Matthew Disney is an associate professor in the Department of Chemistry at the Scripps Research Institute in Jupiter, Fla. He has MDA support to design small molecules that target the toxic RNA that underlies myotonic muscular dystrophy (MMD, also known as DM).
MDA Medical and Science Editor Margaret Wahl talked with Matthew Disney about his work.
An interview with MDA research grantee Matthew Disney explores his group's findings about small molecules as a potential treatment for myotonic dystrophy
In type 1 myotonic dystrophy (MMD1, or DM1), expansions of DNA on chromosome 19 known as CTG repeats are converted to expansions in RNA called CUG repeats, which are toxic to nerve and muscles cells in a variety of ways.
Several MDA-supported research teams are targeting the toxic CUG repeats, with the goal of either blocking their interaction with other cellular substances or destroying them...
Scientists have developed synthetic compounds that reduce the harmful effects of MMD1-associated expansions of RNA in cellular models of the disease
Editor's note 3/15/12: This story was updated to reflect the availability of a podcast with Matthew Disney.
Small, laboratory-designed molecules can make a big difference in cells carrying the genetic defect that causes type 1 myotonic dystrophy (DM1, or MMD1), researchers have found.