Therapeutic Agents for Treatment of Myotonic Dystrophy
- Detailed Technology Description
- None
- Countries
- Not Available
- Application No.
- None
- *Abstract
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Dr. Zimmerman's lab from the University of Illinois 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.
- *IP Issue Date
- None
- *IP Type
- Other Patent
- Country/Region
- USA

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