Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders

Kathryn H Morelli; Aaron A Smargon; Gene W Yeo

doi:10.1261/rna.079519.122

Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders

RNA. 2023 Apr;29(4):489-497. doi: 10.1261/rna.079519.122. Epub 2023 Jan 24.

Authors

Kathryn H Morelli^{1

2

3}, Aaron A Smargon^{1

2

3}, Gene W Yeo^{4

2

3}

Affiliations

¹ Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, California 92093, USA.
² Stem Cell Program, University of California San Diego, La Jolla, California 92093, USA.
³ Institute for Genomic Medicine, University of California San Diego, La Jolla, California 92039, USA.
⁴ Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, California 92093, USA geneyeo@ucsd.edu.

Abstract

Disruptions in RNA processing play critical roles in the pathogenesis of neurological diseases. In this Perspective, we discuss recent progress in the development of RNA-targeting therapeutic modalities. We focus on progress, limitations, and opportunities in a new generation of therapies engineered from RNA binding proteins and other endogenous RNA regulatory macromolecules to treat human neurological disorders.

Keywords: RNA-targeting therapeutics; gene therapy; neurological disorders; programmable macromolecules.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Humans
Nervous System Diseases* / drug therapy
Nervous System Diseases* / genetics
RNA Processing, Post-Transcriptional
RNA* / genetics

Substances

RNA

Abstract

Publication types

MeSH terms

Substances

Grants and funding