Emerging gene and stem cell therapies for the treatment of erectile dysfunction.

Erectile dysfunction is a prevalent condition that leads to significant morbidity and distress, not just for affected men but also for their partners. Very few currently available treatments ameliorate the underlying causes of the disorder and 'cure' the disease state. Much recent effort has been focused on the development of gene and cell-based approaches to rectify the molecular and tissue defects responsible for ED. Gene therapy has been investigated in animal models as a means to restore normal function to the penis; at this time, however, only one human trial has been published in the peer-reviewed literature. Recent gene therapy studies have focused on the modulation of enzymes associated with the NOS/cGMP pathway, and supplementation of trophic factors, peptides and potassium channels. Stem cell therapy has been a topic of interest in more recent years but there are currently very few published reports in animal models and none in human men. Although stem cell therapy offers the potential for restoration of functional tissues, legitimate concerns remain regarding the long-term fate of stem cells. The long-term safety of both gene and stem cell therapy must be thoroughly investigated before large-scale human studies can be considered.