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Gene-therapy vectors for treating cardiomyopathy
专利权人:
UNIVERSITÄTSKLINIKUM HAMBURG-EPPENDORF;RUPRECHT-KARLS-UNIVERSITÄT HEIDELBERG;Sorbonne Université;ASSOCIATION INSTITUT DE MYOLOGIE
发明人:
Lucie Carrier,Thomas Eschenhagen,Thomas Voit,Giulia Mearini,Oliver Mueller,Doreen Stimpel,Julia Mourot-Filiatre
申请号:
US14785188
公开号:
US10501756B2
申请日:
2014.04.17
申请国别(地区):
US
年份:
2019
代理人:
摘要:
The present invention relates to a gene therapy vector which is useful in the treatment or prevention of hypertrophic cardiomyopathy in a subject in need thereof. The gene therapy vector of the invention comprises a nucleic acid sequence encoding a cardiac sarcomeric protein and a cardiomyocyte-specific promoter which is operably linked to said nucleic acid sequence. The invention furthermore relates to a cell which comprises the gene therapy vector. Pharmaceutical compositions which comprise the gene therapy vector and/or a cell comprising said vector are also provided. In another aspect, the invention relates to a method for treating or preventing hypertrophic cardiomyopathy in a subject by introducing the gene therapy vector of the invention into a subject in need of treatment.
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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