Preliminary laboratory findings suggest that gene therapy may someday be able to reverse heart failure and thus obviate the need for transplantation.
The results support the premise that gene-based therapies and targeting of specific pathways in human heart failure may offer a new modality for its treatment, Roger J. Hajjar, MD, of Massachusetts General Hospital and colleagues reported on December 7 in Circulation (1999;100:2308-2311).
In the study, the researchers isolated muscle cells from ten failed hearts that had been removed for transplantation, and they then delivered extra copies of the SERCA2a gene to the heart muscle cells. The cells that incorporated the gene and produced elevated levels of its protein began to function normally and contracted more quickly and more powerfully. The cycling of calcium, which is vital for heart function, also appeared normal in cells incorporating the additional copies of SERCA2a.
Although previous research showed that increasing the production of SERCA2a improved heart function both in individual cells of animals and in living animals, it was not clear that the gene transfer techniques also would work in human heart cells, according to Massachusetts General Hospital.
The new results in isolated heart cells of humans "need to be validated in the whole human heart, but we're optimistic that this goal will be accomplished," said Dr. Hajjar. The investigators wrote in Circulation that they also need to determine whether the gene transfer therapy will offer long-term benefits.
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