Gene therapy treatments for erectile and bladder dysfunction

The postgenomic age presents many exciting challenges and opportunities for the application of molecular medicine to the treatment of lower urinary tract diseases. Chief among these are the therapeutic possibilities afforded to selectively modulate/alter gene expression in somatic cells to "normalize" aberrant cellular responses to the existing hormonal milieu. In this paper, this therapeutic strategy will be referred to as gene therapy or gene transfer. This article specifically reviews the potential use of gene therapy/transfer to the treatment of bladder and erectile dysfunction. Available treatments for both of these common urologic diseases/disorders have contraindications, untoward side effects, or limited efficacy in certain patient populations. Moreover, that genetic material can be locally administered in the bladder and penis removes a major therapeutic obstacle to the use of gene transfer. Thus far, "proof-of-concept" has been demonstrated in preclinical studies using cellular and molecular strategies on a variety of gene targets. The continuing evolution of gene transfer vectors and gene delivery technologies is expected to further enhance the selectivity, efficacy, and duration of gene therapy, making it a viable treatment option for the amelioration of lower urinary tract diseases/disorders.