Ion channel gene therapy for smooth muscle disorders: Relaxing smooth muscles to treat erectile dysfunction

Jonathan D. Schiff, Arnold Melman

Research output: Contribution to journalArticle

7 Scopus citations

Abstract

The promise of gene therapy to treat diseases remains largely unfulfilled. Past setbacks and the complexity of the delivery systems used, in terms of both targeting the appropriate cells and inducing expression of products at therapeutic levels, thus far have prevented significant success for gene therapy. Smooth muscle disorders represent a unique target for gene therapy. In many cases, smooth muscle is readily accessible and, to induce a therapeutic effect, will not require very high levels of gene product expression. This allows a lower efficiency of gene transfer to be successful. With these important features in mind, we believe that naked DNA transfer of potassium ion channels represents a novel and successful way to treat smooth muscle disorders. Herein, we present a rationale for treating erectile dysfunction, a smooth muscle disorder of the cavernosal bodies of the penis, with naked DNA gene transfer therapy. By inserting the hSlo gene, which codes for Maxi-potassium channels, into smooth muscle cells, we can improve smooth muscle relaxation in the corporal bodies and thus improve erectile function. This method of gene transfer has proven to be safe and effective for erectile dysfunction, and human trials are ongoing.

Original languageEnglish (US)
Pages (from-to)89-95
Number of pages7
JournalAssay and Drug Development Technologies
Volume4
Issue number1
DOIs
StatePublished - Feb 1 2006

ASJC Scopus subject areas

  • Molecular Medicine
  • Drug Discovery

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