Rational drug design offers potential cure for muscular dystrophy

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MatthewDisney

UB chemists have used rational drug design to synthesize small, cell-permeable molecules that are effective in vitro against two common types of myotonic muscular dystrophy, a result that has implications for potentially curing muscular dystrophy, as well as other diseases.

The UB research was reported in two papers published in the May and July issues of ACS Chemical Biology and Journal of the American Chemical Society, respectively.

Together, the papers demonstrate that rational drug design—where information about a target’s molecular structure is used to “custom-design” potential drugs—can greatly expedite the drug-discovery process in the fight against RNA-mediated diseases, including myotonic dystrophy type 1 and type 2. There currently is no cure for these diseases, which attack muscle tissue.

Read more here.

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