C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/85 (2006.01) A61K 31/70 (2006.01) A61K 48/00 (2006.01) C12N 9/00 (2006.01) C12N 15/11 (2006.01) C12N 15/86 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2287495
The subject invention provides materials and methods for efficient, specific expression of proteins in retinal photoreceptor cells. Specifically, the constructs are composed of an adeno-associated vital vector containing a rod or cone-opsin promoter. These materials and methods can be used in therapies for retinal diseases. In one embodiment, ribozymes which degrade mutant mRNA are used to treat retinitis pigmentosa.
La présente invention concerne des produits et des traitements suscitant une expression satisfaisante et spécifique de protéines dans les cellules photorécepteurs de la rétine. Plus particulièrement, les produits de recombinaison sont constitués d'un vecteur viral associé aux adénovirus renfermant un promoteur d'opsine des bâtonnets ou des cônes rétiniens. Ces produits et traitement conviennent à des thérapies adaptées à des affections rétiniennes. Selon une réalisation, pour soigner la rétinite pigmentaire, on utilise des ribozymes qui dégradent l'ARNm mutant.
Flannery John G.
Hauswirth William W.
Deeth Williams Wall Llp
Flannery John G.
Hauswirth William W.
University Of Florida
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