The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Sep. 06, 2016

Filed:

Jun. 20, 2014
Applicants:

The Trustees of the University of Pennsylvania, Philadelphia, PA (US);

University of Florida Research Foundation, Incorporated, Gainesville, FL (US);

Cornell Research Foundation, Inc., Ithaca, NY (US);

Inventors:

Gregory M. Acland, Kennett Square, PA (US);

Gustavo D. Aguirre, Philadelphia, PA (US);

Jean Bennett, Bryn Mawr, PA (US);

William W. Hauswirth, Gainesville, FL (US);

Samuel G. Jacobson, Penn Valley, PA (US);

Albert M. Maguire, Bryn Mawr, PA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C12N 15/12 (2006.01); C12N 15/861 (2006.01); A61K 31/70 (2006.01); C07K 14/705 (2006.01); C12N 15/86 (2006.01); A61K 38/51 (2006.01); A61K 38/52 (2006.01); C12N 15/864 (2006.01); C12N 7/00 (2006.01); A61K 38/00 (2006.01);
U.S. Cl.
CPC ...
A61K 48/0058 (2013.01); A61K 31/70 (2013.01); A61K 38/51 (2013.01); A61K 38/52 (2013.01); A61K 48/0075 (2013.01); C07K 14/705 (2013.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); C12N 15/8645 (2013.01); A61K 38/00 (2013.01); A61K 48/00 (2013.01); C12N 2750/14132 (2013.01); C12N 2750/14142 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01); C12N 2830/008 (2013.01); C12N 2830/85 (2013.01); C12N 2840/203 (2013.01);
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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