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Compound That Repairs RNA Defects Identified

Matthew Disney (Scripps Research Institute)

Matthew Disney (Scripps Research Institute)

Researchers at Scripps Research Institute in Jupiter, Florida have identified a compound that can help repair defects in ribonucleic acid (RNA), a genetic material similar to DNA used in protein synthesis. The team’s findings appear online in the journal ACS Chemical Biology (paid subscription required).

The researchers, led by Scripps chemistry professor Matthew Disney (pictured left), are developing a process for identifying compounds that can repair defective RNA, and in this study focused on a particular defect called an expanded triplet repeat. This defect results in a a series of three nucleotides repeated many more times than normal in the genetic code of affected individuals. The triplet repeat has been associated with various neurological and neuromuscular disorders, such as Huntington’s disease, Spinocerebellar ataxia, and Kennedy disease.

Disney and colleagues, including scientists from Adam Mickiewicz University in Poland, leveraged recent studies showing the toxicity of the RNA and the protein translated from the RNA to be toxic. The toxic RNA binds to and removes other proteins used in RNA processing, which contributes to the disease conditions. The Scripps team’s findings identified a small molecule that binds to the RNA and shuts off its toxicity. The new compound targets the toxic RNA and inhibits protein binding.

Disney says the discovery, “not only further demonstrates that the RNA is toxic but also opens up new avenues for therapeutic development because we have clearly demonstrated that small molecules can reverse this type of defect.” He adds, “These studies can open up new avenues to exploit RNA drug targets that cause a host of other RNA-mediated diseases.”

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