The DOI matches its Crossref record; 161 of the 161 references it cites were found in a registry or an index.
Antisense Drugs Make Sense for Neurological Diseases
Open accessAbstract
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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“The FDA has approved eight ASO drugs since 2016, and the approval of the ASO-based drug nusinersen provides a strong basis for using this technology to develop novel and effective therapies for neurodegenerative diseases”
From ApoE4 reduction: An emerging and promising therapeutic strategy for Alzheimer's disease (2022) · cites this paper as Bennett et al 2021
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“Currently, ASO targeting MAPT (Tau) for AD treatment is under Phase 1/2 clinical trial”
From ApoE4 reduction: An emerging and promising therapeutic strategy for Alzheimer's disease (2022) · cites this paper as Bennett et al 2021
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Emergencies in Amyotrophic Lateral Sclerosis
Uysal, S. P., Dudley, K. A., Sullivan, S. L., Brizzi, K. T., Burke, K. M., Hall, S., Pease, K., Goldstein, J. N., Berry, J. D., & Ho, D. T. (2026) · Muscle & Nerve · doi:10.1002/mus.70398
Esmaeili, H., Das, S., Rytel, K., Sidransky, E., & Chen, Y. (2026) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2026.110265
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Liu, Y., Jiang, M., Li, J., Zhang, L., Zuo, C., Zhang, H., & Tan, W. (2026) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.6c09554
Nagy, C., DEPOND, C., Doisy, M., Vacca, O., & Goyenvalle, A. (2026) · Research Square · doi:10.21203/rs.3.rs-11565868/v1
Metadata
- Authors
- Bennett, C. F., Kordasiewicz, H. B., & Cleveland, D. W.
- Affiliations
- Ionis Pharmaceuticals (United States) (US)
- Ludwig Cancer Research (BE)
- 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
- 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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