Development of an animal model for BIN1-linked centronuclear myopathy provides proof of concept for the efficacy of an antisense oligonucleotide targeting DNM2

The team from the Institute of Genetics and Molecular and Cellular Biology (Strasbourg) has developed a viable mouse model of centronuclear myopathy, defective of the BIN1 gene selectively in muscles.

BIN1 encodes amphiphysin 2, a protein that controls the curvature of membranes. 

Dynamin 2 is an effector protein of amphiphysin 2, encoded by DNM2. 

Injection of an antisense oligonucleotide targeting DNM2 into these model mice improved their strength and corrected their muscle histological abnormalities within five weeks. 


Mice with muscle-specific deletion of Bin1 recapitulate centronuclear myopathy and acute downregulation of dynamin 2 improves their phenotypes. Silva-Rojas R, Nattarayan V, Jaque-Fernandez F, Gomez-Oca R, Menuet A, Reiss D, Goret M, Messaddeq N, Lionello VM, Kretz C, Cowling BS, Jacquemond V, Laporte J. J. Mol Ther. 2021 Aug 8:S1525-0016(21)00400-7.