Targeting of microRNAs for therapeutics
Targeting of microRNAs for therapeutics
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DOI：
10.1042/bst0361197
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发表时间：
2008-12-01
影响因子：
3.9
通讯作者：
Kauppinen, Sakari
中科院分区：
文献类型：
--
作者：
Stenvang, Jan;Lindow, Morten;Kauppinen, Sakari
miRNA（microRNA）包括一类小的内源性非编码RNA，其通过与其靶mRNA的碱基配对在转录后抑制基因表达。最近的证据表明，miRNAs在多种人类疾病中发挥重要作用，如病毒感染、癌症和心血管疾病，因此miRNAs已迅速成为潜在的治疗靶点。LNA（锁核酸）包括一类RNA的双环构象类似物，其表现出对互补RNA分子的高结合亲和力以及在体内血液和组织中的高稳定性。最近关于啮齿类和灵长类动物中LNA介导的miRNA沉默的报道支持LNA修饰的寡核苷酸在体内研究miRNA功能和未来基于miRNA的疗法开发中的潜力。
miRNAs (microRNAs) comprise a class of small endogenous non-coding RNAs that post-transcriptionally repress gene expression by base-pairing with their target mRNAs. Recent evidence has shown that miRNAs play important roles in a wide variety of human diseases, such as viral infections, cancer and cardiovascular diseases, and thus miRNAs have rapidly emerged as potential targets for therapeutics. LNAs (locked nucleic acids) comprise a class of bicyclic conformational analogues of RNA, which exhibit high binding affinity to complementary RNA molecules and high stability in blood and tissues in vivo. Recent reports on LNA-mediated miRNA silencing in rodents and primates support the potential of LNA-modified oligonucleotides in studying miRNA functions in vivo and in the future development of miRNA-based therapeutics.