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US 2018/0021458 A1Published application
Optimized RPE65 Promoter and Coding Sequences
AssigneeUCL Business PLC
InventorsAlexander Smith, Robin Ali
Life of this patent
Estimating the term…
Abstract
The present invention relates to the prevention and/or treatment of retinal dystrophyin a patient, including Leber congenital amaurosis (LCA).
Claims25
A retinal pigment epithelium (RPE)-specific promoter which comprises:
- (a) a sequence of contiguous nucleotides from SEQ ID NO:1 that confers RPE-specific expression on an operably linked polynucleotide sequence, or (b) a sequence having at least 90% sequence identity to said sequence of (a) and that retains RPE-specific promoter activity.
The promoter of any one of the preceding claims, wherein said sequence of (a) or (b) is at least 500 nucleotides in length.
An expression construct comprising the promoter of any one of the preceding claims, operably linked to a sequence to be expressed in an RPE-specific manner.
The vector according 11, which is an adeno-associated virus (AAV) vector or comprises an AAV genome or a derivative thereof.
Dependencies are inferred from the claim text.