DM1 is the most common form of adult-onset muscular dystrophy. “This finding opens a new avenue for a therapeutic strategy for this disease,” said Prof. Andrew Berglund, Ph.D. “This is the first evidence that specifically inhibiting transcription can be effective in knocking down the toxic material that causes the disease.”
In myotonic dystrophy and other related neurological disorders, the symptoms stem from repeated individual nucleotides in the RNA in muscle tissue cells that can build up over time. These repeats, called CTG expansions in myotonic dystrophy type 1, become “toxic” when transcribed from DNA...
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