Gene therapy in heart and lung disease

Abstract

Purpose of review: Gene therapy utilizes viral and non-viral vectors to transfer genetic material into a host in the hope of treating disease. This article will review the potential applications of gene therapy in the treatment of cardiac and pulmonary diseases.

Recent findings: The results from several phase I and II clinical trials have recently been published. In patients with ischemic heart disease, evidence of coronary revascularization has been observed after the delivery of angiogenic factors. Several trials have demonstrated a reduction in anginal symptoms, increases in exercise tolerance, and objective improvements in myocardial perfusion. Evidence of the transfer of therapeutic genes has been observed in human trials of inherited pulmonary diseases. Unfortunately, there has been little evidence of clinical efficacy in these studies. A variety of gene therapy strategies are being explored in the treatment of thoracic malignancies. Partial antitumor responses have occurred in some of the subjects enrolled in these studies.

Summary: Significant progress has been made in the field of gene therapy in the past decade. Data from these early animal and human clinical trials will provide important information to guide future studies.