Viral vectors for the treatment of retinal dystrophy
AssigneeNovartis AG
InventorsVivian Choi, Chad Eric Bigelow, Thaddeus Peter Dryja, Seshidhar Reddy Police
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Abstract
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.
Claims23
A method of expressing a heterologous gene in retinal cells, the method comprising contacting said retinal cells with a viral vector comprising:
- a) an adeno-associated virus (AAV) serotype 2 or 8 capsid, and b) a vector genome comprising a retinaldehyde binding protein 1 (RLBP1) promoter comprising the nucleotide sequence of SEQ ID NO:3 or SEQ ID NO:10 operably linked to the heterologous gene.
A method of expressing an RLBP1 coding sequence in retinal pigment epithelium (RPE) cells and Müller cells in the retina of a subject having RLBP1-associated retinal dystrophy, the method comprising contacting the retina of the subject with a viral vector comprising:
- a vector genome comprising the nucleotide sequences of SEQ ID NOs:5, 6, 8, and 9;
- and
- an adeno-associated virus (AAV) serotype 2 or 8 capsid.
Dependencies are inferred from the claim text.