发明名称 RNA interference in ocular indications
摘要 The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.
申请公布号 US9095504(B2) 申请公布日期 2015.08.04
申请号 US201113636748 申请日期 2011.03.24
申请人 发明人 Libertine Lyn;Khvorova Anastasia;Salomon William;Kamens Joanne;Samarsky Dmitry;Woolf Tod M.;Cardia James
分类号 C12N15/11;A61K9/00;A61K31/713;A61K45/06 主分类号 C12N15/11
代理机构 Wolf, Greenfield & Sacks, P.C. 代理人 Wolf, Greenfield & Sacks, P.C.
主权项 1. A method for delivering a nucleic acid to an eye of a subject in need thereof, comprising administering to the eye of the subject an sd-rxRNA®, in an effective amount to promote RNA interference by the sd-rxRNA® in the eye, wherein the sd-rxRNA® comprises a guide strand and a passenger strand, wherein the sd-rxRNA® includes a double stranded region and a single stranded region, wherein the double stranded region is from 8-15 nucleotides long, wherein the single stranded region is at the 3′ end of the guide strand and is 4-12 nucleotides long, wherein the single stranded region contains 3, 4, 5, 6, 7, 8, 9, 10, 11 or 12 phosphorothioate modifications, wherein at least 40% of the nucleotides of the isolated double stranded nucleic acid molecule are modified, wherein the sd-rxRNA® is directed against a gene encoding VEGF or CTGF, and wherein the sd-rxRNA® comprises at least 12 contiguous nucleotides of a sequence selected from the sequences within Tables 5, 8 or 10.
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