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news.A new research, licensed by miRagen Therapeutics, has demonstrated an essential role for miR-133a in the maintenance of adult skeletal muscle structure, function, bioenergetics, and myofiber identity.
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The study, conducted by researchers at the University of Texas Southwestern Medical Center and Virginia Polytechnic Institute and State University, also found that miR-133a is a potential modulator of human centronuclear myopathy (CNM).
In the study, adult mice missing miR-133a developed progressive CNM, as well as mitochondrial dysfunction and fast-to-slow myofiber conversion.
miRagen Therapeutics chief scientific advisor and co-founder Eric Olson said the similarities in the skeletal muscle abnormalities found in the miR-133a deficient mice and human CNM patients suggest that miR-133a may play a role in the disease.
"The findings further underscore the potential of microRNA modulation as a novel therapeutic approach to treat skeletal muscle diseases," Olson said.
miRagen Therapeutics CEO and president William Marshall said the findings ultimately enhance their commitment to develop innovative microRNA-based therapeutics to treat patients with debilitating muscle diseases.