AAV-MEDIATED GENE THERAPY FOR RPGR X-LINKED RETINAL DEGENERATION
摘要
<p>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 said 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.</p>
申请公布号
WO2014011210(A1)
申请公布日期
2014.01.16
申请号
WO2013US22628
申请日期
2013.01.23
申请人
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA;THE UNIVERSITY OF FLORIDA