Rewriting Duchenne muscular dystrophy therapy.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 42425062.
- Also identified by DOI 10.1016/j.cell.2026.06.011.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
In this issue of Cell, Guo et al. report the development of a new exon-skipping, RNA-editing-based therapy for Duchenne muscular dystrophy. The dual mechanism of action through both ADAR-dependent and -independent pathways has the potential to be more effective and require a lower dosing frequency than currently available ASO-based exon-skipping treatments.