Gene and cell-based therapies for heart disease.
Heart disease remains the prevalent cause of premature death and accounts for a significant proportion of all hospital admissions. Recent developments in understanding the molecular mechanisms of myocardial disease have led to the identification of new therapeutic targets, and the availability of vectors with enhanced myocardial tropism offers the opportunity for the design of gene therapies for both protection and rescue of the myocardium. Genetic therapies have been devised to treat complex diseases such as myocardial ischemia, heart failure, and inherited myopathies in various animal models. Some of these experimental therapies have made a successful transition to clinical trial and are being considered for use in human patients. The recent isolation of endothelial and cardiomyocyte precursor cells from adult bone marrow may permit the design of strategies for repair of the damaged heart. Cell-based therapies may have potential application in neovascularization and regeneration of ischemic and infarcted myocardium, in blood vessel reconstruction, and in bioengineering of artificial organs and prostheses. We expect that advances in the field will lead to the development of safer and more efficient vectors. The advent of genomic screening technology should allow the identification of novel therapeutic targets and facilitate the detection of disease-causing polymorphisms that may lead to the design of individualized gene and cell-based therapies.
Melo
Luis G
LG
Department of Physiology, Queen's University, Kingston, Ontario K7L 3N6, Canada. melol@post.queensu.ca
Pachori
Alok S
AS
Kong
Deling
D
Gnecchi
Massimiliano
M
Wang
Kai
K
Pratt
Richard E
RE
Dzau
Victor J
VJ
D003324:Coronary Artery Disease / Q000628:therapy; D004730:Endothelium, Vascular / Q000166:cytology; D015316:Genetic Therapy; D006330:Heart Defects, Congenital / Q000628:therapy; D006331:Heart Diseases / Q000378:metabolism / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D009200:Myocardial Contraction; D017202:Myocardial Ischemia / Q000628:therapy; D009206:Myocardium / Q000378:metabolism; D033581:Stem Cell Transplantation; D023822:Tissue Engineering