Vascular complications and gene therapy

Abstract

For gene therapy, the last few years have been an exciting period. Encouraging results from several successful gene therapy trials were reported. Children born with a life-threatening immune system disorder, severe combined immune deficiency (SCID), were cured after receiving gene therapy for replacement of their defective adenosine deaminase (ADA) gene. Gene therapy successes related to vascular complications were also reported. The first human gene therapy trial for a blood-vessel disorder was performed successfully, in which copies of an angiogenic gene, the vascular endothelial growth factor (VEGF) gene, were directly delivered to the area surrounding the diseased artery of the leg of a patient with peripheral artery disease. Within a few days, this stimulated the growth of new blood vessels around the blockage in the ailing blood vessel and helped avoid amputation. In 1998, a patient with genetically small arteries became the first to receive VEGF gene therapy in the heart. Multiple copies of a plasmid with the VEGF gene were delivered into the damaged area of the heart, and a few days later angiogenesis ensued that helped bypass the blocked vessel, with markedly reduced chest pain in the patient. Gene therapy is becoming a reality and, more importantly, it appears to be safe and does not require supplementary immuno-suppressing drugs. Gene therapy seems to have begun delivering on its promises.