Researchers at important academic labs around the US have recently published exciting new information about advances in DM research. The Matthew Disney Lab at The Scripps Research Institute in Florida announced the results of a study examining RNA toxicity in DM2 patients. Summaries of the studies and results are below, along with links to the PubMed abstracts and complete research publications.
Structure of the Myotonic Dystrophy Type 2 RNA and Designed Small Molecules that Reduce Toxicity Childs-Disney et al (Matthew Disney Lab)
Researchers at Scripps Research Institute in Florida recently examined the structure of the toxic RNA molecule made from the DNA mutation causing myotonic dystrophy type 2 (DM2). Dr. Matthew Disney and his colleagues used a technique called X-ray crystallography to look at the shape of the RNA at the atomic level in order to determine what types of drugs would best attach to it and reduce its toxicity.
One of the reasons the DM2 RNA is unhealthy in cells is because it changes how other genes are processed and regulated. They showed that their custom-designed drugs were able to reverse the improper processing of a gene known to be affected in DM by varying degrees depending on the design of the drug. This study shows that drugs they previously proposed could bind the toxic RNA are now able to be administered to cells with toxic RNA similar to DM2 and reduce the RNA toxicity.
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