Nano Bio Technology

Identified miR-31 plays a key role in controlling muscle differentiation, this molecule is also a strong repressor of synthesis of dystrophin.

It’s called miR-31, the microRNAs identified by Italian researchers, which may be a new ally in the therapeutic approaches against Duchenne muscular dystrophy, severe genetic disorder that affects one child in 3500 and is caused by a mutation in the X chromosome that determines the failure to produce a protein called dystrophin. The absence of dystrophin leads to a progressive – and relentless at the moment – the death of muscle fibers.

The study “miR-31 modulates dystrophin expression: Implications for Duchenne muscular dystrophy new therapy”, published on January 7, 2011 EMBO Reports, was conducted on mouse and human muscle cells with the support of Parent Project Onlus and Telethon.

The team of Wisdom, by comparing biopsies from healthy subjects and patients with Duchenne muscular dystrophy, has remarked that in the latter, the levels of miR-31 were more abundant. Studies have shown that one of the roles of this molecule is to control the synthesis of dystrophin, repressing translation. Therefore, the inhibition of miR-31, designed to increase the production of dystrophin.

The control of the mir-31, favoring an increase in the synthesis of dystrophin, may represent an effective method to improve the effectiveness dell’exon skipping, a therapeutic approach currently being tested which is already the potential to restore partially the synthesis of dystrophin, transforming the Duchenne muscular dystrophy in the less aggressive variant of the Becker muscular dystrophy.

“The relevance of these studies is the fact that you have identified a microRNA (miR-31) which plays a key role in controlling the transition from early to late those of muscle differentiation and that this molecule is a strong repressor of synthesis of dystrophin. – He explained Professor Irene Bozzoni – controlling the levels of miR-31 in dystrophic cells treated with exon skipping, we have obtained is increased synthesis of dystrophin that improvement of terminal differentiation of muscle fibers, a process compromised in dystrophic muscles. Therefore, these results very important implications for the further improvement of current treatment strategies. The next step will be to be able to control in vivo the amount of miR-31 specifically in muscle cells and then combine this strategy with that dell’exon skipping. ”

“This study gives further confirmation of what we are convinced for some time: there is no single path to follow to defeat this disease – says Filippo Buccella, president of Parent Project Onlus, an association of parents with children affected by Duchenne – Only a collaborative approach that puts all the resources on the network will lead us, we hope to achieve important results and give hope to our children. ”

“This result demonstrates once again how to invest on excellent projects pay more over time-adds Lucia Monaco, scientific director of the Telethon – More than 10 years that our organization supports the work of Irene Bozzoni and his group, which has invested over half a million euro, we can not therefore be satisfied with this further step in the treatment of Duchenne muscular dystrophy, in full accord with our mission. ”

Duchenne muscular dystrophy is a rare disease, the most severe form of muscular dystrophy that occurs in children. Involves progressive muscle degeneration to the impairment of the heart muscle, diaphragm and intercostal muscles to make the required respiratory assistance. It is estimated that in Italy there are 5,000 people living with the disease for which there is currently no specific treatment, but treatment by a multidisciplinary team that has improved the general conditions and double the life expectancy.

The Becker muscular dystrophy it is a less aggressive variant, whose course, however, varies from patient to patient.