Alternative splicing in neurodegenerative disease and the promise of RNA therapies.
Journal
Nature reviews. Neuroscience
ISSN: 1471-0048
Titre abrégé: Nat Rev Neurosci
Pays: England
ID NLM: 100962781
Informations de publication
Date de publication:
08 2023
08 2023
Historique:
accepted:
02
06
2023
medline:
21
7
2023
pubmed:
20
6
2023
entrez:
19
6
2023
Statut:
ppublish
Résumé
Alternative splicing generates a myriad of RNA products and protein isoforms of different functions from a single gene. Dysregulated alternative splicing has emerged as a new mechanism broadly implicated in the pathogenesis of neurodegenerative diseases such as Alzheimer disease, amyotrophic lateral sclerosis, frontotemporal dementia, Parkinson disease and repeat expansion diseases. Understanding the mechanisms and functional outcomes of abnormal splicing in neurological disorders is vital in developing effective therapies to treat mis-splicing pathology. In this Review, we discuss emerging research and evidence of the roles of alternative splicing defects in major neurodegenerative diseases and summarize the latest advances in RNA-based therapeutic strategies to target these disorders.
Identifiants
pubmed: 37336982
doi: 10.1038/s41583-023-00717-6
pii: 10.1038/s41583-023-00717-6
doi:
Substances chimiques
RNA
63231-63-0
Protein Isoforms
0
Types de publication
Journal Article
Review
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
457-473Subventions
Organisme : NIMH NIH HHS
ID : R01 MH116220
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS104041
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS125276
Pays : United States
Informations de copyright
© 2023. Springer Nature Limited.
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