Article

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

2025-07-26

Abstract excerpt

Valosin-containing protein (VCP) related disease, also known as multisystem proteinopathy 1 (MSP1), is an autosomal dominant disease caused by gain-of-function pathogenic variants of the VCP gene. The disease is associated with inclusion body myopathy, early-onset Paget’s disease of bone, frontotemporal dementia, and familial amyotrophic lateral sclerosis. There is currently no treatment for this progressive dise...

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Literature Corpus work: 216765d4-f4b7-5dc1-8824-5b2d50bab8e6
DOI: 10.1101/2025.07.25.665261

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