Inventors:
The Trustees of the University of Pennsylvania - , US
University of Florida Research Foundation, Incorporated - , US
William W. Hauswirth - Gainesville FL, US
Samuel G. Jacobson - Penn Valley PA, US
Albert M. Maguire - Bryn Mawr PA, US
Assignee:
The Trustees of the University of Pennsylvania - Philadelphia PA
Cornell Research Foundation, Inc. - Ithaca NY
University of Florida Research Foundation, Incorporated - Gainesville FL
International Classification:
A61K 38/52, C12N 15/864
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.