Gene therapy as future treatment of erectile dysfunction.

IMPORTANCE OF THE FIELD: Erectile dysfunction (ED) is a major men's health problem. Although the high success rate of treating ED by phosphodiesterase 5 (PDE5) inhibitors has been reported, there are a significant number of ED patients who do not respond to currently available treatment modalities. AREAS COVERED IN THIS REVIEW: To elucidate the current status of gene therapy applications for ED, gene therapy approaches for ED treatment are reviewed. WHAT THE READER WILL GAIN: Gene therapy strategies that can enhance nitric oxide (NO) production or NO-mediated signaling pathways, growth factor-mediated nerve regeneration or K(+) channel activity in the smooth muscle could be promising approaches for the treatment of ED. Although the majority of gene therapy studies are still in the preclinical phase, the first clinical trial using non-viral gene transfer of Ca(2+)-activated, large-conductance K(+) channels into the corpus cavernosum of ED patients showed positive results. TAKE HOME MESSAGE: Gene therapy represents an exciting future treatment option for ED, especially for people with severe ED unresponsive to current first-line therapies such as PDE5 inhibitors although the long-term safety of both viral and non-viral gene therapies should be established.