发明名称 METHODS FOR TREATING EYE DISORDERS
摘要 The present invention relates to compositions and methods for inhibiting loss of a retinal ganglion cell in a subject, comprising non-invasively applying to the surface of the eye of the subject an ophthalmic composition comprising a therapeutically effective amount of at least one siRNA which down regulates expression of a target gene associated with loss of the retinal ganglion cell, thereby inhibiting loss of the retinal ganglion cell in the subject. The methods of the invention also relate to the use of chemically modified siRNA compounds possessing structural motifs which down-regulate the expression of human genes expressed in retinal tissue in the mammalian eye.
申请公布号 US2015361430(A1) 申请公布日期 2015.12.17
申请号 US201514837366 申请日期 2015.08.27
申请人 Quark Pharmaceuticals, Inc. 发明人 Feinstein Elena;Alpert Evgenia;Mett Igor;Bar-Ilan Amir;Spivak Igor;Kalinski Hagar;Slager Natanja
分类号 C12N15/113;A61K9/00 主分类号 C12N15/113
代理机构 代理人
主权项 1. A method for inhibiting loss of a retinal ganglion cell in a subject, comprising non-invasively applying to the surface of the eye of the subject an ophthalmic composition comprising a therapeutically effective amount of at least one siRNA which down regulates expression of a target gene associated with loss of the retinal ganglion cell, thereby inhibiting loss of the retinal ganglion cell in the subject.
地址 Fremont CA US