SG11201906948UA - Human gene correction - Google Patents

Human gene correction

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Publication number
SG11201906948UA
SG11201906948UA SG11201906948UA SG11201906948UA SG11201906948UA SG 11201906948U A SG11201906948U A SG 11201906948UA SG 11201906948U A SG11201906948U A SG 11201906948UA SG 11201906948U A SG11201906948U A SG 11201906948UA SG 11201906948U A SG11201906948U A SG 11201906948UA
Authority
SG
Singapore
Prior art keywords
cell
international
primate cell
mutant allele
allele
Prior art date
Application number
SG11201906948UA
Inventor
Shoukhrat Mitalipov
Gutierrez Nuria Marti
Original Assignee
Univ Oregon Health & Science
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 Univ Oregon Health & Science filed Critical Univ Oregon Health & Science
Publication of SG11201906948UA publication Critical patent/SG11201906948UA/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/873Techniques for producing new embryos, e.g. nuclear transfer, manipulation of totipotent cells or production of chimeric embryos
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0603Embryonic cells ; Embryoid bodies
    • C12N5/0604Whole embryos; Culture medium therefor
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/106Primate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (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)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Animal Husbandry (AREA)
  • Mycology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Cell Biology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Reproductive Health (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 25 October 2018 (25.10.2018) WIP0 I PCT omit IIl °nolo mu loom °nom° oimIE (10) International Publication Number WO 2018/195418 Al (51) International Patent Classification: TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: — of inventorship (Rule 4.17(iv)) 20 April 2018 (20.04.2018) Published: with international search report (Art. 21(3)) (25) Filing Language: English with sequence listing part of description (Rule 5.2(a)) (26) Publication Language: English (30) Priority Data: 62/487,989 20 April 2017 (20.04.2017) US (71) Applicant: OREGON HEALTH & SCIENCE UNIVERSITY [US/US]; 0690 SW Bancroft Street (Mail Code L106TT), Portland, OR 97239 (US). (72) Inventors: MITALIPOV, Shoukhrat, M.; 3303 SW Bond Ave, Center for Embryonic Cell and Gene Therapy, Port- land, OR 97239 (US). MARTI GUTIERREZ, Nuria; 3303 SW Bond Ave, Center for Embryonic Cell and Gene Therapy, Portland, OR 97239 (US). (74) Agent: MITCHELL, Lauren, E.; Klarquist Sparkman LLP, One World Trade Center, Suite 1600, 121 SW Salmon Street, Portland, OR 97204 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, A01K 67/027(2006.01) C12N 15/90 (2006.01) (21) International Application Number: PCT/US2018/028560 (22) International Filing Date: (54) Title: HUMAN GENE CORRECTION 1-1 (57) : Methods are disclosed for correcting a mutant allele of a gene of interest in a primate cell. The methods include a) GO introducing a non-naturally occurring targeted nuclease and site-specific nucleotide-binding guide that act together to introduce dou- ble-stranded breaks in the mutant allele into the primate cell, wherein: i) the primate cell is undergoing mitotic cell division; ii) the 71' in primate cell comprises a genome that is heterozygous for the mutant allele, such that the genome comprises one copy of the mutant allele 01 and one copy of a wild-type allele; iii) single-stranded oligonucleotides homologous to the wild-type allele are not introduced into the 1-1 primate cell. The methods also include b) allowing the primate cell to activate homology-directed repair of the double-stranded DNA breaks in the mutant allele, thereby correcting the mutant allele using the normal wild-type allele as a repair template and producing a 1-1 © primate cell that is homozygous for the wild-type allele. The primate cell can be a one-cell embryo and/or a human cell.
SG11201906948UA 2017-04-20 2018-04-20 Human gene correction SG11201906948UA (en)

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
SG11201906948UA true SG11201906948UA (en) 2019-08-27

Family

ID=62196687

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201906948UA SG11201906948UA (en) 2017-04-20 2018-04-20 Human gene correction

Country Status (7)

Country Link
US (1) US20210130849A1 (en)
KR (1) KR20190140950A (en)
AU (1) AU2018255975A1 (en)
BR (1) BR112019021993A2 (en)
MX (1) MX2019010286A (en)
SG (1) SG11201906948UA (en)
WO (1) WO2018195418A1 (en)

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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 (en) 2016-11-09 2019-10-21 Univ Oregon Health & Science Generation of human oocytes.
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 (en) * 2018-12-26 2019-03-19 山西医科大学 People's endometrium mescenchymal stem cell is improving the application on damaged cardiomyocytes
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 (en) * 2021-05-16 2024-01-16 젠에디트바이오 리미티드 Methods for enriching target nucleic acids, identifying off-targets and evaluating gene editing efficiency

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Also Published As

Publication number Publication date
AU2018255975A1 (en) 2019-08-15
US20210130849A1 (en) 2021-05-06
MX2019010286A (en) 2019-10-21
KR20190140950A (en) 2019-12-20
BR112019021993A2 (en) 2020-05-12
WO2018195418A1 (en) 2018-10-25

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