In vitro packaging of adeno-associated virus dna

Details In vitro packaging of adeno-associated virus dna In vitro packaging of adeno-associated virus dna

C - Chemistry, Metallurgy

12

N

C12N 15/86 (2006.01) A61K 31/70 (2006.01) A61K 48/00 (2006.01) C12N 7/01 (2006.01) C12N 15/864 (2006.01)

Patent

CA 2222534

A method for in vitro packaging of adeno-associated viral particles is described. The procedure involves the preparation of cell-free extracts containing all the essential components for packaging. Homogeneous purified substrate DNA for packaging may be prepared separately. The in vitro packaged AAV particles are useful in transduction of mammalian cells and for gene therapy in animals. In one described method, the DNA packaged into AAV particles is not limited by the size constraints characteristic of in vivo packaged AAV particles.

Procédé d'encapsidation in vitro de particules virales adéno-associées. Le processus décrit consiste à préparer des extraits dépourvues de cellules, contenant tous les constituants essentiels à l'encapsidation. L'ADN homogène purifié servant de substrat, et destiné à l'encapsidation, peut être préparé séparément. Les particules de virus adéno-associé encapsidées in vitro peuvent être utilisées dans la transduction de cellules de mammifères et dans la thérapie génique chez les animaux. Selon l'un des procédés décrits, l'ADN encapsidé dans les particules de virus adéno-associé n'est pas soumis aux restrictions de taille caractérisant les particules de virus adéno-associé encapsidées in vivo.

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