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1. WO2015171523 - HUMANIZED C5 AND C3 ANIMALS

Publication Number WO/2015/171523
Publication Date 12.11.2015
International Application No. PCT/US2015/029111
International Filing Date 04.05.2015
IPC
A01K 67/027 2006.1
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
67Rearing or breeding animals, not otherwise provided for; New breeds of animals
027New breeds of vertebrates
A61K 49/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
49Preparations for testing in vivo
C07K 14/47 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
C12N 15/85 2006.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
CPC
A01K 2207/15
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2207Modified animals
15Humanized animals
A01K 2217/072
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2217Genetically modified animals
07Animals genetically altered by homologous recombination
072maintaining or altering function, i.e. knock in
A01K 2227/105
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2227Animals characterised by species
10Mammal
105Murine
A01K 2267/03
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2267Animals characterised by purpose
03Animal model, e.g. for test or diseases
A01K 2267/0368
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2267Animals characterised by purpose
03Animal model, e.g. for test or diseases
035Animal model for multifactorial diseases
0368Animal model for inflammation
A01K 67/0278
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
67Rearing or breeding animals, not otherwise provided for; New breeds of animals
027New breeds of vertebrates
0275Genetically modified vertebrates, e.g. transgenic
0278Humanized animals, e.g. knockin
Applicants
  • REGENERON PHARMACEUTICALS, INC. [US]/[US]
Inventors
  • HU, Ying
  • LATUSZEK, Adrianna
  • CAO, Jingtai
  • MUJICA, Alexander
  • WIEGAND, Stanley
  • MCWHIRTER, John
  • MURPHY, Andrew
  • MACDONALD, Lynn
Agents
  • SHIEH-NEWTON, Terri
Priority Data
61/988,58105.05.2014US
62/067,83623.10.2014US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) HUMANIZED C5 AND C3 ANIMALS
(FR) ANIMAUX PORTEURS DES SÉQUENCES C5 ET C3 HUMANISÉES
Abstract
(EN) Non-human animals comprising a human or humanized C3 and/or C5 nucleic acid sequence are provided as well as methods for using the same to identify compounds capable of modulating the complement system. Non-human animals that comprise a replacement of the endogenous C5 gene and/or C3 gene with a human or humanized C5 gene and/or C3 gene, and methods for making and using the non-human animals, are described. Non-human animals comprising a human or humanized C5 gene under control of non-human C5 regulatory elements is also provided, including non-human animals that have a replacement of non-human C5-encoding sequence with human C5-encoding sequence at an endogenous non-human C5 locus. Non-human animals comprising a human or humanized C3 gene under control of non-human C3 regulatory elements is also provided, including non-human animals that have a replacement of non-human C3 protein-encoding sequence with human or humanized C3 protein-encoding sequence at an endogenous non-human C3 locus. Non-human animals comprising human or humanized C3 and/or C5 sequences, wherein the non-human animals are rodents, e.g., mice or rats, are provided.
(FR) La présente invention concerne des animaux non humains comprenant une séquence d'acide nucléique C3 et/ou C5 humain ou humanisé ainsi que des procédés d'utilisation de celle-ci pour identifier des composés capables de moduler le système du complément. L'invention décrit des animaux non humains pour lesquels on a remplacé le gène C5 et/ou le gène C3 endogène par un gène C5 et/ou un gène C3 humain ou humanisé, et des procédés de production et d'utilisation des animaux non humains. Des animaux non humains comprenant un gène C5 humain ou humanisé sous le contrôle d'éléments régulateurs C5 non humains sont également prévus, lesquels comprennent des animaux non humains dont une séquence codant pour C5 non humaine est remplacée par une séquence codant pour C5 humaine au niveau d'un locus C5 non humain endogène. Des animaux non humains comprenant un gène C3 humain ou humanisé sous le contrôle d'éléments régulateurs C3 non humains sont également prévus, lesquels comprennent des animaux non humains dont une séquence codant pour la protéine C3 non humaine est remplacée par une séquence codant pour la protéine C3 humaine ou humanisée au niveau d'un locus C3 non humain endogène. L'invention concerne des animaux non humains comprenant des séquences C3 et/ou C5 humaines ou humanisées, lesdits animaux non humains étant des rongeurs, par exemple des souris ou des rats.
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