Gene therapy for retinal disease
AssigneeOxford University Innovation Limited
InventorsHarry O. Orlans, Robert E. Maclaren, Michelle E. McClements, Alun R Barnard
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Abstract
A method of treating a retinal disease in a subject in need thereof, the method comprising administering to the subject a vector that comprises a mirtron for knocking down expression of a target gene expressed in the retina and a gene therapy vector comprising a mirtron for rhodopsin knock-down.
Claims8
A method of treating a retinitis pigmentosa in a subject in need thereof, the method comprising administering to the subject a vector that comprises a mirtron for knocking down expression of rhodopsin expressed in the retina, and wherein a guide strand of the mirtron complements a rhodopsin nucleotide sequence selected from SEQ ID NO:
- 5 or SEQ ID NO:
- 7 or a nucleotide sequence having at least 80% sequence identity to SEQ ID NO:
- 5 or SEQ ID NO:
- 7.
Dependencies are inferred from the claim text.