Advances in gene therapy for erectile dysfunction: promises and challenges

Erectile dysfunction (ED) is a major health problem in men. Over the past few decades, oral phosphodiesterase type 5 (PDE5) inhibitors have been adopted as the recommended strategy to treat ED in the majority of cases. However, these oral medications require on-demand access and are not effective in some hard-to-treat populations. Other alternative treatments are also not satisfactory. In recent years, a novel therapeutic approach against ED, gene therapy, has shown great potential in preclinical models designed to provide long-term improvement of erectile function and the underlying conditions. 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 of 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 few of them have been evaluated in clinical trials. Safety concerns are the major obstacles to be addressed before clinical application can be considered. In this review, we summarize the 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 its future application for ED treatment are also discussed.

Current gene therapy. 2018 Oct 04 [Epub ahead of print]

Botao Yu, Changjing Wu, Tao Li, Feng Qin, Jiuhong Yuan

Andrology Laboratory, West China Hospital, Sichuan University, Chengdu 610041. China.