Enhanced human opsin promoter for rod specific expression
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsFrank M. Dyka, William W. Hauswirth
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Abstract
The present disclosure provides short enhanced human opsin promoters for rod-specific expression that satisfy a need in the art for promoters that are able to control transgene expression both efficiently and selectively in rod cells, with little to no off-target expression in other photoreceptor cells. The disclosure provides vector constructs comprising transgenes operably controlled by this enhanced human opsin promoter that may be delivered and targeted with high specificity to rod cells. The disclosure also provides compositions comprising these vector constructs and methods for administration of these compositions to subjects in need thereof.
Claims16
A recombinant viral vector comprising a polynucleotide encoding a transgene operably linked to promoter that has a nucleotide sequence having at least 90%, at least 95%, or at least 99% sequence identity to SEQ ID NO:
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Dependencies are inferred from the claim text.