发明名称 GENE THERAPY FOR RETINITIS PIGMENTOSA
摘要 Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.
申请公布号 WO2015143418(A2) 申请公布日期 2015.09.24
申请号 WO2015US21896 申请日期 2015.03.20
申请人 GENZYME CORPORATION 发明人 O'RIORDAN, CATHERINE;ADAMOWICZ, MATTHEW
分类号 A61K48/00 主分类号 A61K48/00
代理机构 代理人
主权项
地址