The DOI matches its Crossref record; 141 of the 141 references it cites were found in a registry or an index.
Antisense Oligonucleotide Therapies for Neurodegenerative Diseases
Open accessAbstract
Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases. Antisense drugs are currently in development for the treatment of amyotrophic lateral sclerosis, Huntington's disease, and Alzheimer's disease, and multiple research programs are underway for additional neurodegenerative diseases. One antisense drug, nusinersen, has been approved for the treatment of spinal muscular atrophy. Importantly, nusinersen improves disease symptoms when administered to symptomatic patients rather than just slowing the progression of the disease. In addition to the benefit to spinal muscular atrophy patients, there are discoveries from nusinersen that can be applied to other neurological diseases, including method of delivery, doses, tolerability of intrathecally delivered antisense drugs, and the biodistribution of intrathecal dosed antisense drugs. Based in part on the early success of nusinersen, antisense drugs hold great promise as a therapeutic platform for the treatment of neurological diseases.
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“optimized ASOs to other targets have previously been shown to yield dose dependent target RNA suppression to 5% of initial level13,14,48.”
From Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB (2021) · cites this paper as [48]
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Metadata
- Authors
- Bennett, C. F., Krainer, A. R., & Cleveland, D. W.
- Affiliations
- Ionis Pharmaceuticals (United States) (US)
- Cold Spring Harbor Laboratory (US)
- University of California San Diego (US)
- Venue
- Annual Review of Neuroscience
- Publisher
- Annual Reviews
- Published
- 2019-07-08
- Volume
- 42
- Issue
- 1
- Pages
- 385-406
- ISSN
- 0147-006X (ISSN-L)
- 1545-4126
- PMC
- PMC7427431
- OpenAlex
- W2961133941
- Cited by
- 353
- References
- 141
- Citations by year
- 2026: 47 · 2025: 45 · 2024: 62 · 2023: 44 · 2022: 64 · 2021: 47 · 2020: 38 · 2019: 5
- FWCI
- 3.40
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Annual Review of Neuroscience (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Cold Spring Harbor Laboratory Institutional Repository (Cold Spring Harbor Laboratory) (submittedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Funders
- National Cancer Institute (P01CA013106)
- National Institute of General Medical Sciences (R37GM042699, R01GM042699)
- 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 › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
- Health Sciences › Medicine › Clinical Neurology › Amyotrophic Lateral Sclerosis Research
- Keywords
- antisense oligonucleotides, neurodegenerative diseases, nusinersen, spinal muscular atrophy, amyotrophic lateral sclerosis, Huntington's disease, Alzheimer's disease, intrathecal drug delivery, drug biodistribution, drug tolerability, antisense drug development
- MeSH terms
- Animals, Brain, Humans, Muscular Atrophy, Spinal, Oligonucleotides, Tissue Distribution, Oligonucleotides, Antisense, Neurodegenerative Diseases
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