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Significance In this work, we report the use of chloroquine as a treatment against myotonic dystrophy type 1 (DM1), a neuromuscular disease caused by expanded CUG repeats with no available effective treatment. We tested the effect of the drug in 2 different animal models of the disease,Drosophila and mouse, and also in human-derived myoblasts. We demonstrate that chloroquine treatment was able to upregulate Muscleblind levels. These proteins are a key component in the pathogenic mechanism of the disease, as they are sequestered by mutant RNA expressing expanded CUG repeats, and are thus deprived of their normal function. Moreover, we observed that Muscleblind boosting was functional, detecting amelioration of disease-related phenotypes in all 3 models at molecular, cellular, and physiological levels.

Increased Muscleblind levels by chloroquine treatment improve myotonic dystrophy type 1 phenotypes in in vitro and in vivo models