Gene Therapy may Treat Heart Failure


A heart transplant is the best current treatment for heart failure. However, the limited number of hearts available for transplant mandates the need for alternative treatments.

‘Novel promising gene therapy helps in treating heart failure in pig models. This holds a potentially transformational strategy to treat human heart failure using gene therapy through further human clinical trials.


“One of the interests of my lab is to develop ways to heal heart muscle by studying pathways involved in heart development and regeneration,” says corresponding author Dr. James Martin, professor, and Vivian L. Smith Chair in Regenerative Medicine at Baylor and director of the Cardiomyocyte Renewal Lab at the Texas Heart Institute.

Gene Therapy in Heart Failure

Earlier studies have shown that there is an increase in the activity of the Hippo signaling pathway (inhibits heart repair) during a heart failure. Turning this pathway off in the mouse model revealed recovery of the heart’s pumping function.

The study team created a pig model to assess the effect of gene therapy by knocking down the Hippo signaling pathway in a heart attack. Since the pig’s heart is closely associated with the human heart, the study may yield promising results for gene therapy in treating human heart failure.

“We were excited by the results. Three months after we treated the pigs, those that received the gene therapy had improved their heart function, showed signs that their cardiomyocytes (heart muscles) were regenerating, had less fibrosis or scarring and had evidence that new blood vessels had formed. The procedure was shown to be safe since the pigs tolerated the therapy very well,” says first author Dr. Shijie Liu, a postdoctoral associate in the Martin lab.

Thus these findings hold a potentially transformational strategy to treat human heart failure using gene therapy. The team anticipates next bring the therapy to human clinical trials for further benefits.


Source: Medindia



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