Snøve Ola, Rossi John J
Division of Molecular Biology, Beckman Research Institute of the City of Hope, Duarte, California 91010, USA.
Nat Methods. 2006 Sep;3(9):689-95. doi: 10.1038/nmeth927.
Promoter-based expression of short hairpin RNAs (shRNAs) may in principle provide stable silencing of genes in any tissue. As for all approaches that require transgene expression, safe delivery is the biggest obstacle, but toxicity can also occur via expression of the sequence itself. Innate immunity mechanisms can be triggered by expressed hairpin RNAs, critical cellular factors can be saturated, and genes other than the intended target can be silenced. Nevertheless, shRNAs constitute a valuable tool for in vivo research and have great therapeutic potential if the challenges with delivery and side effects are appropriately addressed.
基于启动子的短发夹RNA（shRNA）表达原则上可在任何组织中实现基因的稳定沉默。对于所有需要转基因表达的方法而言，安全递送是最大的障碍，但毒性也可能通过序列本身的表达而产生。表达的发夹RNA可触发天然免疫机制，关键细胞因子可能会饱和，并且非预期靶标的其他基因也可能被沉默。尽管如此，如果能够妥善解决递送和副作用方面的挑战，shRNA仍是体内研究的宝贵工具，具有巨大的治疗潜力。
