MX2019010286A - Correccion de genes humanos. - Google Patents
Correccion de genes humanos.Info
- Publication number
- MX2019010286A MX2019010286A MX2019010286A MX2019010286A MX2019010286A MX 2019010286 A MX2019010286 A MX 2019010286A MX 2019010286 A MX2019010286 A MX 2019010286A MX 2019010286 A MX2019010286 A MX 2019010286A MX 2019010286 A MX2019010286 A MX 2019010286A
- Authority
- MX
- Mexico
- Prior art keywords
- cell
- primate cell
- allele
- mutant allele
- wild
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 108700028369 Alleles Proteins 0.000 abstract 10
- 241000288906 Primates Species 0.000 abstract 8
- 210000004027 cell Anatomy 0.000 abstract 8
- 101710163270 Nuclease Proteins 0.000 abstract 1
- 108091034117 Oligonucleotide Proteins 0.000 abstract 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 abstract 1
- 230000032823 cell division Effects 0.000 abstract 1
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 abstract 1
- 210000005260 human cell Anatomy 0.000 abstract 1
- 210000001161 mammalian embryo Anatomy 0.000 abstract 1
- 230000000394 mitotic effect Effects 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 230000003007 single stranded DNA break Effects 0.000 abstract 1
Classifications
-
- 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
-
- 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
-
- 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
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- C12N15/873—Techniques for producing new embryos, e.g. nuclear transfer, manipulation of totipotent cells or production of chimeric embryos
-
- 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
- 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
- C12N5/0603—Embryonic cells ; Embryoid bodies
- C12N5/0604—Whole embryos; Culture medium therefor
-
- 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
- A01K2227/106—Primate
-
- 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
- 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]
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Environmental Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Developmental Biology & Embryology (AREA)
- Animal Husbandry (AREA)
- Biodiversity & Conservation Biology (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Reproductive Health (AREA)
- Gynecology & Obstetrics (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
Se describen métodos para corregir un alelo mutante de un gen de interés en una célula de primate. Los métodos incluyen a) introducir una nucleasa dirigida de ocurrencia no natural y una guía de unión a nucleótidos específicos del sitio que actúan juntos para introducir rupturas bicatenarias en el alelo mutante en la célula de primate, en donde: i) la célula de primate está experimentando división de células mitóticas; ii) la célula de primate comprende un genoma que es heterocigoto para el alelo mutante, de modo que el genoma comprende una copia del alelo mutante y una copia de un alelo silvestre; iii) los oligonucleótidos monocatenarios homólogos al alelo silvestre no se introducen en la célula de primate. Los métodos también incluyen b) permitir que la célula de primate active la reparación dirigida por homología de las rupturas de ADN bicatenario en el alelo mutante, corrigiendo así el alelo mutante utilizando el alelo silvestre normal como una plantilla de reparación y produciendo una célula de primate que es homocigota para el alelo silvestre. La célula de primate puede ser un embrión de una célula y/o una célula humana.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762487989P | 2017-04-20 | 2017-04-20 | |
PCT/US2018/028560 WO2018195418A1 (en) | 2017-04-20 | 2018-04-20 | Human gene correction |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2019010286A true MX2019010286A (es) | 2019-10-21 |
Family
ID=62196687
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2019010286A MX2019010286A (es) | 2017-04-20 | 2018-04-20 | Correccion de genes humanos. |
Country Status (7)
Country | Link |
---|---|
US (1) | US20210130849A1 (es) |
KR (1) | KR20190140950A (es) |
AU (1) | AU2018255975A1 (es) |
BR (1) | BR112019021993A2 (es) |
MX (1) | MX2019010286A (es) |
SG (1) | SG11201906948UA (es) |
WO (1) | WO2018195418A1 (es) |
Families Citing this family (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2016073990A2 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods for improving crispr/cas-mediated genome-editing |
CA2999500A1 (en) | 2015-09-24 | 2017-03-30 | Editas Medicine, Inc. | Use of exonucleases to improve crispr/cas-mediated genome editing |
