UCL BUSINESS PLC;MASSACHUSETTS EYE & EAR INFIRMARY;THE MEDICAL RESEARCH COUNCIL
发明人:
SANDBERG, MICHAEL A.,PAWLYK, BASIL,WRIGHT, ALAN FINLAY,SHU, XINHUA,LI, TIANSEN,ALI, ROBIN
申请号：
CA2991750
公开号：
CA2991750A1
申请日:
2015.07.17
申请国别(地区):
CA
年份:
2016
代理人:
摘要：
Methods for treating a human subject who has X-linked Retinitis Pigmentosa (XLRP) or another clinically-defined ophthalmological condition due to a loss-of-function mutation in the gene encoding the retinitis pigmentosa GTPase regulator (RPGR) protein, the method comprising administering to the subject a nucleic acid comprising an adeno-associated viral vector comprising an abbreviated human RPGR cDNA.
