miR-Synth: a computational resource for the design of multi-site multi-target synthetic miRNAs.

miR-Synth: a computational resource for the design of multi-site multi-target synthetic miRNAs.
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DOI:
10.1093/nar/gku202
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发表时间:
2014-05
影响因子:
14.9
通讯作者:
Croce CM
Croce CM
中科院分区:
生物学2区
文献类型:
--
作者:
Laganà A;Acunzo M;Romano G;Pulvirenti A;Veneziano D;Cascione L;Giugno R;Gasparini P;Shasha D;Ferro A;Croce CM

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RNAi是调控基因表达的有力工具。它被广泛和成功地应用于功能研究,现在正在成为一个有前途的治疗方法。一些基于RNAi的临床试验表明,在治疗包括癌症在内的各种疾病方面取得了令人鼓舞的结果。在这里,我们提出了miR-Synth,一种用于设计合成microRNA的计算资源,能够在多个位点靶向多个基因。所提出的策略构成了使用siRNA的有效替代方案,允许使用较少数量的分子来抑制多个靶标。这可能代表了在设计治疗由多个失调基因改变的关键细胞途径引起的疾病的疗法方面的巨大优势。该系统已成功验证了两个最突出的肺癌相关基因,c-MET和表皮生长因子受体(EGFR)。(See http://microrna.osumc.edu/mir-synth).
RNAi is a powerful tool for the regulation of gene expression. It is widely and successfully employed in functional studies and is now emerging as a promising therapeutic approach. Several RNAi-based clinical trials suggest encouraging results in the treatment of a variety of diseases, including cancer. Here we present miR-Synth, a computational resource for the design of synthetic microRNAs able to target multiple genes in multiple sites. The proposed strategy constitutes a valid alternative to the use of siRNA, allowing the employment of a fewer number of molecules for the inhibition of multiple targets. This may represent a great advantage in designing therapies for diseases caused by crucial cellular pathways altered by multiple dysregulated genes. The system has been successfully validated on two of the most prominent genes associated to lung cancer, c-MET and Epidermal Growth Factor Receptor (EGFR). (See http://microrna.osumc.edu/mir-synth).
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