Gene Therapy and Stem Cells in the Treatment of Congenital Diseases

Several congenital diseases are particularly attractive candidates for intervention using gene therapy since the underlying molecular bases for most of the monogenic disorders are well-understood. Transplantation of ex vivo genetically modified stem cells has also shown promise. Although all of these systems are meritorious and worthy of continued investigation, this mini-review article focused on the platforms that have received the most attention and that are maturing in the clinical setting; in particular, the potential of in vivo gene therapy and human-induced pluripotent stem cells. Studies of apparently disparate diseases that are presumably linked through shared metabolic pathways are likely to provide greater insights into the biology of the diseases. This and other opportunities for exchange will hopefully foster acceleration in the development of new and innovative therapies for these devastating diseases.