Refining gene delivery to skeletal muscle with a dual-strategy approach of muscle-tropic AAV capsids and muscle-specific promoters.
Annalucia L. Darbey, Wenanlan Jin, Charlotte Spicer…·Pietro Fratta
The paper investigates a dual-strategy approach using a muscle-tropic AAV capsid and muscle-specific promoter to improve gene delivery efficiency and reduce off-target expression in skeletal muscle.