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1. (WO2018026723) HUMAN INDUCED PLURIPOTENT STEM CELLS FOR HIGH EFFICIENCY GENETIC ENGINEERING
Latest bibliographic data on file with the International BureauSubmit observation
Pub. No.: WO/2018/026723 International Application No.: PCT/US2017/044719
Publication Date: 08.02.2018 International Filing Date: 31.07.2017
IPC:
C12N 15/113 (2010.01) ,C12N 5/10 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10
Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
Applicants:
UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION [US/US]; 1st Floor Gardner Stell Conference Center 130 Thackeray Avenue Pittsburgh, PA 15260, US
Inventors:
SOTO-GUTIERREZ, Alejandro; US
COLLIN DE I'HORTET, Alexandra, Sylvie; US
HANDA, Kan; US
LEPE, Jorge, Guzman; US
TAKEISHI, Kazuki; US
FOX, Ira, Jacob; US
WANG, Yang; US
Agent:
SIEGEL, Susan, Alpert; US
Priority Data:
62/369,69801.08.2016US
Title (EN) HUMAN INDUCED PLURIPOTENT STEM CELLS FOR HIGH EFFICIENCY GENETIC ENGINEERING
(FR) CELLULES SOUCHES PLURIPOTENTES INDUITES HUMAINES POUR UN GÉNIE GENETIQUE À HAUT RENDEMENT
Abstract:
(EN) Methods are disclosed herein for efficiently generating human induced pluripotent stem cells (iPSC) containing a nucleic acid including a doxycycline promoter operably linked to a nucleic acid encoding Cas9. These methods include transfecting a human somatic cell with a nucleic acid molecule comprising a doxycycline promoter operably linked to a nucleic acid encoding a Cas9, and constitutive promoter operably linked to a tetracycline responsive element and inducing the somatic cell to form an iPSC, thereby producing an iPSC that can undergo CRISPR/Cas9-mediated recombination at a high efficiency. The human iPSC, or a cell differentiated therefrom, is cultured in the presence of doxycycline to induce expression of the Cas9. These cells can then be used to target in any gene of interest by introducing nucleic acids encoding sgRNAs. Induced pluripotent stem cells produced by these methods are also disclosed.
(FR) L'invention concerne des procédés pour générer d'une manière efficace des cellules souches pluripotentes induites (iPSC) humaines contenant un acide nucléique comprenant un promoteur de doxycycline fonctionnellement lié à un acide nucléique codant pour une Cas9. Ces procédés comprennent la transfection d'une cellule somatique humaine avec une molécule d'acide nucléique comprenant un promoteur de doxycycline fonctionnellement lié à un acide nucléique codant pour une Cas9, et un promoteur constitutif fonctionnellement lié à un élément sensible à la tétracycline, et induisant une cellule somatique pour former une iPSC, en produisant de ce fait une iPSC qui peut subir une recombinaison, médiée par CRISPR/Cas9, avec un rendement élevé. L'iPSC humaine, ou une cellule différenciée obtenue à partir de cette dernière, est cultivée en présence de doxycycline pour induire l'expression de la Cas9. Ces cellules peuvent ensuite être utilisées pour cibler tout gène d'intérêt, par introduction d'acides nucléiques codant pour des petits ARN guides (sgRNA). La présente invention concerne en outre les cellules souches pluripotentes induites produites par ces procédés.
Designated States: 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
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (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, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)
Also published as:
CA3030340EP3491134US20190185819