Developing microRNA Therapeutics: Approaching the Unique Complexities
Developing microRNA Therapeutics: Approaching the Unique Complexities
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DOI：
10.1089/nat.2012.0356
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发表时间：
2012-08-01
影响因子：
4
通讯作者：
Levin, Arthur A.
中科院分区：
文献类型：
--
作者：
Jackson, Aimee L.;Levin, Arthur A.
microRNA是内源性小的非编码RNA，其通过干扰靶转录物的翻译或稳定性来调节基因表达。microRNA对于维持生物学功能的重要性通过以下事实来说明：microRNA在自然界中被利用来调节表型，以及当microRNA突变或不适当表达时导致的多种疾病表型。因此，疾病相关的microRNA可能代表了一类新的治疗靶点。随着最近证明miR-122的抑制降低了丙型肝炎患者的病毒载量，microRNA调节剂不再仅仅是理论上的，而是已经成为强有力的候选治疗剂。microRNA生物学的复杂性为治疗性干预提供了一种新的作用机制，但也为治疗性调节剂作为药物的开发提出了独特的挑战。
MicroRNAs are endogenous small non-coding RNAs that regulate gene expression by interfering with translation or stability of target transcripts. The importance of microRNAs for maintaining biological functions is illustrated by the fact that microRNAs are exploited in nature to regulate phenotypes, and by the diverse disease phenotypes that result when microRNAs are mutated or improperly expressed. Disease-associated microRNAs might therefore represent a new class of therapeutic targets. With the recent demonstration that inhibition of miR-122 reduces viral load in hepatitis C patients, microRNA modulators are no longer merely theoretical, but rather, have become strong candidate therapeutics. The complexity of microRNA biology offers a novel mechanism of action for therapeutic intervention but also poses unique challenges for the development of therapeutic modulators as drugs.