Delivery, delivery, delivery: addressing the remaining challenges in exon skipping for Duchenne

Abstract

“Sharing what works, what does not, what is safe, and what is not would advance the field considerably…”Jokūbas Leikauskas, Editor, Nucleic Acid Insights , speaks with Annemieke Aartsma-Rus, Professor of Translational Genetics, Leiden University Medical Center, about why delivery remains the central challenge in exon skipping for Duchenne muscular dystrophy, the difficulty of proving slowed disease progression, the role of humanized and patient-derived preclinical models, and the lessons learned now transferring to rare brain diseases.