RNA and condensates: Disease implications and therapeutic opportunities.
| Autor: | Han TW; Dewpoint Therapeutics, Boston, MA, USA., Portz B; Dewpoint Therapeutics, Boston, MA, USA., Young RA; Whitehead Institute for Biomedical Research, Cambridge, MA 02142, USA; Department of Biology, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Boija A; Dewpoint Therapeutics, Boston, MA, USA. Electronic address: aboija@dewpointx.com., Klein IA; Dewpoint Therapeutics, Boston, MA, USA. Electronic address: iklein@dewpointx.com. |
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| Jazyk: | angličtina |
| Zdroj: | Cell chemical biology [Cell Chem Biol] 2024 Sep 19; Vol. 31 (9), pp. 1593-1609. |
| DOI: | 10.1016/j.chembiol.2024.08.009 |
| Abstrakt: | Biomolecular condensates are dynamic membraneless organelles that compartmentalize proteins and RNA molecules to regulate key cellular processes. Diverse RNA species exert their effects on the cell by their roles in condensate formation and function. RNA abnormalities such as overexpression, modification, and mislocalization can lead to pathological condensate behaviors that drive various diseases, including cancer, neurological disorders, and infections. Here, we review RNA's role in condensate biology, describe the mechanisms of RNA-induced condensate dysregulation, note the implications for disease pathogenesis, and discuss novel therapeutic strategies. Emerging approaches to targeting RNA within condensates, including small molecules and RNA-based therapies that leverage the unique properties of condensates, may revolutionize treatment for complex diseases. Competing Interests: Declaration of interests T.W.H. and B.P. are employees and shareholders of Dewpoint Therapeutics. A.B. and I.K. are employees and shareholders of Dewpoint Therapeutics and hold multiple patents related to the work discussed in this review. R.A.Y. is a founder and shareholder of Syros Pharmaceuticals, Camp4 Therapeutics, Omega Therapeutics, Dewpoint Therapeutics, and Paratus Sciences, has consulting or advisory roles at Precede Biosciences and Novo Nordisk, and holds multiple patents related to the work discussed in this review. (Copyright © 2024 The Author(s). Published by Elsevier Ltd.. All rights reserved.) |
| Databáze: | MEDLINE |
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