Promoter specific for non-pigmented ciliary epithelial cells
AssigneesThe Board of Trustees of the Leland Stanford Junior University, The United States Government as represented by the Department of Veterans Affairs
InventorsVinit Mahajan, Katherine J. Wert
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Abstract
A construct comprising a promoter specific for non-pigmented ciliary epithelial cells (NPCECs) is provided. In particular, the construct comprises a BEST2 minimal promoter that confers NPCEC-specific expression. The BEST2 minimal promoter may be operably linked to an expressible sequence such as a gene encoding a polypeptide of interest, a regulatory RNA sequence, a reporter gene, and the like. Such constructs may be provided in an expression vector, for example, with the BEST2 minimal promoter operably linked to an expressible sequence or in a host cell genetically modified with such an expression vector.
Claims25
A recombinant promoter construct comprising a minimal BEST2 promoter and a transcriptional start site (TSS), wherein the recombinant promoter construct is transcriptionally active in non-pigmented ciliary epithelial cells (NPCECs) and not in retinal pigment epithelial cells, wherein the recombinant promoter construct has a non-native configuration with a non-native spacing between the minimal BEST2 promoter and the TSS, and
- wherein the minimal BEST2 promoter consists of:
- a) the nucleotide sequence corresponding to positions 12,751,703-12,751,803 of human chromosome 19,
- b) the nucleotide sequence of SEO ID NO:
- 1, or
- c) a nucleotide sequence having at least 95% sequence identity to the nucleotide sequence of SEO ID NO:
- 1, wherein the promoter is capable of NPCEC-specific transcription.
Dependencies are inferred from the claim text.