Without gene therapy

Scientists reverse Duchenne muscular dystrophy in mice

Svetlana Maslova, Hi-tech+

Blocking a new therapeutic target led to a number of improvements in the condition of animals, preventing the development of the disease. Currently, Duchenne muscular dystrophy is incurable, begins to progress from the first years of a person's life and leads to early death, so the new results open up great prospects in the treatment of the disease.

An international group of researchers led by scientists from The Polytechnic School of Lausanne presented the first evidence of the connection between muscular dystrophy and sphingolipids, and then demonstrated the effectiveness of targeting them to reverse Duchenne muscular dystrophy, which accounts for about half of all cases of muscular dystrophy.

Article by Laurila et al. Inhibition of sphingolipid de novo synthesis counteracts muscular dystrophy is published in the journal Science Advances – VM.

With muscular dystrophy against the background of gene mutations, weakness and early destruction of skeletal muscles with subsequent concomitant problems are observed. The disease manifests itself from the first years of life, seriously disrupts its quality and causes early mortality.

Scientists have drawn attention to sphingolipids because of their alleged role in the transmission of signals between cells, as well as a number of symptoms of Duchenne muscular dystrophy.

Experiments on mouse models have shown that this is a true therapeutic target — blocking the synthesis of sphingolipids counteracted the loss of muscle function in rodents.

In addition, the sphingolipid inhibitor (myriocin) stabilized calcium homeostasis in muscles, reversed fibrosis in the diaphragm and heart muscle, reduced inflammation, transferring immune macrophages from a pro-inflammatory state to an anti-inflammatory one, the authors specified.

These first preliminary results open up new perspectives in the targeted therapy of Duchenne muscular dystrophy. Currently, only symptomatic support of the condition is available for patients, but it does not provide any significant improvements and does not slow down the progression of the disease. The average life expectancy with such a diagnosis today is about 25 years.

While all such studies of Duchenne muscular dystrophy are at the preclinical stage, however, each group of scientists finds its previously unknown mechanisms involved in the development of the disease. For example, the command from from The University of Houston managed to find a protein necessary for the regeneration of skeletal muscles, and scientists from University of Pennsylvania our way to improve the function and structure of muscle fibers.

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