- Patent Title: AAV-mediated gene therapy for RPGR X-linked retinal degeneration
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Application No.: US14413884Application Date: 2013-01-23
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Publication No.: US09770491B2Publication Date: 2017-09-26
- Inventor: William A Beltran , Gustavo D Aguirre , Samuel G Jacobson , Artur V Cideciyan , Alfred S Lewin , Sanford L Boye , William W Hauswirth , Wen-Tao Deng
- Applicant: The Trustees of the University of Pennsylvania , University of Florida Research Foundation, Inc.
- Applicant Address: US PA Philadelphia US FL Gainesville
- Assignee: The Trustees of the University of Pennsylvania,Univeristy of Florida Research Foundation, Incorporated
- Current Assignee: The Trustees of the University of Pennsylvania,Univeristy of Florida Research Foundation, Incorporated
- Current Assignee Address: US PA Philadelphia US FL Gainesville
- Agency: Howson & Howson LLP
- International Application: PCT/US2013/022628 WO 20130123
- International Announcement: WO2014/011210 WO 20140116
- Main IPC: C07H21/04
- IPC: C07H21/04 ; A61K48/00 ; A61K38/46 ; C07K14/47 ; A61K38/17 ; A61K38/18 ; C12N15/86 ; A01K67/00

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.
Public/Granted literature
- US20150202269A1 AAV-MEDIATED GENE THERAPY FOR RPGR X-LINKED RETINAL DEGENERATION Public/Granted day:2015-07-23
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