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1. (WO2019028032) CAS-TRANSGENIC MOUSE EMBRYONIC STEM CELLS AND MICE AND USES THEREOF
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Pub. No.: WO/2019/028032 International Application No.: PCT/US2018/044615
Publication Date: 07.02.2019 International Filing Date: 31.07.2018
IPC:
A01K 67/027 (2006.01) ,C12N 15/90 (2006.01)
A HUMAN NECESSITIES
01
AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
K
ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
67
Rearing or breeding animals, not otherwise provided for; New breeds of animals
027
New breeds of vertebrates
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
87
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90
Stable introduction of foreign DNA into chromosome
Applicants:
REGENERON PHARMACEUTICALS, INC. [US/US]; 777 Old Saw Mill River Road Tarrytown, New York 10591, US
Inventors:
GONG, Guochun; US
HUNT, Charleen; US
KAJIMURA, Daisuke; US
HARTFORD, Suzanne; US
ZAMBROWICZ, Brian; US
Agent:
SCHILLING, Stephen H.; US
LIEBESCHUETZ, Joe; US
CELLI, Rosemarie; US
SATHER, Susan; US
O'CONNOR, Daniel J.; US
HIGBEE, Zachary A.; US
CHANG, Cindy; US
Priority Data:
62/539,27531.07.2017US
Title (EN) CAS-TRANSGENIC MOUSE EMBRYONIC STEM CELLS AND MICE AND USES THEREOF
(FR) CELLULES SOUCHES EMBRYONNAIRES DE SOURIS TRANSGÉNIQUES CAS ET SOURIS ET LEURS UTILISATIONS
Abstract:
(EN) Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue- specific or temporal- specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.
(FR) La présente invention concerne des procédés et des compositions pour évaluer l'activité de ligature d'extrémités non homologues (NHEJ) à médiation par CRISPR/Cas et/ou la recombinaison induite par CRISPR/Cas d'un locus génomique cible avec un acide nucléique de donneur exogène in vivo et ex vivo. Les procédés et les compositions utilisent des cellules et des animaux non humains comprenant une cassette d'expression de Cas, telle qu'une cassette d'expression de Cas intégrée dans le génome, de telle sorte que la protéine Cas peut être disponible de manière constitutive ou disponible d'une manière spécifique d'un tissu ou spécifique temporellement. La présente invention concerne également des procédés et des compositions pour fabriquer et utiliser ces animaux non humains, y compris l'utilisation de ces animaux non humains pour évaluer l'activité de CRISPR/Cas in vivo par l'intermédiaire d'une administration à médiation par un virus adéno-associé (AAV) d'ARN de guidage aux animaux non humains.
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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 Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (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)