Adeno-associated virus-delivered ribozyme compositions and...

C - Chemistry – Metallurgy – 12 – Q

Patent

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C12Q 1/68 (2006.01) A01K 67/00 (2006.01) C12N 15/10 (2006.01) C12N 15/11 (2006.01)

Patent

CA 2368194

Disclosed are methods for the identification of novel genes involved in a variety of cellular processes, including retinal degeneration, retinal disease, cancer, memory and learning, amylotropic lateral sclerosis, and methods for the identification of the function of a variety of genes and gene fragments of unknown function. Methods are also diclosed for making non-human animal models of diseases by in introduction of selected ribozymes to cleave target genes. The genes thus identified, as well as the compositions used in the identification methods, are also provided.

L'invention concerne des procédés pour l'identification de nouveaux gènes impliqués dans divers processus cellulaires, notamment la dégénérescence rétinienne, les maladies de la rétine, le cancer, la mémoire et l'apprentissage, la sclérose latérale amyotrophique, ainsi que des procédés pour l'identification de la fonction de divers gènes et fragments de gènes de fonction inconnue. L'invention concerne également des procédés permettant de réaliser des modèles animaux non humains de maladies par introduction de ribozymes sélectionnés destinés à couper des gènes cibles. L'invention concerne enfin les gènes ainsi identifiés ainsi que les compositions utilisées dans les procédés d'identification.

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