发明名称 MIRNA MIMETICS AND THEIR USE IN TREATING SENSORY CONDITIONS
摘要 The present invention provides microRNA mimetic compounds that mimic the function or activity of miR-96, miR-182, and/or miR-183. The microRNA mimetic compounds of the invention comprise a first strand of about (22) to about (26) ribonucleotides comprising a mature miR-96, miR-182, or miR-183 sequence; and a second strand of about (20) to about (24) ribonucleotides comprising a sequence that is substantially complementary to the first strand and having at least one modified nucleotide, wherein the first strand has a 3' nucleotide overhang relative to the second strand. The invention additionally provides expression vectors comprising a polynucleotide(s) encoding one or more of miR-96, miR-182, and miR-183. The invention also provides methods of treating ophthalmological or otic conditions by administering the microRNA mimetic compounds of miR-96, miR-182, and/or miR-183 and/or an expression vector encoding at least one of miR-96, miR-182, and miR-183 to a subject in need thereof.
申请公布号 WO2016149370(A1) 申请公布日期 2016.09.22
申请号 WO2016US22645 申请日期 2016.03.16
申请人 MIRAGEN THERAPEUTICS, INC. 发明人 JACKSON, Aimee;DALBY, Christina
分类号 C12N15/86;C12N15/113 主分类号 C12N15/86
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