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1. (CN108779466) Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use

Office : China
Application Number: 201680080439.7 Application Date: 30.11.2016
Publication Number: 108779466 Publication Date: 09.11.2018
Publication Kind : A
Prior PCT appl.: Application Number:PCTUS2016064285 ; Publication Number: Click to see the data
IPC:
C12N 15/113
C12N 15/864
C12N 5/10
A01K 67/027
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
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
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
86
Viral vectors
864
Parvoviral vectors
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
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
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
CPC:
A61K 48/005
A61K 31/7105
A61K 47/26
A61K 47/549
A61K 48/0016
C12N 15/102
C12N 15/113
C12N 15/80
C12N 15/8509
C12N 2310/20
C12Q 1/68
Applicants: DUKE UNIVERSITY
杜克大学
Inventors: GERSBACH CHARLES A.
C.A.格尔斯巴赫
ROBINSON-HAMM JACQUELINE N.
J.N.罗宾逊哈姆
Agents: 中国专利代理(香港)有限公司 72001
中国专利代理(香港)有限公司 72001
Priority Data: 62/260712 30.11.2015 US
62/330336 02.05.2016 US
Title: (EN) Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
(ZH) 用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法
Abstract: front page image
(EN) Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use.
(ZH) 本文披露了用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法。
Also published as:
AU2016362282CA3001623IN201837014278KR1020180081618EP3384055US20180353615
BR112018011133EA201891317WO/2017/095967