Universitätsklinikum Hamburg-Eppendorf (UKE);Ruprecht-Karls-Universität Heidelberg;Université Pierre et Marie Curie;Association Institut de Myologie
发明人:
申请号：
EP13198201.9
公开号：
EP2792742A1
申请日:
2013.12.18
申请国别(地区):
EP
年份:
2014
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.
