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

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.

SUBMITTER: Morelli KH 

PROVIDER: S-EPMC10019361 | biostudies-literature | 2023 Apr

REPOSITORIES: biostudies-literature