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Updated: Aug 12, 2025

A Simple Alternative to Stereotactic Injection for Brain Specific Knockdown of miRNA
Published on: December 26, 2015
Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders
Kathryn H Morelli1,2,3, Aaron A Smargon1,2,3, Gene W Yeo4,2,3
1Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, California 92093, USA.
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.
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