Clinical trials in vascular gene therapy: The missing piece of the puzzle

Objective. - Vascular gene transfer provides unique opportunities for novel therapies for cardiovascular disease. To define the limitations and expectations of translating preclinical strategies to clinical therapy, the kinetics and extent of vascular transgene expression must be determined. Data Sources. - Published clinical and preclinical studies of vascular gene transfer. Data Extraction. - Kinetics and extent of vascular transgene expression. Data Synthesis. - In spite of ongoing clinical studies in vascular gene transfer, data regarding the kinetics and extent of transgene expression are very limited. Conclusions. - For the efficient translation of preclinical to clinical vascular gene transfer studies, the kinetics and extent of transgene expression should be determined. Novel imaging technologies and unique clinical scenarios for obtaining tissue should be considered.