TY  - RPRT
TI  - Antisense oligonucleotides targeting Valosin-containing protein improve muscle pathology and molecular defects in cell and mouse models of multisystem proteinopathy.
AU  - Pal, P.
AU  - Carrer, M.
AU  - Weiss, L.
AU  - Jaime, O. G.
AU  - Cheng, C.
AU  - Shmara, A.
AU  - Boock, V.
AU  - Bosch, D.
AU  - Youssef, M.
AU  - Fazeli, Y.
AU  - Afetian, M.
AU  - Grossman, T.
AU  - Hicks, M. R.
AU  - Jafar-nejad, P.
AU  - Kimonis, V.
PY  - 2025
DO  - 10.1101/2025.07.25.665261
UR  - https://www.biorxiv.org/content/10.1101/2025.07.25.665261v1
ID  - 10.1101/2025.07.25.665261
ER  -