료적 게놈 편집 - Google Patents

X-연관 고 igm 증후군에서의 치료적 게놈 편집 Download PDF

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KR20210005178A
KR20210005178A KR1020207034098A KR20207034098A KR20210005178A KR 20210005178 A KR20210005178 A KR 20210005178A KR 1020207034098 A KR1020207034098 A KR 1020207034098A KR 20207034098 A KR20207034098 A KR 20207034098A KR 20210005178 A KR20210005178 A KR 20210005178A
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cell
cells
nucleic acid
nuclease
vector
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데이비드 제이. 롤링스
다니엘 톰슨
이람 에프. 칸
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시애틀 칠드런즈 호스피탈 디/비/에이 시애틀 칠드런즈 리서치 인스티튜트
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • 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
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0387—Animal model for diseases of the immune system
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Bioinformatics & Cheminformatics (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Communicable Diseases (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020207034098A 2018-04-27 2019-04-24 X-연관 고 igm 증후군에서의 치료적 게놈 편집 Ceased KR20210005178A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862663485P 2018-04-27 2018-04-27
US62/663,485 2018-04-27
PCT/US2019/028858 WO2019209912A2 (en) 2018-04-27 2019-04-24 Therapeutic genome editing in x-linked hyper igm syndrome

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KR20210005178A true KR20210005178A (ko) 2021-01-13

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KR1020207034098A Ceased KR20210005178A (ko) 2018-04-27 2019-04-24 X-연관 고 igm 증후군에서의 치료적 게놈 편집

Country Status (8)

Country Link
US (1) US20210324381A1 (https=)
EP (1) EP3784292A4 (https=)
JP (1) JP2021521850A (https=)
KR (1) KR20210005178A (https=)
CN (1) CN112312931A (https=)
AU (1) AU2019261385A1 (https=)
CA (1) CA3098489A1 (https=)
WO (1) WO2019209912A2 (https=)

Families Citing this family (4)

* 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
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US20230158110A1 (en) * 2018-05-30 2023-05-25 The Regents Of The University Of California Gene editing of monogenic disorders in human hematopoietic stem cells -- correction of x-linked hyper-igm syndrome (xhim)
WO2025073293A1 (en) * 2023-10-02 2025-04-10 Consorcio Centro De Investigación Biomédica En Red Gene editing methods for cd34+ cells to achieve clinically relevant efficacies

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4102099A (en) * 1998-05-29 1999-12-13 Heska Corporation Canine Interleukin -4 Proteins, Nucleic Acid Molecules, and Uses Thereof
PL3360964T3 (pl) * 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
EP3289080B1 (en) * 2015-04-30 2021-08-25 The Trustees of Columbia University in the City of New York Gene therapy for autosomal dominant diseases
US10179918B2 (en) * 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
CA2985650A1 (en) * 2015-05-13 2016-11-17 Seattle Children's Hospital (dba Seattle Children's Research Institute) Enhancing endonuclease based gene editing in primary cells
WO2018035387A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018073393A2 (en) * 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
EP3535396A1 (en) * 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
GB201618414D0 (en) * 2016-11-01 2016-12-14 Patterson James Regulated cell lines and methods of use thereof
WO2018206577A1 (en) * 2017-05-10 2018-11-15 Aarhus Universitet Interferon primed plasmacytoid dendritic cells
MA50833A (fr) * 2017-10-17 2020-08-26 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique pour l'hémophilie a
DK3701028T3 (da) * 2017-10-24 2024-11-11 Editas Medicine Inc Systemer og fremgangsmåder til behandling af hyper-igm-syndrom
US20200263206A1 (en) * 2017-11-07 2020-08-20 Editas Medicine, Inc. Targeted integration systems and methods for the treatment of hemoglobinopathies
AU2018370217A1 (en) * 2017-11-17 2020-05-28 Memorial Sloan-Kettering Cancer Center Methods and compositions for alleviating Cytokine Release Syndrome

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Publication number Publication date
EP3784292A2 (en) 2021-03-03
US20210324381A1 (en) 2021-10-21
CA3098489A1 (en) 2019-10-31
EP3784292A4 (en) 2022-01-19
WO2019209912A3 (en) 2020-01-16
JP2021521850A (ja) 2021-08-30
AU2019261385A1 (en) 2020-11-19
WO2019209912A2 (en) 2019-10-31
CN112312931A (zh) 2021-02-02

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