RNA dysregulation in neurodegenerative diseases

Yini Li; Shuying Sun

doi:10.1038/s44318-024-00352-6

RNA dysregulation in neurodegenerative diseases

EMBO J. 2025 Jan 9. doi: 10.1038/s44318-024-00352-6. Online ahead of print.

Authors

Yini Li^{1

2}, Shuying Sun^{3

4

5}

Affiliations

¹ Department of Physiology, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.
² Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.
³ Department of Physiology, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA. shuying.sun@jhmi.edu.
⁴ Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA. shuying.sun@jhmi.edu.
⁵ Departments of Neuroscience, Pathology, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA. shuying.sun@jhmi.edu.

PMID: 39789319
DOI: 10.1038/s44318-024-00352-6

Abstract

Dysregulation of RNA processing has in recent years emerged as a significant contributor to neurodegeneration. The diverse mechanisms and molecular functions underlying RNA processing underscore the essential role of RNA regulation in maintaining neuronal health and function. RNA molecules are bound by RNA-binding proteins (RBPs), and interactions between RNAs and RBPs are commonly affected in neurodegeneration. In this review, we highlight recent progress in understanding dysregulated RNA-processing pathways and the causes of RBP dysfunction across various neurodegenerative diseases. We discuss both established and emerging mechanisms of RNA-mediated neuropathogenesis in this rapidly evolving field. Furthermore, we explore the development of potential RNA-targeting therapeutic approaches for the treatment of neurodegenerative diseases.

Keywords: Neurodegenerative Diseases; RNA Metabolism; RNA-binding Protein; RNA-targeting Therapeutics.

Publication types

Review

Abstract

Publication types

Grants and funding