C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/864 (2006.01) A61K 48/00 (2006.01)
Patent
CA 2720178
A method for the treatment of diseases associated with mutations in MY07A or CEP290 genes, especially the Usher Syndrome type IB and Leber congenital amaurosis, by administering to a subject in need thereof an adeno-associated viral vector encoding a MYO7A or a CEP290 protein; genetic constructs and adeno- associated viral vectors for use in this method.
L'invention concerne une méthode de traitement de maladies associées à des mutations des gènes MY07A ou CEP290, notamment le syndrome d'Usher du type IB et l'amaurose congénitale de Leber, qui consiste à administrer à un sujet nécessitant un tel traitement un vecteur de virus associé aux adénovirus qui code pour une protéine de MYO7A ou de CEP290. L'invention concerne aussi des gènes hybrides et des vecteurs de virus associé aux adénovirus s'utilisant dans le procédé.
Fondazione Telethon
Kirby Eades Gale Baker
LandOfFree
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