BELTRAN WILLIAM A.,AGUIRRE GUSTAVO D.,JACOBSON SAMUEL G.,CIDECIYAN ARTUR V.,LEWIN ALFRED S.,BOYE SANFORD L.,HAUSWIRTH WILLIAM W.,DENG WEN-TAO
申请号：
NZ70427513
公开号：
NZ704275A
申请日:
2013.01.23
申请国别(地区):
NZ
年份:
2016
代理人:
摘要：
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.
