Gene therapy of surfactant protein B deficiency

Abstract

Surfactant protein B (SP-B) is encoded by a single gene, and the mature protein is expressed by alveolar type II epithelial (ATII) cells of the lungs. Studies in transgenic mice and its hereditary deficiency in humans have established its indispensable role in postnatal survival. An established gene therapy regime for treating SP-B deficiency could overcome the limitations of surfactant replacement therapies or lung transplantation. Among the various viral and non-viral gene delivery tools available, only adenoviral vectors have been tested for delivering SP-B cDNA to the lungs of animal models. This review discusses the various vectors that are available for delivering therapeutic genes into ATII cells.