This AAV gene therapy transduces vascular endothelial cells and targets complications associated with diabetes and other vascular diseases. Available gene therapy vectors do not efficiently or selectively transduce vascular endothelial cells, which are associated with vascular diseases and disorders such as diabetes, diabetic retinopathy, blood clotting, aneurysms, and varicose veins. Diabetes alone affects 29 million Americans, and 40 percent of those with diabetes develop a stage of diabetic retinopathy. Researchers at the University of Florida have developed an AAV- based system that can selectively transduce vascular endothelia cells and will offer many more gene therapy treatment options for vascular diseases and disorders. The AAV vector relies on the use of tissue-specific promoters to efficiently drive gene expression in vascular endothelia cells and target vascular diseases, a problem that affects a large segment of the population.
Gene therapy targeting vascular endothelial cells
This gene therapy relies on the use of tissue-specific promoters that when combined with a specific AAV capsid mutant can efficiently drive gene expression in vascular endothelial cells. University of Florida researchers derived the synthetic vascular endothelial cells-targeting promoters from the human VE-cadherin gene control region and combined them with other regulatory sequences. When used in combination, the tissue-specific promoters efficiently and selectively transduce vascular endothelial cells. Vascular endothelial cells play a pivotal role in the pathophysiology of a multitude of diseases. Endothelial impairment or dysfunction is a hallmark of coronary artery disease, diabetes mellitus, hypertension, hypercholesterolemia and atherosclerosis. The ability to selectively express protein coding DNA, miRNAs, or other genetic components will jumpstart the development of interventional approaches for these diseases.
