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.
Funder
National Institutes of Health
NIH
a University of California President's Postdoctoral Fellowship
Biomedical Research Fellowship from the Hartwell Foundation
Allen Distinguished Investigator Award
a Paul G. Allen Frontiers Group advised grant of the Paul G. Allen Foundation
Publisher
Cold Spring Harbor Laboratory