H compounds

An interview with MDA research grantee Matthew Disney explores his group's findings about small molecules as a potential treatment for myotonic dystrophy

posted on March 12, 2012 - 6:00am
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...

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