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Dystrophin restoration after AAV U7-mediated Dmd exon skipping is modulated by muscle exercise in a mouse model of severe DMD
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A team of French researchers, involving researchers from the Institute, showed in a mouse model of severe DMD that voluntary exercise has an impact on a Dmd exonPartie codante de l’ADN au sein d’un gène. skipping approach and on muscle physiology.
D2-mdx mice were administered an AAV-U7 snRNA intra-muscularly to correct the Dmd reading frame and then allowed to run in a wheel for one month.
The results show that voluntary running :
- did not induce muscle damage,
- did not have a markedly negative effect on the exon skipping approach by AAV« adeno-associated virus » , ou virus adéno-associé est un petit virus à ADN simple brin. Il fait partie de la famille des Parvoviridae et appartient au genre des Dependovirus. La particule virale est constituée d’un brin d’ADN de polarité positive ou négative protégé par une capside. La taille moyenne d’une particule d’AAV et de 18 à 22 nm. Les AAV sont les seuls parvovirus non autonomes. Lorsqu’on emploie « rAAV » , il s’agit du virus AAV recombinant, c’est-à-dire qu’il a été modifié pour devenir un vecteur (et n’est donc plus virulent). gene therapy, but somewhat decreased the restoration of dystrophin for reasons which are not yet well understood.
The authors conclude that exercise should be an additional element to be considered in the conception and design of future therapeutic approaches for DMD.