Cited by 353RNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders ResearchAmyotrophic Lateral Sclerosis Research

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Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

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Bennett, C. Frank, Krainer, Adrian R., Cleveland, Don W.·2019·Annual Review of Neuroscience

DOI 10.1146/annurev-neuro-070918-050501PMC PMC7427431

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Abstract

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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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)
ORCID: Bennett, C. Frank
Krainer, Adrian R.
Cleveland, Don W.
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
DOI: 10.1146/annurev-neuro-070918-050501
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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