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CHS Research Shows Promising Gene Therapy Surgery
June 13, 2012

A team of researchers at Carolinas HealthCare System’s Sanger Heart & Vascular Institute were recently recognized for their outstanding research breakthrough which includes results that have never been reported elsewhere. Their abstract was one of only six selected internationally for an “Excellence in Research Award”. This honor, awarded by The American Society of Gene and Cell Therapy, is given to the highest ranking papers submitted by PhD or junior researchers out of approximately 1,000 abstracts. The abstract is titled “Molecular Cardiac Surgery Mediated ssAAV1-SERCA2a Gene Therapy Restores Global and Regional Contractility in Ovine Ischemic Cardiomyopathy” and was recently published in Molecular Therapy.

The gene and stem cell therapy research is conducted in the NIH-funded research laboratory of Charles R. Bridges, MD, ScD, Chair of the Thoracic and Cardiovascular Surgery Department. The award was received by Anthony Fargnoli MS, Dr. Bridges’ Research Manager and Ph.D. candidate in the Department of Bioengineering at the University of Pennsylvania. Dr. Bridges is also an adjunct Professor of Surgery/Bioengineering at the University of Pennsylvania and serves as Anthony’s Ph.D. thesis supervisor. Michael G. Katz, MD, PhD a research scientist in the Bridges laboratory was also a coauthor.

The concept of gene therapy looks at using the body’s own proteins or DNA to treat itself, targeting the root cause of the disease. The award highlights a surgical technique developed by Dr. Bridges that is more efficient at delivering therapeutic DNA sequences to the heart of large animals than any other method currently available in the world. Dr. Bridges’ team has pioneered the use of this technique in animal models of heart failure to reverse the disease – something that has had only limited success with other research teams in the past. The key problem the group has solved is getting enough DNA into the heart to maximize its effect, which is done through a state of the art patented molecular cardiac surgery with recirculating delivery technology (MCARDTM). Dr. Bridges was recently awarded another U.S. patent for the application of MCARDTM to cardiac stem cell delivery.

Heart attacks damage or overstress an individual’s heart cells, which cannot be corrected, and leads to heart failure. Heart failure drugs, devices and therapies aid in treating the disease, but cannot cure it. A heart transplant is the standard of care; however the number of hearts does not meet the demand. With gene therapy, a copy of the DNA is transferred into the cells with an engineered non-infectious virus. The DNA remains after a heart attack to re-program cells to restore a lost protein that essentially fixes the root cause of the problem and increases the strength of heart contraction.

In order for gene therapy to work optimally; however, the cells must only be injected in the organ that needs to be healed. MCARDTM, by isolating the heart temporarily, is the only proven method to do so effectively. In the future, molecular cardiac surgery may be conducted minimally invasively or on patients who have heart failure and need to undergo a cardiac surgical procedure such as valve repair or replacement.
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