Ruben D. Artero

This study develops chemically optimized miR-23b antimiRs that enhance muscle uptake and restore MBNL1 function in DM1 models, showing improved efficacy and safety.

This paper develops a 3D human muscle model from DM1 patient myoblasts that recapitulates CLCN1 mis-splicing and myotonia, and tests therapeutic responses.

This study shows MEG3 is upregulated in DM1 and exhibits nuclear retention, with antisense conjugates correcting MEG3 levels in mice.

This study identifies FasII upregulation as a key factor in DM1 neuropathology using a Drosophila model, showing that manipulating FasII isoforms can rescue synaptic and behavioral defects.