When a number of derivatized small-molecule inhibitors bind to an RNA repeat sequence in cells, their alkyne and azide groups react with one another, producing potent oligomers.
If a multicomponent bioactive agent is too big to slip into cells, it would be nice to get its components into cells first and then combine them on-site. That’s what Matthew D. Disney, Suzanne G. Rzuczek, and HaJeung Park at Scripps Research Institute Florida did with a potential myotonic dystrophy type 2 (DM2) treatment (Angew. Chem. 2014, DOI: 10.1002/ange.201406465).
DM2 is a rare condition characterized by muscle pain and weakness. It’s caused by a genetic defect that generates toxic RNA with a four-nucleotide repeat pattern.
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