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VECTEUR AAV POUR LA DISRUPTION DU GÈNE DU FACTEUR LIÉ À LA COAGULATION SUR LE GÉNOME DU FOIE
专利权人:
Jichi Medical University
发明人:
OHMORI Tsukasa,NAGAO Yasumitsu,MIZUKAMI Hiroaki,SAKATA Asuka,OZAWA Keiya,MURAMATSU Shin-ichi,TOMINAGA Shin-ichi,HANAZONO Yutaka,NISHIMURA Satoshi,SAKATA Yoichi,KAMOSHITA Nobuhiko
申请号:
EP18738823
公开号:
EP3569708A4
申请日:
2018.01.09
申请国别(地区):
EP
年份:
2020
代理人:
摘要:
The present invention provides a recombinant adeno-associated virus (rAAV) vector for treating a blood coagulation-related disease to provide a novel gene therapy means for hemophilia. The virus vector comprises a virus genome comprising a liver-specific promoter sequence and a polynucleotide sequence encoding a genome editing means operably linked to the promoter sequence, wherein the genome editing means is (a) a means comprising CRISPR/Cas9 composed of a Cas9 protein and a guide RNA (gRNA) and a repair gene, or (b) a means comprising CRISPR/Cas9 composed of a Cas9 protein and a gRNA, and the gRNA comprises a nucleotide region complementary to a part of a region related to expression of a disease-related protein on the genome of a patient and a region that interacts with the Cas9 protein.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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