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Therapeutic Agents for Treatment of Myotonic Dystrophy

Therapeutic Agents for Treatment of Myotonic Dystr...(TF14097) [1]

 

Dr. Zimmerman's lab has developed a series of bioactive compounds for the treatment of myotonic dystrophy. The compounds disrupt the MBNL-(CUG) foci in vivo and partially rescue the missplicing of cTNT and IR mRNAs. These novel compounds, utilizing bisamidinium-based inhibitors, show a 1000-fold greater inhibition potency than a previous compound and are able to degrade the causative agent of the disease.

Steven
Zimmerman

Inventors:

US Pat #: 
9376421
Issue Date: 
6/28/2016
US Pat #: 
10,266,520
Issue Date: 
4/23/2019
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