Shukla Siddharth, Parker Roy
Department of Chemistry and Biochemistry, University of Colorado Boulder, Boulder, CO, USA.
Department of Chemistry and Biochemistry, University of Colorado Boulder, Boulder, CO, USA; Howard Hughes Medical Institute, Chevy Chase, MD, USA.
Trends Mol Med. 2016 Jul;22(7):615-628. doi: 10.1016/j.molmed.2016.05.005. Epub 2016 Jun 3.
A key aspect of cellular function is the proper assembly and utilization of ribonucleoproteins (RNPs). Recent studies have shown that hyper- or hypo-assembly of various RNPs can lead to human diseases. Defects in the formation of RNPs lead to 'RNP hypo-assembly diseases', which can be caused by RNA degradation outcompeting RNP assembly. By contrast, excess RNP assembly, either in higher order RNP granules, or due to the expression of repeat-containing RNAs, can lead to 'RNP hyper-assembly diseases'. Here, we discuss the most recent advances in understanding the cause of disease onset, as well as potential therapies from the aspect of modulating RNP assembly in the cell, which presents a novel route to the treatment of these diseases.
细胞功能的一个关键方面是核糖核蛋白(RNP)的正确组装和利用。最近的研究表明,各种RNP的过度组装或组装不足都可能导致人类疾病。RNP形成缺陷会导致“RNP组装不足疾病”,这可能是由RNA降解超过RNP组装所致。相比之下,无论是在高阶RNP颗粒中,还是由于含重复序列RNA的表达导致的RNP过度组装,都可能导致“RNP过度组装疾病”。在这里,我们讨论了在理解疾病发病原因方面的最新进展,以及从调节细胞中RNP组装的角度探讨潜在治疗方法,这为治疗这些疾病提供了一条新途径。