Antisense Drugs Make Sense for Neurological Diseases.
Huntington's disease
amyotrophic lateral sclerosis
antisense oligonucleotide
neurodegenerative disease
siRNA
spinal muscular atrophy
Journal
Annual review of pharmacology and toxicology
ISSN: 1545-4304
Titre abrégé: Annu Rev Pharmacol Toxicol
Pays: United States
ID NLM: 7607088
Informations de publication
Date de publication:
06 01 2021
06 01 2021
Historique:
pubmed:
10
10
2020
medline:
10
9
2021
entrez:
9
10
2020
Statut:
ppublish
Résumé
The genetic basis for most inherited neurodegenerative diseases has been identified, yet there are limited disease-modifying therapies for these patients. A new class of drugs-antisense oligonucleotides (ASOs)-show promise as a therapeutic platform for treating neurological diseases. ASOs are designed to bind to the RNAs either by promoting degradation of the targeted RNA or by elevating expression by RNA splicing. Intrathecal injection into the cerebral spinal fluid results in broad distribution of antisense drugs and long-term effects. Approval of nusinersen in 2016 demonstrated that effective treatments for neurodegenerative diseases can be identified and that treatments not only slow disease progression but also improve some symptoms. Antisense drugs are currently in development for amyotrophic lateral sclerosis, Huntington's disease, Alzheimer's disease, Parkinson's disease, and Angelman syndrome, and several drugs are in late-stage research for additional neurological diseases. This review highlights the advances in antisense technology as potential treatments for neurological diseases.
Identifiants
pubmed: 33035446
doi: 10.1146/annurev-pharmtox-010919-023738
pmc: PMC8682074
mid: NIHMS1749063
doi:
Substances chimiques
Oligonucleotides, Antisense
0
Pharmaceutical Preparations
0
RNA
63231-63-0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
831-852Subventions
Organisme : NINDS NIH HHS
ID : R01 NS027036
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS112503
Pays : United States
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