Aav directed targeted integration

Details Aav directed targeted integration Aav directed targeted integration

C - Chemistry, Metallurgy

12

N

C12N 15/86 (2006.01) A01K 67/027 (2006.01) C12N 5/10 (2006.01) C12N 15/11 (2006.01) C12N 15/864 (2006.01) C12N 15/90 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2223741

The present invention relates to novel recombinant AAV (Adeno-associated virus) vectors that may be used to direct integration of recombinant DNA sequences into specific regions of the human host genome. These vectors may be used in gene therapies designed to replace or supplement defective genetic information with normal genetic information. The invention also relates to the isolation and characterization of the specific cellular host sequences, herein referred to as host target sequences, that signal the integration of AAV recombinant DNA sequences into specific regions of the host genome. Transgenic animals containing the generated target sequences may be used as model systems for testing of AAV based gene therapies.

L'invention se rapporte à deux nouveaux vectors AAV recombinés susceptibles d'être utilisés pour orienter l'intégration des séquences d'ADN recombiné vers des régions spécifiques du génome hôte humain. On peut utiliser ce type de vecteur en thérapie génétique visant à offrir une information génétique normale tenant lieu de substitution ou de supplément pour une information génétique défectueuse. On décrit par ailleurs l'isolement et la caractérisation des séquences hôtes cellulaires spécifiques, dénomées séquences cibles hôtes dans l'invention, qui indiquent l'intégration des séquences ADN recombiné par AAV vers des régions spécifiques du génome hôte. Les animaux transgéniques contenant les séquences cibles engendrées peuvent aussi être utilisés comme systèmes modèles pour les tests propres aux thérapies géniques à base d'AAV.

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