Oxford University Innovation Limited;NightstaRx Limited
发明人:
Robert MACLAREN,Christina MARTINEZ-FERNANDEZ DE LA CAMAR,Gregory S. ROBINSON
申请号：
US16376808
公开号：
US20190307900A1
申请日:
2019.04.05
申请国别(地区):
US
年份:
2019
代理人:
摘要：
The disclosure provides composition comprising a nucleic acid sequence comprising (a) a sequence encoding a vitelliform macular dystrophy-2 (VMD2) promoter, and (b) a sequence encoding a Bestrophin-1 (BEST1) protein as well as the use of these compositions for the treatment of macular dystrophy in a subject comprising administration of the composition to an eye of a subject via a subretinal or a suprachoroidal route.
