NeuroscienceiSciencePeer-reviewed
Evaluating exon-skipping therapies targeting the central nervous system in Duchenne muscular dystrophy using high-resolution spatial transcriptomics
Qirong Mao, Alireza Ahmadi, Sharon I. de Vries…·Pietro Spitali
This study uses spatial transcriptomics to evaluate exon-skipping therapies for DMD in the CNS, showing U7ex51 effectively restores full-length dystrophin isoforms in neurons.
DOI 10.1016/j.isci.2026.116906