Cited by 110RNA Interference and Gene DeliveryAmyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders Research

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Antisense Drugs Make Sense for Neurological Diseases

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Bennett, C. Frank, Kordasiewicz, Holly B., Cleveland, Don W.·2021·Annual Review of Pharmacology and Toxicology

DOI 10.1146/annurev-pharmtox-010919-023738PMC PMC8682074

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Abstract

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.

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Additional file 2 of Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy
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Authors: Bennett, C. F., Kordasiewicz, H. B., & Cleveland, D. W.
Affiliations: Ionis Pharmaceuticals (United States) (US)
Ludwig Cancer Research (BE)
ORCID: Bennett, C. Frank
Kordasiewicz, Holly B.
Cleveland, Don W.
Venue: Annual Review of Pharmacology and Toxicology
Publisher: Annual Reviews
Published: 2021-01-06
Volume: 61
Issue: 1
Pages: 831-852
ISSN: 0362-1642 (ISSN-L)
1545-4304
DOI: 10.1146/annurev-pharmtox-010919-023738
PMC: PMC8682074
OpenAlex: W3091999396
Cited by: 108
References: 161
Citations by year: 2026: 15 · 2025: 11 · 2024: 29 · 2023: 24 · 2022: 19 · 2021: 10
FWCI: 1.26
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Locations: The Annual Review of Pharmacology and Toxicology (publishedVersion)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
Funders: National Institute of Neurological Disorders and Stroke (R01NS112503, R01NS027036)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Health Sciences › Medicine › Clinical Neurology › Amyotrophic Lateral Sclerosis Research
Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
Keywords: antisense oligonucleotides, neurodegenerative diseases, RNA degradation, intrathecal drug delivery, nusinersen, amyotrophic lateral sclerosis, Huntington's disease, Alzheimer's disease, Parkinson's disease, Angelman syndrome, disease-modifying therapy
MeSH terms: Pharmaceutical Preparations, Humans, Parkinson Disease, RNA, Oligonucleotides, Antisense, Neurodegenerative Diseases

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Antisense Drugs Make Sense for Neurological Diseases

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