BR112021008134A2 - Composições e métodos para edição de genoma mediado por nhej - Google Patents

Composições e métodos para edição de genoma mediado por nhej

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Publication number
BR112021008134A2
BR112021008134A2 BR112021008134A BR112021008134A BR112021008134A2 BR 112021008134 A2 BR112021008134 A2 BR 112021008134A2 BR 112021008134 A BR112021008134 A BR 112021008134A BR 112021008134 A BR112021008134 A BR 112021008134A BR 112021008134 A2 BR112021008134 A2 BR 112021008134A2
Authority
BR
Brazil
Prior art keywords
genome editing
compositions
methods
nhej
mediated genome
Prior art date
Application number
BR112021008134A
Other languages
English (en)
Inventor
I Uenishi Gene
J Cost Gregory
Original Assignee
Bayer Healthcare Llc
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 Bayer Healthcare Llc, Crispr Therapeutics Ag filed Critical Bayer Healthcare Llc
Publication of BR112021008134A2 publication Critical patent/BR112021008134A2/pt

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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
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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
    • 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
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    • A—HUMAN NECESSITIES
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    • 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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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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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    • C12N15/87—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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    • 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
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    • C12N5/0602—Vertebrate cells
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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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    • 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
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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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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

composições e métodos para edição de genoma mediado por nhej. o presente pedido se refere a composições e métodos para edição de genoma em células por mecanismos independentes de homologia, em particular para edição de genoma em células que não possuem a maquinaria necessária para reparo por mecanismos dependentes de homologia.
BR112021008134A 2018-10-30 2019-10-30 Composições e métodos para edição de genoma mediado por nhej BR112021008134A2 (pt)

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
BR112021008134A2 true BR112021008134A2 (pt) 2021-11-03

Family

ID=68655644

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021008134A BR112021008134A2 (pt) 2018-10-30 2019-10-30 Composições e métodos para edição de genoma mediado por nhej

Country Status (12)

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

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
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
MX2021004923A (es) 2021-07-16
CA3118090A1 (en) 2020-05-07

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