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Gene Therapy

Heart Failure Treated with Gene Therapy

16 years ago

2147  0
Posted on Dec 07, 2003, 2 a.m. By Bill Freeman

Researchers at the University of California at San Diego have used gene therapy to stop the progression of heart failure in hamsters. The treatment uses a virus called an adeno-associated virus (AAV) to deliver a gene called phospholamban (PLN) into the heart. Previous research by the authors revealed that PLN malfunctions in people with heart failure, thus the theory was that the new version of PLN would replace the defective gene and stop the heart from losing anymore of its pumping power.

Researchers at the University of California at San Diego have used gene therapy to stop the progression of heart failure in hamsters. The treatment uses a virus called an adeno-associated virus (AAV) to deliver a gene called phospholamban (PLN) into the heart. Previous research by the authors revealed that PLN malfunctions in people with heart failure, thus the theory was that the new version of PLN would replace the defective gene and stop the heart from losing anymore of its pumping power. Study results revealed that the therapy stopped the disease from progressing and significantly improved the heart's ability to both pump and contract. While the study was conducted on hamsters, study leader Dr Kenneth R Chien is confident that it could be used to treat humans suffering from advanced heart failure, which is currently untreatable. The researchers hope to begin human trials of the technique "within 18 months or so."

SOURCE/REFERENCE: Nature Medicine 2002; 10.1038/nm739

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