Annalucia Darbey

Combining a muscle-tropic AAV capsid with a muscle-specific promoter enhances skeletal muscle gene delivery while reducing off-target expression

Viral vector technologies based on adeno-associated viruses (AAVs) have demonstrated promising ability to deliver genetic cargo to a range of organs in vivo, with several novel candidates showing clinical efficacy in human trials over the past decade. However, naturally occurring AAV serotypes are limited in thei...