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1. WO2013176772 - METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION

Publication Number WO/2013/176772
Publication Date 28.11.2013
International Application No. PCT/US2013/032589
International Filing Date 15.03.2013
IPC
C12N 15/11 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
11DNA or RNA fragments; Modified forms thereof
C12N 15/63 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
C07K 19/00 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
19Hybrid peptides
C12N 5/10 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
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
CPC
A01H 6/4684
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
HNEW PLANTS OR ; NON-TRANSGENIC; PROCESSES FOR OBTAINING THEM; PLANT REPRODUCTION BY TISSUE CULTURE TECHNIQUES
6Angiosperms, i.e. flowering plants, characterised by their botanic taxonomy
46Gramineae or Poaceae, e.g. ryegrass, rice, wheat or maize
4684Zea mays [maize]
A01K 67/027
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
A61K 38/465
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
465acting on ester bonds (3.1), e.g. lipases, ribonucleases
A61K 48/00
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 31/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
A61P 31/04
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
04Antibacterial agents
Applicants
  • THE REGENTS OF THE UNIVERSITY OF CALIFORNIA [US]/[US]
  • UNIVERSITY OF VIENNA [AT]/[AT]
  • CHARPENTIER, Emmanuelle
Inventors
  • CHARPENTIER, Emmanuelle
  • DOUDNA, Jennifer A.
  • JINEK, Martin
  • CHYLINSKI, Krzysztof
  • DOUDNA CATE, James Harrison
  • LIM, Wendell
  • QI, Lei
Agents
  • BORDEN, Paula A.
Priority Data
61/652,08625.05.2012US
61/716,25619.10.2012US
61/757,64028.01.2013US
61/765,57615.02.2013US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
(FR) PROCÉDÉS ET COMPOSITIONS PERMETTANT LA MODIFICATION DE L'ADN CIBLE DIRIGÉE PAR L'ARN ET LA MODULATION DE LA TRANSCRIPTION DIRIGÉE PAR L'ARN
Abstract
(EN) The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.
(FR) Cette invention concerne un ARN ciblant l'ADN qui comprend une séquence de ciblage et qui, avec un polypeptide de modification, permet la modification spécifique de site d'un ADN cible et/ou d'un polypeptide associé à l'ADN cible. Cette invention concerne également des polypeptides de modification spécifique de site; des procédés de modification spécifique de site d'un ADN cible et/ou d'un polypeptide associé à l'ADN cible; ainsi que des procédés de modulation de la transcription d'un acide nucléique cible dans une cellule cible, impliquant généralement la mise en contact de l'acide nucléique cible avec un polypeptide Cas9 enzymatiquement inactif et un ARN ciblant l'ADN. Des kits et des compositions pour la mise en œuvre des procédés selon l'invention sont également décrits. La présente invention permet d'obtenir des cellules génétiquement modifiées qui produisent Cas9; et des organismes Cas9 multicellulaires transgéniques non humains.
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