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Could cells taken from a small patch of skin become heart muscle and repair a rare congenital heart defect? It's a scientific question that Timothy Nelson, M.D., Ph.D., and collaborators hope to answer for those born with an underdeveloped left heart chamber — a rare, complex condition known as hypoplastic left heart syndrome (HLHS).
Discovering the first cell-based therapy to rebuild heart tissue for HLHS has been a decade-long research passion for Dr. Nelson, who is the director of the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome at Mayo Clinic.
"The problem is that the majority of complex single ventricle patients born with congenital heart defects, like HLHS, are going to eventually develop heart failure. Unfortunately, heart transplantation is less than ideal for this patient population, that can include children with complex comorbidities, even if there wasn’t a severe shortage of suitable organs," says Dr. Nelson. "Mayo Clinic research plays an important role in advancing innovative discoveries for rare and complex heart conditions to expand options."
Approximately 10,000 people in the U.S. are living with HLHS — a condition that severely weakens the heart. Surgery to reposition arteries enables a single ventricle to pump blood to the lungs and the rest of the body, but that restores only a portion of circulatory function. While advancements are being made, few people with HLHS live past the age of 30.
"HLHS is essentially like taking your lawn mower engine and putting it into your SUV and driving to work at 70 miles an hour every day," says Dr. Nelson. "Our research is trying to understand if we can convert defective hearts from a 5-horsepower engine to a 50-horsepower engine. We probably will never make it into the 100-horsepower engine that a normal heart is, but our goal is to make the heart bigger and stronger."
Read the rest of the article on the Center for Regenerative Biotherapeutics blog.
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