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    • 6. 发明申请
    • MIRNA MIMETICS AND THEIR USE IN TREATING SENSORY CONDITIONS
    • MIRNA MIME及其在处理传感条件中的应用
    • WO2016149370A1
    • 2016-09-22
    • PCT/US2016/022645
    • 2016-03-16
    • MIRAGEN THERAPEUTICS, INC.
    • JACKSON, AimeeDALBY, Christina
    • C12N15/86C12N15/113
    • C12N15/113A61K31/7088A61K31/7105A61K48/00C12N2310/14C12N2310/141C12N2310/315C12N2310/32C12N2310/321C12N2310/3515C12N2320/31C12N2310/322C12N2310/3533C12N2310/3521
    • 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.
    • 本发明提供了模拟miR-96,miR-182和/或miR-183的功能或活性的微小RNA模拟化合物。 本发明的微RNA模拟化合物包含包含成熟miR-96,miR-182或miR-183序列的约(22)至约(26)个核糖核苷酸的第一链; 和约(20)至约(24)个核糖核苷酸的第二链,其包含与第一链基本上互补并且具有至少一个修饰的核苷酸的序列,其中第一链相对于第二链具有3'核苷酸突出端。 本发明另外提供了包含编码miR-96,miR-182和miR-183中的一种或多种的多核苷酸的表达载体。 本发明还提供了通过施用miR-96,miR-182和/或miR-183的微小RNA模拟化合物和/或编码miR-96,miR-182中的至少一种的表达载体来治疗眼科或耳部病症的方法 ,和miR-183与需要的对象相关联。