National Heart, Lung, and Blood Institute resources and programs for gene therapy in heart, lung, and blood diseases.

摘要

The advent of gene therapy holds great promise but the field is still in its infancy. Advances have been made but the translation of gene therapy from the bench to the clinical application has been slow. Relatively few clinical trials of gene therapy have been initiated for heart, lung, and blood disorders, and very few are currently ongoing.The future of gene therapy depends on our ability to translate basic research studies into clinical applications. These "translational studies" are expensive and complex, but necessary if we are to develop gene-based treatments that treat or prevent cardiovascular, lung and blood diseases. This article describes the National Heart, Lung, and Blood Institute's (NHLBI) current gene therapy programs which have been created to help support academic research efforts to successfully translate gene therapy into viable clinical applications.