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VEGF-C C156S AAV is a preclinical gene therapy construct consisting of an adeno-associated viral (AAV) vector encoding VEGF-C C156S, a mutant form of human vascular endothelial growth factor C (VEGF-C) in which cysteine 156 is substituted with serine. The active ingredient, VEGF-C C156S, is a lymphangiogenesis-specific variant of VEGF-C that selectively targets the receptor VEGFR-3 (vascular endothelial growth factor receptor 3), which is expressed on lymphatic endothelial cells. Unlike wild-type VEGF-C, the C156S mutation eliminates potential angiogenic (blood vessel-forming) side effects by reducing affinity for VEGFR-2, making it more selective for lymphatic vessel growth. The AAV delivery vehicle enables sustained expression of the transgene in vivo. This construct has been investigated in preclinical models for the treatment of lymphedema and for cardiac repair following myocardial infarction, where it has been shown to stimulate cardiac lymphangiogenesis and reduce inflammation. It is also used as a research tool to study the progression of non-alcoholic steatohepatitis (NASH) and hepatocellular carcinoma (HCC).
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