A Molecular Advancement for Muscular Dystrophy

By Disney Scripps Florida

Introduction With almost 10 years of experience as a

professor, Matthew Disney has worked at Florida’s Scripps Research Institute since 2010. An award-winning member of its department of chemistry, he received his PhD in biophysical chemistry from the University of Rochester. Matthew Disney’s research group at Scripps Florida is dedicated to developing highly selective therapeutics using molecular genetic techniques.

Research Recently, his research team developed a

method to treat an uncommon form of muscular dystrophy called myotonic dystrophy type 2, using the diseased cells as manufacturing sites for beneficial molecules. Using “click chemistry,” which quickly creates substances via their RNA precursors, the method could potentially help treat incurable diseases like Huntington’s disease, in addition to other types of muscular dystrophy.

Conclusion The study showed that the modular

small molecules generated from the cell’s own genes are able to bind defective parts of living cells together, which could reverse the defects entirely. It allows such a selective treatment because it uses only diseased cells to construct its own inhibitors and does not affect healthy cells.