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US 11,969,478 B2Granted
Optimized RPE65 promoter and coding sequences
AssigneeUCL Business PLC
InventorsAlexander Smith, Robin Ali
Life of this patent
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Abstract
The present invention relates to the prevention and/or treatment of retinal dystrophy in a patient, including Leber congenital amaurosis (LCA).
Claims12
A method of treating retinal dystrophy caused by Retinal pigment epithelium-specific 65 kDa protein (RPE65) deficiency in a patient in need thereof, the method comprising administering to the patient via direct retinal, subretinal, or intravitreal injection, a therapeutically effective amount of an AAV vector comprising an expression construct comprising a promoter and an operably linked polynucleotide sequence, wherein the promoter consists of:
- (a) nucleotides 12-761 of SEQ ID NO:2, or
- (b) SEQ ID NO:2;
- wherein the operably linked polynucleotide sequence comprises the sequence of SEQ ID NO:
- 4;
- and wherein expression of the operably linked polynucleotide sequence results in treatment of retinal dystrophy in the patient.
2 more elements
Dependencies are inferred from the claim text.