Cardiovascular gene therapy

• Published in: The Asia Pacific Heart Journal 1998, Vol.7 (2), p.114-120
• Main Authors: Li, Zhihe, Grove, Robert I.
• Format: Article
• Language: English
• ISSN: 1328-0163
• DOI: 10.1016/S1328-0163(98)90008-2

Gene therapy involves the transfer of therapeutic genes to cells in vivo and is therefore a potentially novel way to treat cardiovascular diseases. Especially good candidate diseases are those arising from inherited mutations in the genetic sequence and which are intransigent to current therapies. An example of an inherited disease is familial hypercholesterolaemia, which arises from a point mutation in the low-density lipoprotein receptor gene and for which effective therapeutics are lacking. Other diseases, like occlusive vascular disease, may be caused by abnormal cellular or tissue responses and may also be good candidates for gene therapy. A number of preclinical efforts have begun to culminate in clinical studies for conditions like hypercholesterolaemia and occlusive vascular disease. Other cardiovascular indications are under investigation, with late stage preclinical studies focusing on restenosis, transplant atherosclerosis and rejection, and a number of lipoprotein abnormalities.