Raav-vectored ribozyme compositions and methods for the...

C - Chemistry – Metallurgy – 12 – N

Patent

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C12N 15/55 (2006.01) A61K 31/7088 (2006.01) A61K 38/46 (2006.01) A61K 48/00 (2006.01) A61P 27/02 (2006.01) C12N 9/22 (2006.01) C12N 15/11 (2006.01) C12N 15/63 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2446101

Disclosed are ribozymes, as well as compositions, vectors, virus particles, host cells, and therapeutic kits that comprise them, which have been shown to be useful in the manufacture of medicaments and in the treatment of diseases of the mammalian eye, and in particular retinopathy and macular degeneration. Also disclosed are methods and uses of these compositions in the reduction, elimination, and/or amelioration of symptoms of diseases of the human eye, including for example, loss of vision, retinitis, retinopathy, and blindness.

L'invention concerne des ribozymes, ainsi que des compositions, des vecteurs, des particules de virus, des cellules hôtes, et des kits thérapeutiques englobant ces produits, lesquels sont utiles pour l'élaboration de médicaments et le traitement de maladies de l'oeil chez un mammifère, en particulier la rétinopathie et la dégénérescence maculaire. L'invention concerne également des procédés et des utilisations liés à ces compositions, pour la réduction, l'élimination et/ou l'amélioration des symptômes propres aux maladies de l'oeil humain, à savoir entre autres: perte de vision, rétinite, rétinopathie et cécité.

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