Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for Myotonic Dystrophy type 1
Irene González-Martínez, Estefanía Cerro-Herreros, Marc Carrascosa-Sàez, Andrea García-Rey, Diego Piqueras‐Losilla, Anna Colom-Rodrigo, Nerea Moreno, Mouli Chakraborty, Aline Huguet-Lachon, Anchel González‐Barriga, Neia Naldaiz‐Gastesi, Martxel Dehesa-Etxebeste, Ana Díaz-Maqueda, Nuria Barquero, Miguel A. Varela, , 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.
The American Journal of Human Genetics · Feb 20, 2026Peer-reviewedGenetics