Article
A Small-Molecule Approach to Restore a Slow-Oxidative Phenotype and Defective CaMKIIβ Signaling in Limb Girdle Muscular Dystrophy.
Cell reports. Medicine - 20 Oct 2020
Liu Jian, Campagna Jesus, John Varghese, Damoiseaux Robert, Mokhonova Ekaterina, Becerra Diana, Meng Huan, McNally Elizabeth M, Pyle April D, Kramerova Irina, Spencer Melissa J
Abstract excerpt
Mutations in CAPN3 cause limb girdle muscular dystrophy R1 (LGMDR1, formerly LGMD2A) and lead to progressive and debilitating muscle wasting. Calpain 3 deficiency is associated with impaired CaMKIIβ signaling and blunted transcriptional programs that encode the slow-oxidative muscle phenotype. We conducted a high-throughput screen on a target of CaMKII (Myl2) to identify compounds to override this signaling...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
