Method of treating or retarding the development of blindness

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United States of America Patent

PATENT NO 9433688
APP PUB NO 20140377224A1
SERIAL NO

14310015

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Abstract

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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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Patent Owner(s)

  • CORNELL UNIVERSITY;UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.;THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

International Classification(s)

Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Acland, Gregory M Kennett Square, US 18 91
Aguirre, Gustavo D Philadelphia, US 20 58
Bennett, Jean Bryn Mawr, US 48 426
Hauswirth, William W Gainesville, US 44 658
Jacobson, Samuel G Penn Valley, US 15 84
Maguire, Albert M Bryn Mawr, US 9 74

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