Adventure of gene therapy into the brain: A new era for cardiovascular gene therapy.

Gene therapy is emerging as a potential strategy for the treatment of cardiovascular diseases such as peripheral arterial disease, myocardial infarction, restenosis after angioplasty, and vascular bypass graft occlusion, for which current therapy is often inadequate. The first federally approved human gene therapy protocol started on September 14, 1990, in patients with adenosine deaminase deficiency. Ten years after the commencement of the first trial, more than 30 clinical studies of gene therapy for cardiovascular disease are under investigation. First, Isner and colleagues demonstrated the potential utility of gene therapy using an angiogenic growth factor (vascular endothelial growth factor [VEGF]) for the treatment of critical limb ischemia in human patients.1,2 More recently, his group revealed the usefulness of gene therapy using VEGF to treat ischemic heart disease.3,4 Although there are still many unresolved issues, human gene therapy for cardiovascular disease is now becoming a reality.