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

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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… Click to show full 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; programmable macromolecule; neurological disorders; human neurological; treat human

Journal Title: RNA
Year Published: 2023

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