Thanks to the manipulation of a protein, American researchers have managed to curb muscular dystrophy in the laboratory, suppressing the death of myofibers and improving muscle function.
US scientists have discovered a method capable of slow down the progression from the Duchenne muscular dystrophy (DMD) and to improve the muscle function. Forced activation of a protein signaling of the immune system, linked to cell survival, growth and inflammation, is in fact able to promote the growth of muscle fibers e you suppress myonecrosisthe process leading to the muscle wasting typical of serious medical condition. At the moment the effectiveness of protein manipulation – called SO 1 – has only been demonstrated in the laboratory through experiments on mouse models (topi), however there are significant hopes that it could also be replicated in humans.
A US research team led by scientists at the University of Houston College of Pharmacy has discovered that manipulating an immune protein can slow the progression of Duchenne muscular dystrophy and improve muscle function. The same team had previously discovered that the transforming growth factor β-activated kinase1 (TAK 1) protein is critical for the regulation of skeletal muscle massalso had determined that its activation beyond normal levels can improve skeletal muscle growth, as noted in a Texas University press release. In the new study, involving genetically modified mice affected by the mouse model of muscular dystrophy, researchers led by Professor Ashok Kumar altered the levels of TAK 1 to verify its impact on the musculature.
Scientists found that inactivation of the TAK1 protein resulted in decreased muscle mass and muscle contractile function, as well as loss of muscle mass in adult mice. Forced activation, on the other hand, catalyzes the growth of myofibers, “without having any deleterious effect on muscle histopathology,” the scientists explain in the study abstract. “Our results suggest that TAK1 is a regulator of skeletal muscle mass. By specifically targeting this protein, we can suppress the death of muscle fibers, known as myonecrosis, and slow disease progression in DMD,” Professors Ashok Kumar, Else and Philip Hargrove said in a press release. “Our research shows that TAK1 activation can stimulate myofiber growth in a model of DMD, with no negative impact on muscle health,” the experts added.
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As explained by Orpha.net, Duchenne and Becker muscular dystrophy “are neuromuscular disorders characterized by progressive muscle wasting and weakness from degeneration of skeletal, smooth and cardiac muscles”. Of the two, the more serious and frequent is the first, which strikes about 1 child for every 3,300. It is usually diagnosed at age 5 and patients die in their 30s. Progressive and constant muscle wasting, in fact, leads first to paralysis and finally to death from respiratory failure. The hope is that this discovery can be translated into innovative treatments capable of effectively counteracting the ominous evolution of the rare pathology. “Our results suggest that TAK1 is a positive regulator of skeletal muscle mass and that targeted regulation of TAK1 can suppress myonecrosis and improve disease progression in DMD,” commented Professor Kumar and colleagues. The details of the research “Targeted regulation of TAK1 counteracts dystrophinopathy in a DMD mouse model” have been published in JCI insight.