Haussecker Dirk
Rastatt, Germany.
FEBS J. 2016 Sep;283(17):3249-60. doi: 10.1111/febs.13742. Epub 2016 May 5.
Both RNA interference (RNAi) and clustered regularly-interspaced short palindromic repeats (CRISPR) technologies allow for the sequence-specific inhibition of gene function and therefore have the potential to be used as therapeutic modalities. By judging the current public and scientific journal interest, it would seem that CRISPR, by enabling clean, durable knockouts, will dominate therapeutic gene inhibition, also at the expense of RNAi. This review aims to look behind prevailing sentiments and to more clearly define the likely scope of the therapeutic applications of the more recently developed CRISPR technology and its relative strengths and weaknesses with regards to RNAi. It is found that largely because of their broadly overlapping delivery constraints, while CRISPR presents formidable competition for DNA-directed RNAi strategies, its impact on RNAi therapeutics triggered by synthetic oligonucleotides will likely be more moderate. Instead, RNAi and genome editing, and in particular CRISPR, are poised to jointly promote a further shift toward sequence-targeted precision medicines.
RNA干扰（RNAi）技术和规律成簇间隔短回文重复序列（CRISPR）技术都能够实现对基因功能的序列特异性抑制，因此都有潜力用作治疗手段。从当前公众及科学期刊的关注度判断，CRISPR技术能够实现彻底、持久的基因敲除，似乎将主导治疗性基因抑制领域，RNAi技术可能会受到冲击。本综述旨在探究这些普遍观点背后的情况，更清晰地界定最新开发的CRISPR技术在治疗应用方面可能的范围，以及其相对于RNAi技术的相对优势和劣势。研究发现，很大程度上由于它们在递送限制方面广泛重叠，虽然CRISPR对DNA导向的RNAi策略构成了巨大竞争，但其对合成寡核苷酸引发的RNAi治疗的影响可能较为温和。相反，RNAi技术和基因组编辑技术，尤其是CRISPR技术，有望共同推动向序列靶向精准药物的进一步转变。