WO2017165826A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
EP4047092A1 (en) | 2016-04-13 | 2022-08-24 | Editas Medicine, Inc. | Cas9 fusion molecules, gene editing systems, and methods of use thereof |
MX2019005368A (es) | 2016-11-09 | 2019-10-21 | Univ Oregon Health & Science | Generación de ovocitos humanos. |
EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
CN109481467A (zh) * | 2018-12-26 | 2019-03-19 | 山西医科大学 | 人子宫内膜间充质干细胞在改善损伤心肌细胞上的应用 |
WO2020237088A1 (en) * | 2019-05-21 | 2020-11-26 | University Of Miami | Treatment of sensorineural deafness |
WO2021072361A1 (en) * | 2019-10-10 | 2021-04-15 | The Trustees Of Columbia University In The City Of New York | Gene editing to correct aneuploidies and frame shift mutations |
WO2021144574A1 (en) * | 2020-01-14 | 2021-07-22 | Pig Improvement Company Uk Limited | Gene editing of unfertilized porcine and bovine oocytes |
KR20240007765A (ko) * | 2021-05-16 | 2024-01-16 | 젠에디트바이오 리미티드 | 표적 핵산을 농축하는 방법, 오프-타겟을 식별하고 유전자 편집 효율성을 평가하는 방법 |
Family Cites Families (33)
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US4589402A (en) | 1984-07-26 | 1986-05-20 | Serono Laboratories, Inc. | Method of in vitro fertilization |
US4725579A (en) | 1985-02-21 | 1988-02-16 | Serono Laboratories, Inc. | Method of in vitro fertilization by a unique combination of gonadotropins |
US5206347A (en) | 1985-08-06 | 1993-04-27 | La Jolla Cancer Research Foundation | Isolation and use of receptors binding to a peptide column |
US5750342A (en) | 1990-06-11 | 1998-05-12 | Nexstar Pharmaceuticals, Inc. | Nucleic acid ligands of tissue target |
US5264563A (en) | 1990-08-24 | 1993-11-23 | Ixsys Inc. | Process for synthesizing oligonucleotides with random codons |
US5226914A (en) | 1990-11-16 | 1993-07-13 | Caplan Arnold I | Method for treating connective tissue disorders |
US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
CA2146747C (en) | 1992-10-09 | 2006-12-19 | Brian A. Naughton | Liver reserve cells |
ATE310812T1 (de) | 1994-01-18 | 2005-12-15 | Scripps Research Inst | Derivate von zinkfingerproteinen und methoden |
US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
ATE407205T1 (de) | 1994-08-20 | 2008-09-15 | Gendaq Ltd | Verbesserung in bezug auf bindungsproteine bei der erkennung von dna |
GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
US5843780A (en) | 1995-01-20 | 1998-12-01 | Wisconsin Alumni Research Foundation | Primate embryonic stem cells |
US5789538A (en) | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
US5622699A (en) | 1995-09-11 | 1997-04-22 | La Jolla Cancer Research Foundation | Method of identifying molecules that home to a selected organ in vivo |
US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
AU2103601A (en) | 1999-12-17 | 2001-06-25 | Oregon Health And Science University | Methods for producing transgenic animals |
US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
WO2001088197A2 (en) | 2000-05-16 | 2001-11-22 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
JP2005500061A (ja) | 2001-08-20 | 2005-01-06 | ザ スクリップス リサーチ インスティテュート | Cnnについての亜鉛フィンガー結合ドメイン |
WO2008144580A2 (en) | 2007-05-17 | 2008-11-27 | Oregon Health & Science University | Primate stem cells produced by somatic cell nuclear transfer |
AU2010235161B2 (en) | 2009-04-09 | 2015-01-22 | Sangamo Therapeutics, Inc. | Targeted integration into stem cells |
WO2016097751A1 (en) | 2014-12-18 | 2016-06-23 | The University Of Bath | Method of cas9 mediated genome engineering |
US20160376610A1 (en) * | 2015-06-24 | 2016-12-29 | Sigma-Aldrich Co. Llc | Cell cycle dependent genome regulation and modification |
-
2018
- 2018-04-20 MX MX2019010286A patent/MX2019010286A/es unknown
- 2018-04-20 WO PCT/US2018/028560 patent/WO2018195418A1/en active Application Filing
- 2018-04-20 KR KR1020197032695A patent/KR20190140950A/ko not_active Application Discontinuation
- 2018-04-20 BR BR112019021993-5A patent/BR112019021993A2/pt not_active Application Discontinuation
- 2018-04-20 US US16/605,770 patent/US20210130849A1/en not_active Abandoned
- 2018-04-20 SG SG11201906948UA patent/SG11201906948UA/en unknown
- 2018-04-20 AU AU2018255975A patent/AU2018255975A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
SG11201906948UA (en) | 2019-08-27 |
AU2018255975A1 (en) | 2019-08-15 |
US20210130849A1 (en) | 2021-05-06 |
KR20190140950A (ko) | 2019-12-20 |
BR112019021993A2 (pt) | 2020-05-12 |
WO2018195418A1 (en) | 2018-10-25 |
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