Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders.
RNA-targeting therapeutics
gene therapy
neurological disorders
programmable macromolecules
Journal
RNA (New York, N.Y.)
ISSN: 1469-9001
Titre abrégé: RNA
Pays: United States
ID NLM: 9509184
Informations de publication
Date de publication:
04 2023
04 2023
Historique:
pubmed:
25
1
2023
medline:
22
3
2023
entrez:
24
1
2023
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 36693761
pii: rna.079519.122
doi: 10.1261/rna.079519.122
pmc: PMC10019361
doi:
Substances chimiques
RNA
63231-63-0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
489-497Subventions
Organisme : NINDS NIH HHS
ID : F32 NS112654
Pays : United States
Organisme : NHGRI NIH HHS
ID : R01 HG004659
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS103172
Pays : United States
Informations de copyright
© 2023 Morelli et al.; Published by Cold Spring Harbor Laboratory Press for the RNA Society.
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