Topic: A potential treatment for muscular dystrophy?

[Nemesis]

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An international team of researchers has successfully treated dogs with the canine form of Duchenne muscular dystrophy (DMD), a rapidly progressing and ultimately fatal muscle disease that afflicts one out of every 3,600 boys. The researchers used a novel technique called exon skipping to restore partial function to the gene involved in Duchenne. The study, published in Annals of Neurology, gives hope that a similar approach could work in humans.

Several years ago, scientists found that the difference is not in how much of the gene is missing, but in how those missing portions affect the remaining gene sequence. Most Duchenne patients have frameshift mutations, which interfere with the cell's reading of three-letter DNA code. These deletions shift the remaining DNA sequence into different triplet groupings, rendering the gene unreadable. In Becker patients, the remaining DNA can still be read normally, allowing them to produce a smaller but still functional version of dystrophin.

[Nemesis]

Another advance.

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The researchers used a synthetic molecule to break up deposits of toxic genetic material and re-establish the cellular activity that is disrupted by the disease. Because scientists believe that potentially all of the symptoms of myotonic dystrophy – the most common form of muscular dystrophy in adults – flow from this single genetic flaw, neutralizing it could potentially restore muscle function in people with the disease.