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1. (WO2019067875) NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS AND METHODS OF USE
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Pub. No.: WO/2019/067875 International Application No.: PCT/US2018/053389
Publication Date: 04.04.2019 International Filing Date: 28.09.2018
IPC:
A01K 67/027 (2006.01) ,C07K 14/47 (2006.01) ,A61K 48/00 (2006.01) ,C12N 15/113 (2010.01)
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
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435
from animals; from humans
46
from vertebrates
47
from mammals
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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
Applicants:
REGENERON PHARMACEUTICALS, INC. [US/US]; 777 Old Saw Mill River Road Tarrytown, New York 10591, US
Inventors:
DRUMMOND-SAMUELSON, Meghan; US
HAINES, Jeffery; US
HARTFORD, Suzanne; US
FRENDEWEY, David; US
ZAMBROWICZ, Brian; US
MURPHY, Andrew J.; US
Agent:
SCHILLING, Stephen H.; US
EKENA, Kirk; US
LIEBESCHUETZ, Joe; US
CELLI, Rosemarie; US
CHANG, Cindy; US
HIGBEE, Zachary A.; US
Priority Data:
62/565,98029.09.2017US
62/679,14201.06.2018US
62/720,29221.08.2018US
Title (EN) NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS AND METHODS OF USE
(FR) ANIMAUX NON HUMAINS COMPRENANT UN LOCUS TTR HUMANISÉ ET PROCÉDÉS D'UTILISATION
Abstract:
(EN) Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TTR locus and methods of using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TTR locus express a human transthyretin protein or a chimeric transthyretin protein, fragments of which are from human transthyretin. Methods are provided for using such non-human animals comprising a humanized TTR locus to assess in vivo efficacy of human-TTR-targeting reagents such as nuclease agents designed to target human TTR. Methods are also provided for making such non-human animals comprising a humanized TTR locus.
(FR) L'invention concerne des génomes animaux non humains, des cellules animales non humaines et des animaux non humains comprenant un locus TTR humanisé et des procédés d'utilisation de tels génomes animaux non humains, cellules animales non humaines et animaux non humains. Des cellules animales non humaines ou des animaux non humains comprenant un locus TTR humanisé expriment une protéine ASGR1 humaine ou une protéine de type transthyrétine chimérique, dont des fragments proviennent de transthyrétine humaine. L'invention concerne également des procédés d'utilisation de tels animaux non humains comprenant un locus TTR humanisé pour évaluer l'efficacité in vivo de réactifs ciblant le TTR humain tels que des agents à base de nucléase conçus pour cibler le TTR humain. L'invention concerne également des procédés de production de tels animaux non humains comprenant un locus TTR humanisé.
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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)