Antisense oligonucleotides targeting Valosin-containing protein improve muscle pathology and molecular defects in cell and mouse models of multisystem proteinopathy.
Pallabi Pal, Michele Carrer, Lan Nguyen Weiss, Olga G. Jaime, Cheng Cheng, Alyaa Shmara, Victoria Boock, Danae Bosch, Marwan Youssef, Yasamin Fazeli, Megan E. Afetian, Tamar R. Grossman, Michael R. Hicks, Paymaan Jafar‐Nejad, Virginia E. Kimonis
This study shows that antisense oligonucleotides targeting VCP reduce disease pathology and improve muscle function in cell and mouse models of multisystem proteinopathy.
bioRxiv · Jul 25, 2025 · PreprintMolecular Biology