Hereditary diseases: Cancer drug fights muscular dystrophy

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Hereditary diseases: Cancer drug fights muscular dystrophy
Hereditary diseases: Cancer drug fights muscular dystrophy

Cancer drug fights muscular dystrophy

An anticancer agent also h alts the typical progressive breakdown of muscle cells and fibers that characterizes muscular dystrophy. Scientists at the Dulbecco Telethon Institute in Rome hope that the previously inevitably fatal course of this incurable hereditary disease could be slowed down with the drug.

The scientists had treated mice suffering from Duchenne muscular dystrophy with histone deacetylase inhibitors. These active ingredients are already being used as drugs to treat breast cancer. Under the influence of the enzyme inhibitors, the skeletal muscle mass of the affected rodents regenerated. The regenerated muscle cells were also less likely to degenerate due to contraction, which is the main cause of muscle wasting in patients. Treated animals with muscular dystrophy were able to perform physical exercises almost as well as he althy conspecifics, the researchers report.

The results are very promising, says Puri, but still require unpredictably long and intensive studies before use on humans can be considered. First of all, the scientists want to clarify how different deacetylase inhibitors affect muscle regeneration. Puri's team has been working on a therapy against the dystrophy for ten years and it became aware that the now promisingly regulated acetylases and acyltransferases can activate and block genes that play a role in cell regeneration.

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