Raav vector compositions and methods for the treatment of...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/861 (2006.01) C12N 15/864 (2006.01) C12N 15/867 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2479167

Disclosed are methods for the use of therapeutic polypeptide-encoding polynucleotides in the creation of transformed host cells and transgenic animals is disclosed. In particular, the use of recombinant adeno-associated viral (rAAV) vector compositions comprising polynucleotide sequences that express one or more mammalian PEDF or anti-angiogenesis polypeptides is described. In particular, the invention provides gene therapy methods for the prevention, long-term treatment and/or amelioration of symptoms of a variety of conditions and disorders in a mammalian eye, including, for example blindness, loss of vision, retinal degeneration, macular degeneration, and related disorders resulting from retinal or choroidal neovascularization in affected individuals.

L'invention concerne des procédés relatifs à l'utilisation de polynucléotides codant des polypeptides, dans un cadre thérapeutique, pour la création de cellules hôtes transformées et d'animaux transgéniques. En particulier, on décrit l'utilisation de compositions à base de vecteur rAAV comprenant des séquences polynucléotidiques qui expriment un ou plusieurs polypeptides de facteur pigmentaire dérivé de l'épithélium (PEDF) ou antiangiogéniques de type mammalien. L'invention concerne spécifiquement des procédés de thérapie génique pour la prévention, le traitement à long terme et/ou l'atténuation des symptômes correspondant à un éventail d'états et de troubles qui affectent l'oeil mammalien, à savoir par exemple la cécité, la perte de vision, la dégénérescence rétinienne, la dégénérescence maculaire et autres troubles connexes résultant de la néovascularisation rétinienne ou choroïdienne chez les personnes concernées.

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