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New gene therapy shows promising results in reversing heart failure

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Researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai, have made remarkable progress in the treatment of heart failure. In their experiments on laboratory animals, they were able to reverse heart failure using gene therapy injected into the heart. SUMO -1 gene therapy may soon be tested on humans as the results of the studies so far showed promising outcomes.

Heart failure is a leading cause of hospitalization in older people; according to statistics, it is responsible for approximately 300,000 deaths each year in the United States. Heart failure means that the heart can no longer send blood so as to meet the needs of the body. Heart failure is the final stage of many cardiovascular diseases such as heart valve disease ( mitral stenosis , aortic insufficiency ), hypertension, myocardial infarction, etc.

There are several signs and symptoms of heart failure such as dyspnea, fatigue, ankle edema, ascites, hepatomegaly, etc. The main symptom and perhaps the most troubling to the patient is dyspnea or intolerance to exercise, because the heart cannot supply enough blood ( and therefore oxygen) when the patient makes an effort. Dyspnea that occurs at little effort means an advanced stage of heart failure.


Of course, there are drugs that improve cardiac function such as beta -blockers, diuretics, angiotensin converting enzyme inhibitors, calcium blockers, antiplatelet agents, etc. . It is worth mentioning that these drugs control some of the symptoms of heart failure but does not cure the disease; in addition, the treatment should aim to heart failure etiology: mitral stenosis, ischemic heart disease , aortic insufficiency, etc.

The study ‘s senior investigator Roger J. Hajjar, MD, Director of the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai, said that SUMO -1 may be the first gene therapy treatment that shrinks significantly the enlarged heart and improves cardiac function. In fact, this is actually the second gene therapy to treat heart failure developed by Dr. Hajjar and his Icahn at the Cardiovascular Research Center at Mount Sinai School of Medicine. CUPID, as it is called the first trial, had promising results and is now in the final testing phase of the SERCA 2 gene therapy. In heart failure, SERCA 2, a gene involved in myocardial fiber contraction is dysfunctional, and therefore researchers have thought to deliver this gene with an inactivated virus; the virus is deliver through coronary artery during cardiac catheterization.

But researchers found that not only SERCA2 is decreased in heart failure but also SUMO -1 gene. It seems that SUMO -1 gene enhances SERCA2 gene function, therefore the researchers combined the two therapies and compared the results. It seems that, compared to SERCA2 gene therapy alone, high dose SUMO -1 alone, as well as SUMO -1 and SERCA2 together, led to stronger heart contractions and better blood flow in the heart. “These new study findings support the critical role SUMO-1 plays for SERCA2 function, and underlie the therapeutic potential of SUMO-1 gene replacement therapy for heart failure patients,”  researchers said.