MX2021001850A - Edicion genica in situ. - Google Patents

Edicion genica in situ.

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Publication number
MX2021001850A
MX2021001850A MX2021001850A MX2021001850A MX2021001850A MX 2021001850 A MX2021001850 A MX 2021001850A MX 2021001850 A MX2021001850 A MX 2021001850A MX 2021001850 A MX2021001850 A MX 2021001850A MX 2021001850 A MX2021001850 A MX 2021001850A
Authority
MX
Mexico
Prior art keywords
cells
gene editing
stem cells
situ gene
situ
Prior art date
Application number
MX2021001850A
Other languages
English (en)
Inventor
Amy J Wagers
Jill Goldstein
Leo Wang
Ya-Chieh Hsu
Meryem Gonzalez Celeiro
Original Assignee
Harvard College
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 Harvard College filed Critical Harvard College
Publication of MX2021001850A publication Critical patent/MX2021001850A/es

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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
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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
    • C12N15/63—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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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
    • C12N15/10—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
    • 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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    • C12N15/00—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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    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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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)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se describen métodos de modificación genómica in situ de células (por ejemplo, células madre, células madre tisulares, células madre musculares, células progenitoras mesenquimatosas Sca-1* en el músculo esquelético, células mesenquimatosas dérmicas CD140a*) usando nucleasas dirigidas a secuencias administradas a través de un virus (por ejemplo, un AAV).
MX2021001850A 2018-08-18 2019-08-19 Edicion genica in situ. MX2021001850A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862719628P 2018-08-18 2018-08-18
PCT/US2019/047113 WO2020041217A1 (en) 2018-08-18 2019-08-19 In situ gene editing

Publications (1)

Publication Number Publication Date
MX2021001850A true MX2021001850A (es) 2021-07-15

Family

ID=69591186

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021001850A MX2021001850A (es) 2018-08-18 2019-08-19 Edicion genica in situ.

Country Status (13)

Country Link
US (1) US20210180087A1 (es)
EP (1) EP3836974A4 (es)
JP (1) JP2021533784A (es)
KR (1) KR20210058816A (es)
CN (1) CN113301924A (es)
AU (1) AU2019326392A1 (es)
BR (1) BR112021002916A2 (es)
CA (1) CA3110079A1 (es)
CO (1) CO2021003461A2 (es)
IL (1) IL280853A (es)
MX (1) MX2021001850A (es)
SG (1) SG11202101576UA (es)
WO (1) WO2020041217A1 (es)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2918323T3 (es) * 2006-05-31 2022-07-15 Childrens Medical Center Células madre mesenquimatosas ABC5 positivas como inmunomoduladores
EP3003391B1 (en) * 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
KR20230136697A (ko) * 2013-06-05 2023-09-26 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
WO2016089866A1 (en) * 2014-12-01 2016-06-09 President And Fellows Of Harvard College Rna-guided systems for in vivo gene editing
MX2017016844A (es) * 2015-07-16 2018-08-15 Biokine Therapeutics Ltd Composiciones y procedimientos para tratar cancer.
WO2018191440A1 (en) * 2017-04-11 2018-10-18 President And Fellows Of Harvard College In vivo gene editing of blood progenitors

Also Published As

Publication number Publication date
KR20210058816A (ko) 2021-05-24
WO2020041217A1 (en) 2020-02-27
EP3836974A4 (en) 2022-06-15
WO2020041217A8 (en) 2020-09-03
BR112021002916A2 (pt) 2021-05-11
JP2021533784A (ja) 2021-12-09
IL280853A (en) 2021-04-29
SG11202101576UA (en) 2021-03-30
US20210180087A1 (en) 2021-06-17
CN113301924A (zh) 2021-08-24
AU2019326392A1 (en) 2021-03-11
CA3110079A1 (en) 2020-02-27
EP3836974A1 (en) 2021-06-23
CO2021003461A2 (es) 2021-08-19

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