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1. (WO2017070428) GENETICALLY MODIFIED NON-HUMAN MAMMALS HAVING MODIFIED LIVER CELLS AND/OR TISSUE AND METHODS OF MAKING SAME
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Pub. No.: WO/2017/070428 International Application No.: PCT/US2016/058043
Publication Date: 27.04.2017 International Filing Date: 21.10.2016
IPC:
C12N 15/09 (2006.01) ,C12N 15/12 (2006.01) ,C12N 15/63 (2006.01) ,C12N 15/861 (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
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
12
Genes encoding animal proteins
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
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
86
Viral vectors
861
Adenoviral vectors
Applicants:
HERA TESTING LABORATORIES, INC. [US/US]; 535 West 2nd Street Lexington, KY 40508, US
Inventors:
CRAWFORD, John, Stuart; US
YESHI, Tseten; US
NOTO, Fallon; US
Agent:
TEPE, Nicole, M.; US
SCHOEN, Ann, G.; US
ACHESON, Edwin Jr., R.; US
GOLDSTEIN, Steven, J.; US
CONLEY, Scott, R.; US
Priority Data:
62/245,04922.10.2015US
Title (EN) GENETICALLY MODIFIED NON-HUMAN MAMMALS HAVING MODIFIED LIVER CELLS AND/OR TISSUE AND METHODS OF MAKING SAME
(FR) MAMMIFÈRES NON HUMAINS GÉNÉTIQUEMENT MODIFIÉS AYANT DES CELLULES ET/OU DES TISSUS HÉPATIQUES MODIFIÉS ET LEURS PROCÉDÉS DE FABRICATION
Abstract:
(EN) Disclosed are non-human mammals having a modified liver and methods of making such non-human mammals having a modified liver. The modified liver may be characterized by a non-germline, stable integration of a non-endogenous gene targeted to the liver of the non-human mammal. In certain aspects, the modified liver may have greater than at least 30% ablation of the endogenous hepatocyte population of the non-human mammal. In certain aspects, the non-human mammal may comprise at least 30% non-endogenous hepatocytes. The disclosed non-human mammals may be useful for pharmacology, drug absorption, distribution, metabolism, and excretion studies, collectively ADME and toxicology studies (ADME-tox), and/or drug screening.
(FR) L'invention concerne des mammifères non humains présentant un foie modifié et des procédés de fabrication de ces mammifères non humains présentant un foie modifié. Le foie modifié peut être caractérisé par une intégration stable, non dans la lignée germinale, d'un gène non endogène ciblé vers le foie du mammifère non humain. Selon certains aspects, le foie modifié peut présenter une ablation supérieure à 30 % au moins de la population d'hépatocytes endogènes du mammifère non humain. Selon certains aspects, le mammifère non humain peut comprendre au moins 30 % d'hépatocytes non endogènes. Les mammifères non humains décrits peuvent être utiles pour des études de pharmacologie, d'absorption, de distribution, de métabolisme, d'excrétion de médicaments, des études d'ADME et de toxicologie collectivement (ADME-tox), et/ou le criblage de médicaments.
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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, JP, KE, KG, 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)