Novel Hydrophobically Modified Asymmetric RNAi Compounds (sd-rxRNA) Demonstrate Robust Efficacy in the Eye
Novel Hydrophobically Modified Asymmetric RNAi Compounds (sd-rxRNA) Demonstrate Robust Efficacy in the Eye
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DOI：
10.1089/jop.2013.0148
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发表时间：
2013-12-01
影响因子：
2.3
通讯作者：
Pavco, Pamela
中科院分区：
文献类型：
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作者：
Byrne, Michael;Tzekov, Radouil;Pavco, Pamela
目的：开发RNAi治疗剂的主要挑战包括有效递送和进入感兴趣的细胞类型。传统的（裸露的和化学稳定的）小干扰RNA（siRNA）在过去已经用于眼睛，但它们表现出有限的临床疗效。在这里，我们研究了最近开发的一类小的，疏水的，不对称的RNAi化合物。这些化合物被称为自递送rxRNA（sd-rxRNA（（R），被广泛修饰，具有小的双链体区域，
Purpose: The major challenges of developing an RNAi therapeutic include efficient delivery to and entry into the cell type of interest. Conventional (naked and chemically stabilized) small interfering RNAs (siRNAs) have been used in the eye in the past but they demonstrated limited clinical efficacy. Here we investigated a recently developed class of small, hydrophobic, asymmetric RNAi compounds. These compounds, termed self-delivering rxRNAs (sd-rxRNA((R))), are extensively modified, have a small duplex region of