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AssigneeNovartis AG
InventorsChad Eric BIGELOW, Vivian CHOI, Thaddeus Peter DRYJA, Akshata Ninad GUJAR, Shawn Michael HANKS, Terri MCGEE, Seshidhar Reddy POLICE, Joanna VROUVLIANIS
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The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.
A viral vector having a vector genome comprising a retinaldehyde binding protein 1 (RLBP1) coding sequence, wherein said vector is adapted to direct expression of an RLBP1 coding sequence in retinal pigment epithelial (RPE) cells and Muller cells of the retina.
The viral vector of 4, wherein the nucleotide sequence of said promoter is selected from SEQ ID NO:
The viral vector of 8, wherein the nucleotide sequence of said promoter comprises SEQ ID NO:
A viral vector comprising a vector genome capable of expressing a heterologous gene in RPE cells and Muller cells of the retina, said vector comprising a) an AAV8 or AAV2 capsid and b) said vector genome comprising an RLBP1 promoter operably linked to the heterologous gene, wherein said RLBP1 promoter has a sequence selected from SEQ ID NOs:
A composition comprising the viral vector of any of the preceding claims .
A method of expressing a heterologous gene in retinal cells, the method comprising contacting said retinal cells with a viral vector comprising:
A nucleic acid comprising a gene cassette, said gene cassette comprises, in the 5′ to 3′ direction:
Dependencies are inferred from the claim text.