Potent Drugs Synthesized in Diseased Cells Hold Promise for ALS

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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