Abstract
In the past, many researchers considered viral vectors to be the most promising candidates to transfer genetic material into the corpora for the treatment of erectile dysfunction. However, at present, no viral vectors have progressed to human trials. In contrast, the use of naked gene therapy, a plasmid expressing the human Maxi-K potassium channel, is the only gene therapy treatment to be evaluated in clinical phase I trials to date. The success of these studies, proving the safety of this treatment, has paved the way for the development of future gene transfer techniques based on similar transfer methods, as well as novel treatment vectors, such as stem cell transfer.
Highlights
Human gene therapy is a novel, promising step towards the prevention and treatment of disease[1]
The target gene is packaged into a viral vector so that the gene can survive without enzymatic breakdown in the body fluids and cytoplasm of the target cells, and be persistently expressed from the nucleoplasm[2,3,4]
Smooth muscle disorders of the genitourinary system, such as overactive bladder (OAB) syndrome and erectile dysfunction (ED), share important differences from malignancies or extensive genetic disorders that make them attractive targets for gene therapy and, in particular, allow the use of naked DNA rather than viral vectors to effect cell membrane penetration with the gene of interest
Summary
Human gene therapy is a novel, promising step towards the prevention and treatment of disease[1]. Smooth muscle disorders of the genitourinary system, such as overactive bladder (OAB) syndrome and erectile dysfunction (ED), share important differences from malignancies or extensive genetic disorders that make them attractive targets for gene therapy and, in particular, allow the use of naked DNA rather than viral vectors to effect cell membrane penetration with the gene of interest.
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