A viral-vector strategy to selectively target skeletal muscle fibro-adipogenic progenitors is developed and validated in vivo.
Key Takeaways
- 826 base-pair Pdgfra promoter fragment drove higher reporter expression in fibro-adipogenic progenitors
- Recombinant AAV5 with that promoter achieved the best in vivo FAP specificity and expression
- Minimal off-target reporter expression occurred in myofibers, endothelial, immune, and satellite cells