RNA Therapeutics for Duchenne Muscular Dystrophy: Exon Skipping, RNA Editing, and Translational Insights from Genome-Edited Microminipig Models - PubMed
7 hours ago
- #Duchenne Muscular Dystrophy
- #RNA Therapeutics
- #Animal Models
- Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease caused by DMD gene mutations.
- RNA-based therapies like exon skipping and RNA editing are promising approaches to modulate splicing or correct transcripts without altering DNA.
- Phosphorodiamidate morpholino oligomer (PMO) drugs target specific exons, while advanced delivery methods aim to improve tissue exposure.
- RNA editing strategies offer potential to correct nonsense or missense variants and restore dystrophin expression.
- Genome-edited microminipig (MMP) models with DMD mutations replicate human symptoms and serve as practical platforms for long-term therapeutic studies.