Compounds and methods for myotonic dystrophy therapy

Steven C Zimmerman (Inventor), Long Minh Luu (Inventor), Lien T T Nguyen (Inventor)

Research output: Patent


The invention provides rationally designed multi-targeting therapeutic agents for myotonic dystrophy type 1 (DM1), an incurable neuromuscular disease that originates in an abnormal expansion of CTG repeats (CTGexp) in the DMPK gene. The rationally designed small molecules target the DM1 pathobiology in three distinct ways: (1) binding the expanded trinucleotide repeat, CTGexp, and inhibiting its transcription to the toxic CUGexp RNA, (2) binding the CUGexp RNA and releasing sequestered muscleblind-like protein (MBNL1), and (3) cleaving the toxic CUGexp in an RNase-like manner. Importantly, the compounds can reduce the levels of CUGexp in DM1 model cells and reverse two separate CUGexp-induced phenotypes of DM1.
Original languageEnglish (US)
U.S. patent number9376421
Filing date8/10/15
StatePublished - Jun 28 2016


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