Our overall goals include developing meaningful treatment for DMD boys at any stage of disease and one of the challenges to treat DMD is the accumulation of fibrosis or scar tissue as muscle loss progresses. We have previously demonstrated a way to replace the missing dystrophin gene with a miniature dystrophin using gene therapy. We have now combined that therapy with the delivery of microRNA-29. This is a naturally occurring molecule in the body that regulates several of the proteins that make up scar tissue. In DMD, microRNA-29 is abnormally reduced; thus it was our goal increase levels using gene therapy.
In a DMD disease model, we delivered both micro-dystrophin and microRNA-29 and the results showed reduced scar tissue and enhanced function of the muscle compared to either treatment alone. We plan to test this very promising combination therapy in future clinical trials.
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