Antisense oligonucleotides targeting valosin-containing protein ameliorate muscle pathology and molecular defects in cell and mouse models of multisystem proteinopathy
Clin Transl Med. 2025 Dec;15(12):e70530. doi: 10.1002/ctm2.70530. ABSTRACT BACKGROUND: Valosin-containing protein (VCP) related disease, also known as multisystem proteinopathy 1 (MSP1), is an autosomal dominant …