Investigators led by Matthew Disney at the Scripps Research Institute in Jupiter, FL have successfully demonstrated an approach to synthesize potent small molecules inside diseased cells in a highly selective and sensitive manner. The research, published August 27 in Angewandte Chemie, adapted a method called ‘click chemistry’ to create modular small molecules which bind internal loops in the RNA tetranucleotide repeats that cause myotonic dystrophy type 2, a rare form of muscular dystrophy. Inside the cells, the compounds assemble to form a drug that is 1000 times more potent than the original small molecule. This approach holds potential for other diseases associated with nucleotide repeat expansions, such as C9ORF72 ALS/FTD (see Jan 2013 news story). Click here to read more.
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