Effective Cure for Duchenne Muscular Dystrophy Found by Correcting the Defective Gene
Doctors in Italy have treated mice with the most common form of Muscular Dystrophy by correcting the defective gene responsible for the disease.
The technique uses RNA with a genetic code complementary to that of the defective gene, ANSA reported.
People with Duchenne Muscular Dystrophy have a gene that fails to produce Dystrophin, a protein that builds muscle. Those with the disease, which mostly strikes males, are usually confined to wheelchairs by the age of 12 and seldom live past their early 30s.
Irene Bozzoni, who headed the research team at Rome University, said that mice showed significant improvement after the treatment. The findings were published in the Proceedings of the National Academy of Sciences.
Previous research had focussed on replacing defective disease, not changing them.
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