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Using Alternative Genetic Compensation Methods to Treat Muscular Dystrophy

Researchers at UPenn have found a way to modify gene expression without modifying genome coding, opening the door for future possibilities of treatment for patients with Duchenne muscular dystrophy (DMD).

The team of researchers have identified a group of small molecules that may aid in the  development of new therapies in DMD, an as-of-yet incurable disease that results in devastating muscle weakening and loss of bodily functions in patients over time.

The molecules tested by the team from the Perelman School of Medicine work by easing the repression of a specific gene, utrophin. This gene codes for a protein expressed in muscle cells. When the suppressor mechanism is removed from this gene, cells are free to produce excess utrophin protein—a protein that can substitute dystrophin, the protein whose absence causes DMD. These novel findings were published this month in Scientific Reports.


Using Alternative Genetic Compensation Methods To Treat Muscular Dystrophy