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

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Publication Number WO/2013/176772

Publication Date 28.11.2013

International Application No. PCT/US2013/032589

International Filing Date 15.03.2013

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CPC

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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

61/652,086	25.05.2012	US
61/716,256	19.10.2012	US
61/757,640	28.01.2013	US
61/765,576	15.02.2013	US

Publication Language English (en)

Filing Language English (EN)

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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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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

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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

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07	ORGANIC CHEMISTRY
K	PEPTIDES
19	Hybrid peptides

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5	Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10	Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells

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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

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

A	HUMAN NECESSITIES
01	AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
H	NEW PLANTS OR ; NON-TRANSGENIC; PROCESSES FOR OBTAINING THEM; PLANT REPRODUCTION BY TISSUE CULTURE TECHNIQUES
6	Angiosperms, i.e. flowering plants, characterised by their botanic taxonomy
46	Gramineae or Poaceae, e.g. ryegrass, rice, wheat or maize
4684	Zea mays [maize]

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

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38	Medicinal preparations containing peptides
16	Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43	Enzymes; Proenzymes; Derivatives thereof
46	Hydrolases (3)
465	acting on ester bonds (3.1), e.g. lipases, ribonucleases

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
P	SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31	Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics

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Q	MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS
1	Measuring or testing processes involving enzymes, nucleic acids or microorganisms
68	involving nucleic acids
6844	Nucleic acid amplification reactions
686	Polymerase chain reaction [PCR]

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
Y	ENZYMES
301	Hydrolases acting on ester bonds (3.1)
04	Phosphoric diester hydrolases (3.1.4)

H	ELECTRICITY
01	BASIC ELECTRIC ELEMENTS
L	SEMICONDUCTOR DEVICES; ELECTRIC SOLID STATE DEVICES NOT OTHERWISE PROVIDED FOR
21	Processes or apparatus adapted for the manufacture or treatment of semiconductor or solid state devices or of parts thereof
02	Manufacture or treatment of semiconductor devices or of parts thereof
02104	Forming layers
02107	Forming insulating materials on a substrate
02296	characterised by the treatment performed before or after the formation of the layer
02299	pre-treatment
02312	treatment by exposure to a gas or vapour

Y	SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
02	TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
A	TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
50	in human health protection, e.g. against extreme weather
30	Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change