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1. (CA2914519) RNA-GUIDED GENE EDITING AND GENE REGULATION

Office : Canada
Application Number: 2914519 Application Date:
Publication Number: 2914519 Publication Date: 11.12.2014
Publication Kind : A1
Prior PCT appl.: Application Number:PCTUS2014041190 ; Publication Number: Click to see the data
IPC:
C07K 19/00
A61K 47/48
A61K 48/00
A61P 3/00
A61P 25/28
C12N 15/62
C12N 15/85
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
19
Hybrid peptides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47
Medicinal preparations characterised by the non-active ingredients used, e.g. carriers, inert additives
48
the non-active ingredient being chemically bound to the active ingredient, e.g. polymer drug conjugates
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3
Drugs for disorders of the metabolism
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
28
for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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
62
DNA sequences coding for fusion 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
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
CPC:
C12N 15/85
A61K 38/465
A61K 48/005
A61K 48/0058
C07K 14/4708
C07K2319/71
C12N 9/22
C12N 9/96
C12N 15/907
C12N2740/16043
C12N2800/40
C12N2840/20
C12Y 301/00
Applicants: DUKE UNIVERSITY
Inventors:
Priority Data: 61/831,481 05.06.2013 US
61/839,127 25.06.2013 US
61/904,911 15.11.2013 US
61/967,466 19.03.2014 US
61/981,575 18.04.2014 US
Title: (EN) RNA-GUIDED GENE EDITING AND GENE REGULATION
(FR) EDITION ET REGULATION GENIQUES A GUIDAGE ARN
Abstract: front page image
(EN) Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) 9-based system related compositions and methods of using said CRISPR/Cas9-based system related compositions for altering gene expression and genome engineering. Also disclosed herein are compositions and methods of using said compositions for altering gene expression and genome engineering in muscle, such as skeletal muscle and cardiac muscle.


Also published as:
EP3004370KR1020160023765CN105658805US20160201089AU2014274840WO/2014/197748