GeneticsThe American Journal of Human GeneticsPeer-reviewed
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…·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.