RPGRIP1 GENE THERAPY FOR LEBER CONGENITAL AMAUROSIS
摘要
<p>This invention relates to methods for treating subjects with vision loss due to advanced Leber Congenital Amaurosis (LCA), e.g., LCA6, which is due to loss-of- function mutations in the gene encoding the retinitis pigmentosa GTPase regulator interacting-protein-1 (RPGRIP1) protein.</p>
申请公布号
WO2012167109(A2)
申请公布日期
2012.12.06
申请号
WO2012US40498
申请日期
2012.06.01
申请人
MASSACHUSETTS EYE & EAR INFIRMARY;BERSON, ELIOT L.;PAWLYK, BASIL;LI, TIANSEN;SANDBERG, MICHAEL A.
发明人
BERSON, ELIOT L.;PAWLYK, BASIL;LI, TIANSEN;SANDBERG, MICHAEL A.
