Using gene therapy to alter levels of specific proteins in a targeted fashion is an intermediary advance in medicine for age-related conditions. Practical applications of this technology should provide an improvement over what came before, but at the same time they don't treat the underlying issue, which is the accumulation of cellular and molecular damage that causes aging and the diseases of aging. At some point the medical community must change its focus from patching over the problem to addressing its root causes.
[Researchers] have successfully tested a powerful gene therapy, delivered directly into the heart, to reverse heart failure in large animal models. The new research study findings [are] the final study phase before human clinical trials can begin testing SUMO-1 gene therapy. SUMO-1 is a gene that is "missing in action" in heart failure patients.
[In an earlier trial] a gene known as SERCA2 is delivered via an inert virus - a modified virus without infectious particles. SERCA2 is a gene that produces an enzyme critical to the proper pumping of calcium out of cells. In heart failure, SERCA2 is dysfunctional, forcing the heart to work harder and in the process, to grow larger. The virus carrying SERCA2 is delivered through the coronary arteries into the heart during a cardiac catheterization procedure. Studies show only a one-time gene therapy dose is needed to restore healthy SERCA2a gene production of its beneficial enzyme.
But previous research [discovered] SERCA2 is not the only enzyme that is missing in action in heart failure. [The] SUMO-1 gene is also decreased in failing human hearts. But SUMO-1 regulates SERCA2a's activity, suggesting that it can enhance the function of SERCA2a without altering its levels. A follow-up study in a mouse model of heart failure demonstrated that SUMO-1 gene therapy substantially improved cardiac function. This new study tested delivery of SUMO-1 gene therapy alone, SERCA2 gene therapy alone, and a combination of SUMO-1 and SERCA2.
In large animal models of heart failure, the researchers found that gene therapy delivery of high dose SUMO-1 alone, as well as SUMO-1 and SERCA2 together, result in stronger heart contractions, better blood flow, and reduced heart volumes, compared to just SERCA2 gene therapy alone.