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US 10,568,973 B2Granted
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).
Claims21
An expression construct comprising a promoter and an operably linked polynucleotide sequence, wherein the promoter consists of:
- (a) a sequence of no more than 800 contiguous nucleotides from SEQ ID NO:1 comprising nucleotides 12-761 of SEQ ID NO:2, or
- (b) a sequence having at least 90% sequence identity to said sequence of (a);
- and
- wherein the operably linked polynucleotide sequence comprises the sequence of SEQ ID NO:
- 4.
2 more elements
A promoter which:
- (a) comprises the sequence of SEQ ID NO:
- 2, or (b) 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 promoter consisting of:
- (a) a sequence of no more than 800 contiguous nucleotides from SEQ ID NO:1 comprising nucleotides 12-761 of SEQ ID NO:2, or
- (b) a sequence having at least 90% sequence identity to said sequence of (a).
Dependencies are inferred from the claim text.