Engineered Cas9 exosome vesicles as a novel gene editing tool for targeted ASPN editing in osteoarthritis - PubMed
8 days ago
- #Osteoarthritis
- #CRISPR-Cas9
- #Exosomes
- CRISPR-Cas9 is a promising genome-editing technique but lacks effective in vivo delivery methods for osteoarthritis (OA) treatment.
- Exosomes, natural nanosized vesicles, are explored as delivery vehicles for CRISPR/Cas9 components.
- Chondrocyte affinity peptide (Cap)-modified MSC-derived exosomes were used to target OA-affected chondrocytes.
- The modification efficiency of Cap was 79.1%, and encapsulation efficiency of ASPN-Cas9 plasmid into exosomes was 9.5% ± 0.6%.
- This delivery method improved cellular uptake and gene-editing efficacy, reducing ASPN expression by 61.7%.
- Benefits included alleviated ferroptosis, improved mitochondrial function, reduced chondrocyte senescence, and inhibited inflammation.
- The study highlights the potential of this method as a precise gene-targeting therapy for OA.