Self complementary aav-mediated delivery of interfering rna...

Details Self complementary aav-mediated delivery of interfering rna... Self complementary aav-mediated delivery of interfering rna...

A - Human Necessities

61

K

A61K 48/00 (2006.01)

Patent

CA 2694091

The invention provides methods for delivering interfering RNA molecules to an eye of a patient to treat ocular disorders. In particular, the methods of the invention comprise the use of a self-complementary adeno-associated (scAAV) viral vector that can deliver an interfering RNA molecule to an eye of a patient to inhibit expression of a gene that is associated with an ocular disorder.

L'invention concerne des procédés d'administration de molécules d'ARN interférentes à l'il d'un patient pour traiter des troubles oculaires. En particulier, les procédés décrits par l'invention comprennent l'utilisation d'un vecteur viral adénoassocié autocomplémentaire (scAAV) qui peut administrer une molécule d'ARN interférente à l'il d'un patient pour inhiber l'expression d'un gène associé à un trouble oculaire.

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