Advances in Gene Therapy for Erectile Dysfunction: Promises and Challenges

Curr Gene Ther. 2018;18(6):351-365. doi: 10.2174/1566523218666181004145424.


Erectile Dysfunction (ED) is one of the most common conditions affecting middle-aged and older men. Over the past few decades, oral phosphodiesterase type 5 inhibitors have been used to treat ED. However, these oral medications require on-demand access and are not effective in some hard-totreat populations. Moreover, there are no effective alternative treatments for ED. Based on results from preclinical models designed to provide long-term improvement of ED and other related conditions, gene therapy has shown great potential as a novel therapy for clinical management of ED. Gene therapy refers to the use of viral and non-viral vectors to deliver therapeutic genes to tissues via direct or transduced cell-mediated approaches. With the growing knowledge on the molecular mechanisms involved in the pathophysiology of ED, a number of therapeutic gene strategies have been extensively tested and proven to be effective in many animal models. However, only a few of them have been evaluated in clinical trials. This is due to safety concerns that need to be addressed before the clinical application can be considered. In this review, we summarize the key advancements in gene therapy for ED treatment, with an emphasis on the emerging stem cell-based approaches as well as other combinational strategies. The challenges facing the clinical application of gene therapy for ED treatment are also discussed.

Keywords: Erectile dysfunction; cell-based therapy; erection; gene delivery; gene therapy; stem cells..

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Erectile Dysfunction / therapy*
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Humans
  • Male
  • Molecular Targeted Therapy / methods
  • Nerve Growth Factors / genetics
  • Nerve Growth Factors / metabolism
  • Nitric Oxide Synthase Type I / genetics
  • Nitric Oxide Synthase Type II / genetics
  • Nitric Oxide Synthase Type III / genetics
  • Peptides / genetics
  • Potassium Channels / genetics


  • Nerve Growth Factors
  • Peptides
  • Potassium Channels
  • NOS1 protein, human
  • NOS2 protein, human
  • NOS3 protein, human
  • Nitric Oxide Synthase Type I
  • Nitric Oxide Synthase Type II
  • Nitric Oxide Synthase Type III