Compositions and methods for recombinant adeno-associated...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/864 (2006.01) C12N 7/01 (2006.01) C12N 7/02 (2006.01) C12N 15/35 (2006.01) C12Q 1/70 (2006.01)

Patent

CA 2365293

The present invention relates to methods and compositions for the production of recombinant Adeno-Associated Viruses (rAAV). In particular, the invention discloses nucleic acid constructs and packaging cells having improved properties for rAAV production, as well as methods of titration and characterization of rAAV preparations. The invention also describes sequences which promote or increase the packaging of nucleic acids in rAAV, and their use for producing rAAV with high efficiency. The invention can be used for producing or testing high quality rAAV preparations, for biological, preclinical, clinical or pharmaceutical uses.

La présente invention concerne des procédés et compositions de production de virus recombinés adéno-associés (rAAV), et elle concerne notamment des constructions d'acides nucléiques et des cellules d'encapsidation possédant des propriétés améliorées destinées à la production de rAAV. L'invention concerne encore des procédés de titrage et de caractérisation de préparations de rAAv, de même que des séquences nouvelles qui favorisent ou augmentent l'encapsidation d'acides nucléiques dans rAAV, ainsi que l'utilisation de ces séquences pour produire des rAAV très efficaces. L'invention concerne enfin la production ou l'essai de préparations de rAAV de grande qualité, destinées à des usages biologiques, précliniques, cliniques ou pharmaceutiques.

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