Several subsequent all-in-one rAAV vector studies have focused on two paradigms with opposite objectives, namely, the correction of disruptions to the ORF of the dystrophin gene, the most common cause of Duchenne muscular dystrophy (DMD) [150,214,215], and the disruption of the proprotein convertase subtilisin/kexin type 9 serine protease (PCSK9) gene that is understood to play a critical role in cholesterol metabolism. This evidence concerns the gene PCSK9 and Duchenne muscular dystrophy.