Defective Ribonucleoproteins, Mistakes in RNA Processing, and Diseases

by Mihailovic, MK; Chen, A; Gonzalez-Rivera, JC; Contreras, LM

BIOCHEMISTRY; Volume: 56; Issue: 10; Pages: 1367-1382; DOI: 10.1021/acs.biochem.6b01134; Published: MAR 14 2017

Ribonucleoproteins (RNPs) are vital to many cellular events. To this end, many neurodegenerative diseases and cancers have been linked to RNP malfunction, particularly as this relates to defective processing of cellular RNA. The connection of RNPs and diseases has also propagated a shift of focus onto RNA targeting from traditional protein targeting treatments. However, therapeutic development in this area has been limited by incomplete molecular insight into the specific contributions of RNPs to disease. This review outlines the role of several RNPs in diseases, focusing on molecular defects in processes that affect proper RNA handling in the cell. This work also evaluates the contributions of recently developed methods to understanding RNP association and function. We review progress in this area by focusing on molecular malfunctions of RNPs associated with the onset and progression of several neurodegenerative diseases and cancer and conclude with a brief discussion of RNA-based therapeutic efforts.

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