MX2021004923A - Composiciones y métodos para la edición del genoma mediada por nhej. - Google Patents

Composiciones y métodos para la edición del genoma mediada por nhej.

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Publication number
MX2021004923A
MX2021004923A MX2021004923A MX2021004923A MX2021004923A MX 2021004923 A MX2021004923 A MX 2021004923A MX 2021004923 A MX2021004923 A MX 2021004923A MX 2021004923 A MX2021004923 A MX 2021004923A MX 2021004923 A MX2021004923 A MX 2021004923A
Authority
MX
Mexico
Prior art keywords
genome editing
compositions
methods
nhej
mediated genome
Prior art date
Application number
MX2021004923A
Other languages
English (en)
Inventor
Gregory J Cost
Gene I Uenishi
Original Assignee
Crispr Therapeutics Ag
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Crispr Therapeutics Ag filed Critical Crispr Therapeutics Ag
Publication of MX2021004923A publication Critical patent/MX2021004923A/es

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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • 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
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0271—Chimeric vertebrates, e.g. comprising exogenous cells
    • 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
    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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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
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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
    • 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
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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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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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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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 RNAses, DNAses
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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 [CRISPRs]
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    • C12N2500/00—Specific components of cell culture medium
    • C12N2500/02—Atmosphere, e.g. low oxygen conditions
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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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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Cell Biology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

La presente solicitud se refiere a composiciones y métodos para la edición del genoma en células mediante mecanismos independientes de homología, en particular para la edición del genoma en células que carecen de la maquinaria necesaria para la reparación mediante mecanismos dependientes de homología.
MX2021004923A 2018-10-30 2019-10-30 Composiciones y métodos para la edición del genoma mediada por nhej. MX2021004923A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862752959P 2018-10-30 2018-10-30
PCT/US2019/058917 WO2020092611A1 (en) 2018-10-30 2019-10-30 Compositions and methods for nhej-mediated genome editing

Publications (1)

Publication Number Publication Date
MX2021004923A true MX2021004923A (es) 2021-07-16

Family

ID=68655644

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021004923A MX2021004923A (es) 2018-10-30 2019-10-30 Composiciones y métodos para la edición del genoma mediada por nhej.

Country Status (12)

Country Link
US (1) US20220000933A1 (es)
EP (1) EP3874052A1 (es)
JP (1) JP2022513407A (es)
KR (1) KR20210108360A (es)
CN (1) CN113272437A (es)
AU (1) AU2019369527A1 (es)
BR (1) BR112021008134A2 (es)
CA (1) CA3118090A1 (es)
IL (1) IL282608A (es)
MX (1) MX2021004923A (es)
SG (1) SG11202104346YA (es)
WO (1) WO2020092611A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021144692A1 (en) * 2020-01-14 2021-07-22 Crispr Therapeutics Ag Methods for increased efficiency of homology-directed repair

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107429263A (zh) * 2015-01-15 2017-12-01 斯坦福大学托管董事会 调控基因组编辑的方法
WO2016135557A2 (en) * 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2017087961A1 (en) * 2015-11-19 2017-05-26 University Of Washington Hematopoietic cell gene editing
AU2017286835B2 (en) 2016-06-29 2023-12-14 Crispr Therapeutics Ag Compositions and methods for gene editing
US11674158B2 (en) * 2016-07-15 2023-06-13 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
CN112512557A (zh) * 2018-04-27 2021-03-16 克里斯珀医疗股份公司 用于浆细胞耗竭的抗bcma car-t-细胞

Also Published As

Publication number Publication date
KR20210108360A (ko) 2021-09-02
WO2020092611A1 (en) 2020-05-07
CN113272437A (zh) 2021-08-17
BR112021008134A2 (pt) 2021-11-03
US20220000933A1 (en) 2022-01-06
AU2019369527A1 (en) 2021-05-20
IL282608A (en) 2021-06-30
EP3874052A1 (en) 2021-09-08
JP2022513407A (ja) 2022-02-07
SG11202104346YA (en) 2021-05-28
CA3118090A1 (en) 2020-05-07

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