Optimized RPE65 Promoter and Coding Sequences
AssigneeUCL Business PLC
InventorsAlexander Smith, Robin Ali
Life of this patent
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Abstract
The present disclosure relates to the prevention and/or treatment of retinal dystrophy in a patient, including the prevention and/or treatment of Leber congenital amaurosis (LCA). Provided are retinal pigment epithelium (RPE)-specific promoters, expression constructs comprising RPE-specific promoters and vectors comprising said promoters and expression constructs. Further provided are methods of treating retinal dystrophy in a patient in need thereof.
Claims28
A method of selectively expressing a transgene in the retinal pigment epithelium (RPE), the method comprising contacting the RPE with a vector comprising an expression construct comprising a promoter and an operably linked transgene, wherein:
- (a) the promoter consists of a sequence of no more than 800 contiguous nucleotides from SEQ ID NO:1 comprising nucleotides 12-761 of SEQ ID NO:2;
- (b) the promoter consists of a sequence having at least 90% sequence identity to said sequence of (a);
- (c) the promoter comprises the sequence of SEQ ID NO:
- 2;
- or
- (d) the promoter consists of a sequence having at least 90% sequence identity to said sequence of SEQ ID NO:
- 2, wherein the sequence of the promoter is no longer than 800 nucleotides.
A method of selectively expressing a transgene in the RPE of a patient in need thereof, the method comprising administering to the patient via direct retinal, subretinal, or intravitreal injection a vector comprising an expression construct comprising a promoter and an operably linked transgene, wherein:
- (a) the promoter consists of a sequence of no more than 800 contiguous nucleotides from SEQ ID NO:1 comprising nucleotides 12-761 of SEQ ID NO:2;
- (b) the promoter consists of a sequence having at least 90% sequence identity to said sequence of (a);
- (c) the promoter comprises the sequence of SEQ ID NO:
- 2;
- or
- (d) the promoter consists of a sequence having at least 90% sequence identity to said sequence of SEQ ID NO:
- 2, wherein the sequence of the promoter is no longer than 800 nucleotides.
Dependencies are inferred from the claim text.