Antisense oligonucleotides targeting valosin-containing protein ameliorate muscle pathology and molecular defects in cell and mouse models of multisystem proteinopathy

靶向含缬氨酸蛋白的反义寡核苷酸可改善多系统蛋白病细胞和小鼠模型中的肌肉病理和分子缺陷。