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. 26, 2017

Filed:

Jan. 23, 2013
Applicants:

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

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

Inventors:

William A Beltran, Philadelphia, PA (US);

Gustavo D Aguirre, Philadelphia, PA (US);

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

Artur V Cideciyan, Philadelphia, PA (US);

Alfred S Lewin, Gainesville, FL (US);

Sanford L Boye, Gainesville, FL (US);

William W Hauswirth, Gainesville, FL (US);

Wen-Tao Deng, Gainesville, FL (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); A61K 48/00 (2006.01); A61K 38/46 (2006.01); C07K 14/47 (2006.01); A61K 38/17 (2006.01); A61K 38/18 (2006.01); C12N 15/86 (2006.01); A01K 67/00 (2006.01);
U.S. Cl.
CPC ...
A61K 38/46 (2013.01); A61K 38/1709 (2013.01); A61K 38/185 (2013.01); A61K 48/005 (2013.01); C07K 14/4702 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01); C12Y 306/05 (2013.01);
Abstract

Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to a subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.


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