Improving rAAV production and purification: towards the definition of a scaleable process.
Numerous in vivo studies have demonstrated that recombinant AAV-2 vectors (rAAV-2) can efficiently transduce many tissues and lead to stable gene expression 12. These encouraging results have led to a rapid development of clinical trials involving the use of rAAV-2 vectors 3. However, the obtainment of large-scale rAAV-2 vector stocks for clinical assay is still hampered by the conventional production and purification methods that are not efficient and difficult to scale up. This review will describe the current methods available for rAAV-2 vector production and purification and show the advancements achieved, in particular in our group, to develop new scalable procedures, suiting GMP requirements.
Blouin
Véronique
V
INSERM ERM 0105, CHU-Hôtel Dieu, Bât. Jean Monnet, 30 Av. Jean Monnet, 44035 Nantes, France.
Brument
Nicole
N
Toublanc
Estelle
E
Raimbaud
Isabelle
I
Moullier
Philippe
P
Salvetti
Anna
A
D000818:Animals; D000229:Dependovirus / Q000302:isolation & purification; D005822:Genetic Vectors / Q000302:isolation & purification; D006367:HeLa Cells; D006801:Humans; D014161:Transduction, Genetic