Pentatricopeptide Repeat Protein Targeting CUG Repeat RNA Ameliorates RNA Toxicity in a Myotonic Dystrophy Type 1 Mouse Model.

Takayoshi Imai, Maiko Miyai, Joe Nemoto, Takayuki Tamai, Masaru Ohta, Yusuke Yagi, Osamu Nakanishi,Hideki Mochizuki,Masayuki Nakamori

Science translational medicine（2025）

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