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VCP/p97 inhibitor CB-5083 modulates muscle pathology in a mouse model of VCP inclusion body myopathy

Abstract Background Pathogenic gain of function variants in Valosin-containing protein (VCP) cause a unique disease characterized by inclusion body myopathy with early-onset Paget disease of bone and frontotemporal dementia (also known as Multisystem proteinopathy (MSP)). Previous studies in drosoph...

Whakaahuatanga katoa

I tiakina i:
Ngā taipitopito rārangi puna kōrero
Ngā kaituhi matua: Cheng Cheng, Lan Weiss, Henri Leinonen, Alyaa Shmara, Hong Z. Yin, Timothy Ton, Annie Do, Jonathan Lee, Lac Ta, Eshanee Mohanty, Jesse Vargas, John Weiss, Krzysztof Palczewski, Virginia Kimonis
Hōputu: Artigo
Reo:Inglês
I whakaputaina: BMC 2022-01-01
Rangatū:Journal of Translational Medicine
Urunga tuihono:https://doi.org/10.1186/s12967-021-03186-6
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