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GENE-THERAPY VECTORS FOR TREATING CARDIOMYOPATHY
专利权人:
ASSOCIATION INSTITUT DE MYOLOGIE;Sorbonne Université;RUPRECHT-KARLS-UNIVERSITÄT HEIDELBERG;UNIVERSITÄTSKLINIKUM HAMBURG-EPPENDORF
发明人:
Lucie Carrier,Thomas Eschenhagen,Thomas Voit,Giulia Mearini,Oliver Mueller,Doreen Stimpel,Julia Mourot-Filiatre
申请号:
US16707223
公开号:
US20200095609A1
申请日:
2019.12.09
申请国别(地区):
US
年份:
2020
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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