Key result
K63-linked ubiquitination of MBNL1 is required for its cytoplasmic localization and neurite outgrowth, while its deubiquitination by expanded CUG RNA is pathogenic in the DM1 brain.
Population
DM1 mouse brain and cellular models of expanded CUG RNA
Design
Preclinical
Authors
Loading...
Does not inform DM1 care; leaves open whether MBNL1 ubiquitination is a viable therapeutic target in patients.
K63-linked ubiquitination of MBNL1 is required for its cytoplasmic localization, and its deubiquitination is pathogenic in the DM1 brain.
Wang et al. (2018) studied Myotonic dystrophy type 1 (DM1). K63-linked ubiquitination of MBNL1 was evaluated on Neurite morphogenesis and cytoplasmic localization. K63-linked ubiquitination of MBNL1 is required for its cytoplasmic localization and neurite outgrowth, while its deubiquitination by expanded CUG RNA is pathogenic in the DM1 brain.