Estefania Cerro-Herreros

Oct 9, 2024 | science.org | Deborah Carper |Hyun Lee |Nolan Tucker |Estefania Cerro-Herreros

AbstractThis study evaluated therapeutic antimiRs in primary myoblasts from patients with myotonic dystrophy type 1 (DM1). DM1 results from unstable CTG repeat expansions in the DMPK gene, leading to variable clinical manifestations by depleting muscleblind-like splicing regulator protein MBNL1. AntimiRs targeting natural repressors miR-23b and miR-218 boost MBNL1 expression but must be optimized for a better pharmacological profile in humans.