Among the experts presenting are physicians and scientists from the UK Chandler Medical Center, the Institute for Human Gene Therapy at the University of Pennsylvania School of Medicine, the Centers for Disease Control and Prevention, and the University of North Carolina at Chapel Hill Hospital and School of Medicine.
Gene therapy aims to treat a disease by modifying the action of a person's genes.
The goal of gene therapy with genetic diseases, which result from defective, or mutated, genes, is to deliver a correct copy of the mutated gene directly to the nucleus of the abnormal cells, thereby restoring normal function of the gene. Many other types of diseases that are not strictly genetic diseases currently are being investigated for gene therapy, including cardiovascular and infectious diseases. In these instances, gene therapy aims to insert genes that control or affect the action of the disease-causing genes. To insert the corrective gene into the appropriate cells, the gene must be placed in a delivery vehicle, or vector. The corrective gene can be encapsulated in liposomes, small balls made of molecules that resemble cell membranes, or viruses.