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.
Claims21
1-30. (canceled)
A method of treating RLBP1-associated retinal dystrophy comprising administering to a subject in need thereof an effective amount of a composition comprising an adeno-associated virus (AAV) vector, wherein the AAV vector comprises a vector genome comprising:
- a) retinaldehyde binding protein 1 (RLBP1) coding sequence;
- and b) an AAV capsid.
Dependencies are inferred from the claim text.