SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model - PubMed
7 hours ago
- #Muscular Dystrophy
- #Lipid Nanoparticles
- #Gene Editing
- SORT LNPs with Cas9 mRNA enable efficient gene editing in skeletal muscle in a dystrophic mouse model.
- Limb Girdle Muscular Dystrophy (LGMD) is the fourth most common muscular dystrophy, and gene editing offers potential treatment.
- Lipid nanoparticles (LNPs) are promising for delivering gene editing tools to skeletal muscle, but efficiency needs improvement.
- The study evaluates the impact of cargo type (mRNA vs. RNP) on editing efficiency, biodistribution, and immune response.
- Optimized SORT LNPs achieved 40% restoration of Telethonin expression in treated muscle.
- The findings support the development of LNP-based gene editing for neuromuscular diseases.