CMV-Cre AAV (Serotype Retrograde)

The interest in rAAV-based therapeutics has grown over the past few years because rAAV has key features in terms of safety and efficacy compared to other viral and non-viral delivery systems. One of the most attractive features of rAAV is that the entire transduction process is independent of the two viral genes of AAV, rep and cap, which only need to be provided in trans during the packaging process. Therefore, there are no AAV genes in the rAAV payload. In addition, rAAV produces less immunogenicity than other viral transfer technologies, such as HSV- and Ad-based vectors. The third attractive property of rAAV is its relatively high transduction efficiency and the persistence of transgene expression in post-mitotic and slowly latent cells. This property is important because it enables the development of disease-modifying therapies for chronic diseases with a one-time administration. Viral vectors have a long history in therapeutic gene delivery and intervention. Current efforts are beginning to bear fruit as more viral vector-based therapies enter late-stage clinical development. Glybera, an adeno-associated virus serotype 1 (AAV1)-based gene therapy for the treatment of lipoprotein lipase deficiency, was the first viral vector therapy approved by the European Medicines Agency (EMA) in 2012. In addition, the U.S. Food and Drug Administration (FDA) approved another AAV-based gene therapy, Luxturna, from Spark Therapeutics for the treatment of a genetic disease that causes blindness.