Approaches to gene therapy of complex multigenic diseases: cancer as a model and implications for cardiovascular disease and diabetes.
The general concept of gene therapy is now well established and accepted by the medical, scientific and public policy communities, and is rapidly being implemented in human experimental studies. In addition to the initial models of single gene defects, target diseases have now come to include multigenic and multifactorial diseases such as human cancer, neurodegenerative diseases such as Parkinson's disease and firms of cardiovascular disease. While many conceptual and technical obstacles must still be overcome before therapy for disorders such as coronary artery disease and diabetes mellitus will easily be approached at the genetic level, the early results with several multigenic disease models gives some cause for optimism that gene therapies for even those complicated disorders will eventually become available.
Friedmann
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Center for Molecular Genetics, UCSD School of Medicine 92093.
D000818:Animals; D002318:Cardiovascular Diseases / Q000628:therapy*; D003920:Diabetes Mellitus / Q000628:therapy*; D016153:Genes, Suppressor; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D007167:Immunotherapy; D051379:Mice; D008819:Mice, Nude; D005810:Multigene Family; D009369:Neoplasms / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D009374:Neoplasms, Experimental / Q000628:therapy; D013489:Suppression, Genetic; D014162:Transfection; D014407:Tumor Cells, Cultured