Association Institut de Myologie;Universitatsklinikum Hamburg-Eppendorf;Universite Pierre et Marie Curie;Ruprecht-Karls-Universitat Heidelberg
发明人:
Carrier, Lucie,Eschenhagen, Thomas,Voit, Thomas,Mearini, Giulia,Mueller, Oliver,Stimpel, Doreen,Mourot-Filiatre, Julia
申请号：
AU2014255655
公开号：
AU2014255655A1
申请日:
2014.04.17
申请国别(地区):
AU
年份:
2015
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.
