First treatment for muscular dystrophy in sight: Scientists successfully harness exon-skipping

Genetic researchers at Children's National Medical Center and the National Center of Neurology and Psychiatry in Tokyo published the results of the first successful application of "multiple exon-skipping" to curb the devastating effects of Duchenne muscular dystrophy in an animal larger than a mouse. Multiple exon-skipping employs multiple DNA-like molecules as a "DNA band-aids" to skip over the parts of the mutated gene that block the effective creation of proteins.

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<p><a href="http://www.physorg.com/news156435829.html">First treatment for muscular dystrophy in sight: Scientists successfully harness exon-skipping</a></p> <p>Genetic researchers at Children's National Medical Center and the National Center of Neurology and Psychiatry in Tokyo published the results of the first successful application of "multiple exon-skipping" to curb the devastating effects of Duchenne muscular dystrophy in an animal larger than a mouse. Multiple exon-skipping employs multiple DNA-like molecules as a "DNA band-aids" to skip over the parts of the mutated gene that block the effective creation of proteins.</p>