Novel therapies for the treatment of cystic fibrosis: new developments in gene and stem cell therapy.

Cystic fibrosis (CF) was one of the first target diseases for lung gene therapy. Studies of lung gene transfer for CF have provided many insights into the necessary components of successful gene therapy for lung diseases. Many advancements have been achieved with promising results in vitro and in small animal models. However, studies in primate models and patients have been discouraging despite a large number of clinical trials. This reflects a number of obstacles to successful, sustained, and repeatable gene transfer in the lung. Cell-based therapy with embryonic stem cells and adult stem cells (bone marrow or cord blood), have been investigated recently and may provide a viable therapeutic approach in the future. In this article, the authors review CF pathophysiology with a focus on specific targets in the lung epithelium for gene transfer and summarize the current status and future directions of gene- and cell-based therapies.