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1. WO2012138453 - EFFICIENT PROTEIN EXPRESSION IN VIVO USING MODIFIED RNA (MOD-RNA)

Publication Number WO/2012/138453
Publication Date 11.10.2012
International Application No. PCT/US2012/028802
International Filing Date 12.03.2012
IPC
C12N 15/87 2006.01
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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
C12N 15/11 2006.01
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
11DNA or RNA fragments; Modified forms thereof
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 43/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/-A61P41/108
A61P 35/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
CPC
A01K 2207/05
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
05Animals modified by non-integrating nucleic acids, e.g. antisense, RNAi, morpholino, episomal vector, for non-therapeutic purpose
A01K 2217/075
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
075inducing loss of function, i.e. knock out
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 2267/0393
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
0393Animal model comprising a reporter system for screening tests
A01K 67/0276
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
0276Knockout animals
Applicants
  • THE GENERAL HOSPITAL CORPORATION [US]/[US] (AllExceptUS)
  • IMMUNE DISEASE INSTITUTE, INC. [US]/[US] (AllExceptUS)
  • CHIEN, Kenneth, R. [US]/[US] (UsOnly)
  • PTASZEK, Leon, M. [US]/[US] (UsOnly)
  • LUI, Kathy, Oi-Lan (UsOnly)
  • ZANGI, Lior [IL]/[US] (UsOnly)
  • EBINA, Wataru [JP]/[US] (UsOnly)
  • ROSSI, Derrick J. [CA]/[US] (UsOnly)
Inventors
  • CHIEN, Kenneth, R.
  • PTASZEK, Leon, M.
  • LUI, Kathy, Oi-Lan
  • ZANGI, Lior
  • EBINA, Wataru
  • ROSSI, Derrick J.
Agents
  • RESNICK, David S.
Priority Data
61/471,16603.04.2011US
61/471,58404.04.2011US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) EFFICIENT PROTEIN EXPRESSION IN VIVO USING MODIFIED RNA (MOD-RNA)
(FR) EXPRESSION PROTÉIQUE EFFICACE IN VIVO À L'AIDE D'ARN MODIFIÉ (MOD-ARN)
Abstract
(EN)
Aspects of the invention described herein relate to synthetic, modified RNAs and their use in vivo to modulate gene expression. Aspects of the invention further relate to the use of these synthetic, modified RNAs in myocytes, cardiomyoctes, and tumors.
(FR)
Des aspects de la présente invention concernent des ARN synthétiques, modifiés, et leur utilisation in vivo pour moduler l'expression génique. Des aspects de l'invention concernent en outre l'utilisation de ces ARN synthétiques, modifiés, dans des myocytes, des cardiomyocytes et des tumeurs.
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