siRNA as a tool for streamlining functional genomic studies

Queta F. Boese, Stephen A. Scaringe, William S. Marshall
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引用次数: 8

Abstract

RNA interference (RNAi) has the potential to accelerate greatly the pace of discovery biology. The active RNAi intermediate is the small interfering RNA (siRNA), a discrete nucleic acid duplex that can be generated by several methods and used to directly silence gene expression. The choice of methods employed depends largely on the research or therapeutic objective. In most cases, rational design offers several advantages over random design, including greater predictability of function, higher silencing potency and longer duration of suppression. Of the production methods, chemical synthesis provides the fastest production capability, the highest purity and the easiest scalability for high-throughput strategies. Effective coupling of several methods gives the greatest potential for the application of RNAi across functional genomic and target validation studies. Furthermore, the coupling of RNAi with cellular profiling technologies will provide opportunities to streamline drug discovery significantly.

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siRNA作为简化功能基因组研究的工具
RNA干扰(RNAi)有可能大大加快发现生物学的步伐。活性RNAi中间体是小干扰RNA (siRNA),这是一种离散的核酸双链,可以通过几种方法生成,并用于直接沉默基因表达。所采用方法的选择在很大程度上取决于研究或治疗目的。在大多数情况下,与随机设计相比,理性设计提供了一些优势,包括更大的功能可预测性,更高的沉默效力和更长的抑制持续时间。在生产方法中,化学合成提供了最快的生产能力,最高的纯度和最容易的高通量策略的可扩展性。几种方法的有效耦合为RNAi在功能基因组和靶标验证研究中的应用提供了最大的潜力。此外,RNAi与细胞分析技术的耦合将为显着简化药物发现提供机会。
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