Methods, compositions, and cells for encapsidating...

Details Methods, compositions, and cells for encapsidating... Methods, compositions, and cells for encapsidating...

C - Chemistry, Metallurgy

12

N

C12N 15/864 (2006.01) C07K 14/015 (2006.01) C12N 5/10 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2370541

Isolated recombinant polynucleotides comprising elements which promote encapsidation into AAV particles, packaging cells comprising the recombinant polynucleotides, and methods for their use are provided in the present invention. These isolated recombinant polynucleotides comprise a non-AAV ITR encapsidation element (such as the P1 sequence located within the AAV S1 integration site of human chromosome 19) operably linked to one or more heterologous genes to be encapsidated. The constructs may be either integrated into a mammalian cell genome, maintained episomally, or provided transiently.

Polynucléotides de recombinaison isolés comprenant des éléments favorisant l'encapsidation dans des particules de virus adéno-associés (AAV), cellules de conditionnement comprenant ces polynucléotides de recombinaison et procédés permettant de les utiliser. Ces polynucléotides de recombinaison isolés comprennent un élément d'encapsidation différent d'une répétition terminale inverse (ITR) de AAV (tel que la séquence P1 située à l'intérieur du site d'intégration S1 de AAV du chromosome humain 19) relié à un ou plusieurs gènes hétérologues à encapsider. Ces produits de recombinaison peuvent être intégrés dans un génome cellulaire mammifère, maintenus dans l'épisome ou produits de façon transitoire.

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