METHOD OF TREATING OR RETARDING THE DEVELOPMENT OF BLINDNESS
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United States of America Patent
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Issued Date -
Aug 22, 2013
app pub date -
Feb 13, 2013
filing date -
Apr 13, 2001
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Abandoned
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Abstract
A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.
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