University of Florida Research Foundation Inc

University of Pennsylvania

Wen-Tao Deng

William W Hauswirth

Alfred Samuel Lewin

Gustavo D Aguirre

Sanford Leon Boye

William Beltran

Samuel Jacobson

Artur V Cideciyan

Sanford L Boye

William A Beltran

Samuel G Jacobson

Alfred S Lewin

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 the 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.

AAV-mediated gene therapy for RPGR X-linked retinal degeneration

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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Gainesville, FL, United States of America

Wen-Tao Deng