CELF1 and myotonic dystrophy type 1: As aggregation and transport into the cytoplasm may be two competing events within the nucleus, these data predict that small molecules, which serve to decrease the rate of aggregate formation in the nucleus, to allow transport of the toxic CUG RNAs into the cytoplasm, may be sufficient to greatly ameliorate DM1 symptoms in patients whose repeat tract sizes are not long enough to elicit large increases in CUG-BP1 levels.