Regenerative Gene therapy

Gene therapy can be defined as the transfer of nucleic acids to the somatic cells of an individual with a resulting therapeutic effect. Gene therapy has already been used to correct genetic defects or to express gene products that are therapeutically useful. A strong advantage of gene therapy is the possibility that a single local administration can give a long-lasting therapeutic effect in the target tissue. One of the most promising areas of gene therapy for cardiovascular diseases is therapeutic angiogenesis, the stimulation of growth of new blood vessels. Several clinical gene therapy trials have previously been performed to study the efficacy and safety of gene therapy in the treatment of vascular diseases.

The main pathophysiology of atherosclerosis includes endothelial dysfunction, inflammation, vascular proliferation and matrix alteration. These molecular processes can be used as a basis for gene therapy. Gene transfer for therapeutic angiogenesis may provide a novel treatment option for patients that do not benefit from traditional therapies. The concept is that angiogenic gene therapy with an AdVEGF-D construct can ameliorate the heart ischemia by significantly increasing capillary density and blood flow as has been demonstrated in animal models.