Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders
- 1Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, California 92093, USA
- 2Stem Cell Program, University of California San Diego, La Jolla, California 92093, USA
- 3Institute for Genomic Medicine, University of California San Diego, La Jolla, California 92039, USA
- Corresponding author: geneyeo{at}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
This article, published in RNA, is available under a Creative Commons License (Attribution-NonCommercial 4.0 International), as described at http://creativecommons.org/licenses/by-nc/4.0/.
