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US 7,740,836 B2Granted
Methods and compositions for recovering or improving visual function
AssigneeFondazione Telethon
InventorsAlberto Auricchio, Enrico Maria Surace, Andrea Ballabio
Life of this patent
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Abstract
There are disclosed methods and compositions for recovering or improving visual function in a mammal, by means of adeno-associated viral vectors suitable for gene delivery to mammalian retina.
Claims6
A method for increasing an amount of melanosomes in a mammalian subject suffering from ocular albinism type I as compared to a non-treated subject, said method comprising:
- a) providing a recombinant adeno-associated viral (AAV) vector carrying an expression cassette which contains a nucleic acid molecule encoding an orphan G-protein coupled receptor (OA1) that crosses melanosomal membranes, wherein said nucleic acid molecule is selected from the group consisting of SEQ ID Nos. 1 and 2, and wherein said nucleic acid molecule is operably linked to a promoter selected from the group consisting of SEQ ID Nos. 3, 4, and 5;
- and
- b) transducing retinal pigment epithelial cells with said recombinant AAV vector by administering said vector by subretinal injection, whereby said nucleic acid molecule is expressed by the retinal pigment epithelial cells in said mammalian subject.
Dependencies are inferred from the claim text.