AAV8-Syn-iCre

AAV is a small, single-stranded DNA virus that was first discovered in 1965 as a contaminant of adenoviral preparations. In the 1980s, it became known that all viral coding sequences could be replaced by transgenic DNA, and that AAV vectors (carrying transgenic DNA) would result if viral genes were delivered in trans along with other “helper” genes. Over the past 30 years, AAV gene therapy has made its way from the bench to the clinic for a wide range of diseases. In 2017, the FDA approved the first commercial AAV-based gene therapy for a posterior segment disease of the eye. The success of AAV vectors compared to other viral and nonviral gene delivery methods is due in part to: (i) its extremely high infectivity to most human tissues, (ii) the persistence of the AAV vector genome (vg) in an episomal form with low levels of host integration, (iii) serotypes exhibit altered tropism, (iv) the ability to infect dividing or non-dividing cells, and (v) preclinical and clinical data showing transgene expression for many years after a single administration. AAV has emerged as the most promising gene delivery vector and has been clinically used for various diseases via different administration routes.