Cientistas italianos afirmam ter desenvolvido técnica inédita para correção do gene da distrofia de Duchenne

Rome scientists find way to correct genetic fault behind DMD
(ANSA) – Rome, February 20 – Italian scientists say they may have found a way to correct the genetic fault that causes one of the most common forms of muscular dystrophy .

Rome university researchers say they managed to correct defective genes in mice with Duchenne Muscular Dystrophy (DMD) .

They did this using an ‘antisense RNA’ molecule which, when applied to a defective gene, acts like a ‘band aid’ and heals it .

It is an innovative technique because it seeks to heal faulty genes, rather than replace them with new ones, as researchers have attempted to do in the past .

DMD is a hereditary degenerative disorder characterised by muscle weakness and wasting .

It mainly affects males. The incidence is estimated to be one in 3,500 new-born boys. The onset of the disease comes between two and six years of age. Sufferers usually need a wheelchair by the age of 12 and it is rare for them to survive beyond their early 30s. At the moment there are no medicines that can stop or reverse DMD muscle degeneration, although some therapies can slow it .

The problem with the faulty gene is that it does not produce dystrophin, a protein that helps keep muscle cells strong .

The ‘antisense RNA’, a molecule similar to DNA, has a genetic code that is complementary to that of the defective gene. Carried to the defective gene via a ‘shuttle virus’ injected into the muscle, this makes it possible to cover the fault so the gene produces a protein that is similar to dystrophin .

The alternative protein is a little shorter than dystrophin, but works pretty well .

The Rome University team, led by Irene Bozzoni, said the muscle use and mobility of mice treated in this way improved significantly .

The results of the research will be published in the latest issue of US periodical, Proceedings of the National Academy of Sciences .

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