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

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...

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Detalles Bibliográficos
Autores principales: Morelli, Kathryn H., Smargon, Aaron A., Yeo, Gene W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory Press 2023
Materias:
Perspectives
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10019361/
https://www.ncbi.nlm.nih.gov/pubmed/36693761
http://dx.doi.org/10.1261/rna.079519.122
Descripción
Sumario: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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