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US 2020/0390907 A1Published application
Methods and compositions for treating genetically linked diseases of the eye
AssigneeThe U.S.A., as represented by the Secretary, Department of Health and Human Services
InventorsPaul Albert SIEVING, Ronald Avery BUSH, Peter C. COLOSI, Yong ZENG
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Abstract
Expression vectors and therapeutic methods of using such vectors in the treatment of diseases of the eye resulting from failure to produce a specific protein in the eye, or the production of a non-functional protein in the eye.
Claims16
A method of treating an eye disease or disorder in a human comprising:
- administering to a human subject diagnosed with or suspected of having a disease, disorder, or injury of an eye, a therapeutically effective amount of an expression vector comprising:
- an expression vector comprising an expression cassette, wherein the expression cassette comprises a promoter operably-linked to a nucleic acid sequence encoding a retinoschisin gene, including at least a 319-base pair portion of the first intron of the retinoschisin gene,
- wherein administration of the vector causes expression of the human retinoschisin protein in a retinal cell of the human subject, and reduces at least one symptom of the disease, disorder, or injury of the eye.
The method of claim 15, wherein the administration of the anti-inflammatory agent continues for at least 30 days after the administration of the expression vector.
Dependencies are inferred from the claim text.