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Molecule May Correct Toxicity of Muscular Dystrophy Defect

Study finds that oligonucleotide disrupts toxic RNA-protein complexes in mice

FRIDAY, July 17 (HealthDay News) -- A synthetic molecule can correct the consequences of the genetic defect causing myotonic dystrophy in a mouse model of the disease, according to a study in the July 17 issue of Science.

Thurman M. Wheeler, M.D., and colleagues from the University of Rochester in New York examined whether CAG25, a morpholino antisense oligonucleotide, could target the toxic RNA of the gene responsible for myotonic dystrophy type 1 in a mouse model of the disease, releasing the MBNL1 protein that carries out an important cellular function.

The researchers found that CAG25 could disrupt these toxic RNA-protein complexes when injected into muscle, releasing MBNL1 and restoring its ability to perform its function. This led to a restoration of the function of a muscle-specific chloride channel and a marked reduction in myotonia.

"Taken together, these data supply proof of concept that agents inhibiting deleterious RNA-protein interactions have therapeutic potential in RNA-dominant disorders," Wheeler and colleagues conclude.

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