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UNA oligomers having reduced off-target effects in gene silencing

机译:UNA低聚物在基因沉默中的脱靶效应降低

摘要

This invention provides UNA oligomers for gene silencing with reduced off-target effects. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.
机译:本发明提供了具有降低的脱靶作用的用于基因沉默的UNA寡聚物。 UNA低聚物可具有第一链和第二链,每条链的长度为19-29个单体,所述单体为UNA单体和各种核酸单体。实施方案包括通过向对象施用UNA寡聚物来治疗或预防脱靶效应降低的TTR相关淀粉样变性的药物组合物和方法。

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