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Aurora magazine

An artificial gene to combat Duchenne syndrome

A study by the CNR of Rome describes Jazz-Zif1, the artificial gene that could fight Duchenne's syndrome. The new gene increases the levels of utrophin and corrects the lack or absence of dystrophin. In this way it works against Duchenne Muscular Dystrophy and relieves its symptoms.

Duchenne is caused by the lack of a protein, dystrophin. This leads to the progressive degeneration of muscle tissue and thus making motor skills. According to some studies, utrophin would be able to replace dystrophin, at least in the main functions. Injections of the protein have in fact improved the conditions of animal models affected by the disease. How to make the effects permanent?

The team of Rome has developed an artificial regulator gene, able to increase the production of utrophin. Jazz-Zif1 is almost identical to a natural gene, which should zero the host's immune response. Once injected into the body, then, the gene would be able to camouflage itself and regulate protein levels without external interventions.

To make the artificial gene as natural as possible, the researchers used a viral vector. AAV ensures that the gene has excellent specificity and leaves the muscle district to direct gene expression. In this way it lowers the risk of immune reactions in response to Jazz-Zif1.

Researchers studied the action of Jazz-Zif1 on adult mice with dystrophy. According to the first results, the treatment induced a good muscle recovery. In particular, it has proved beneficial for neuromuscular junctions, the areas in which utrophin is more present. It has in fact increased the quantity and quality, with benefits for the guinea pigs' health.

Further studies will be needed. This does not mean that in the long run this strategy could give rise to new treatments against Duchenne syndrome.