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Muscular dystrophies are inherited disorders that result in progressive muscle damage and functional disability. There are currently no effective treatments available for the most common muscular dystrophy in adults, myotonic dystrophy type 1 (DM1). Mani Mahadevan (University of Virginia, Charlottesville) led the team that identified the mutation that causes DM1, which they reported in 1992 (Science 255, 1253–1255). New work from his laboratory, in collaboration with the biotechnology company Biogen Idec (Cambridge, MA), identifies a potential therapeutic target for DM1 and might lead to the development of clinically relevant treatments for the disease.
Lab Anim. (NY) 44, 161 (2015).
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