Generation of high titers of recombinant aav vectors

Details Generation of high titers of recombinant aav vectors Generation of high titers of recombinant aav vectors

C - Chemistry, Metallurgy

12

N

C12N 7/01 (2006.01) C12N 15/12 (2006.01) C12N 15/49 (2006.01) C12N 15/86 (2006.01) C12N 15/864 (2006.01)

Patent

CA 2176117

AAV vectors may have utility for gene therapy but heretofore a significant obstacle has been the in- ability to generate sufficient quantities of such recom- binant vectors in amounts that would be clinically useful for human gene therapy application. Stable, helper-free AAV packaging cell lines have been elusive, mainly due to the activities of Rep protein, which down-regulates its own expression and reverses cellular immortaliza- tion. This invention provides packaging systems and processes for packaging AAV vectors that effectively circumvent these problems by replacing the AAV p5 promoter with a heterologous promoter (as depicted in the figure) and that allow for substantially increased packaging efficiency.

L'utilisation de vecteurs du virus adéno-associé (AAV) en thérapie génique a jusqu'ici rencontré un obstacle notable dans la mesure où il était impossible de générer de tels vecteurs recombinants en quantités suffisantes pour une utilisation d'ordre clinique en thérapie génique humaine. Les lignées cellulaires stables d'encapsidation d'AAV, sans cellules auxiliaires, se sont révélées difficiles à développer, principalement en raison des activités de la protéine Rep, qui sous-régule sa propre expression et inverse le processus d'immortalisation cellulaire. Cette invention permet d'obtenir des procédés et des systèmes d'encapsidation de vecteurs d'AAV qui permettent d'éviter ces problèmes en remplaçant le promoteur p5 d'AAV par un promoteur hétérologue (comme illustré dans la figure), et qui permettent en conséquence d'accroître sensiblement l'efficacité d'encapsidation.

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