Cited by 165RNA Interference and Gene DeliveryAlzheimer's disease research and treatmentsAmyotrophic Lateral Sclerosis Research

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Antisense Oligonucleotides: Treating Neurodegeneration at the Level of RNA

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DeVos, Sarah L., Miller, Timothy M.·2013·Neurotherapeutics

DOI 10.1007/s13311-013-0194-5PMC PMC3701770

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Abstract

Adequate therapies are lacking for Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, and other neurodegenerative diseases. The ability to use antisense oligonucleotides (ASOs) to target disease-associated genes by means of RNA may offer a potent approach for the treatment of these, and other, neurodegenerative disorders. In modifying the basic backbone chemistry, chemical groups, and target sequence, ASOs can act through numerous mechanisms to decrease or increase total protein levels, preferentially shift splicing patterns, and inhibit microRNAs, all at the level of the RNA molecule. Here, we discuss many of the more commonly used ASO chemistries, as well as the different mechanisms of action that can result from these specific chemical modifications. When applied to multiple neurodegenerative mouse models, ASOs that specifically target the detrimental transgenes have been shown to rescue disease associated phenotypes in vivo. These supporting mouse model data have moved the ASOs from the bench to the clinic, with two neuro-focused human clinical trials now underway and several more being proposed. Although still early in development, translating ASOs into human patients for neurodegeneration appears promising.

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Authors: DeVos, S. L., & Miller, T. M.
Affiliations: Washington University in St. Louis (US)
Corresponding author: Miller, Timothy M.
ORCID: DeVos, Sarah L.
Miller, Timothy M.
Venue: Neurotherapeutics
Publisher: Elsevier BV
Published: 2013-07
Volume: 10
Issue: 3
Pages: 486-497
ISSN: 1878-7479 (ISSN-L)
DOI: 10.1007/s13311-013-0194-5
PMC: PMC3701770
OpenAlex: W2093892225
Cited by: 164
References: 136
Citations by year: 2026: 8 · 2025: 11 · 2024: 11 · 2023: 9 · 2022: 11 · 2021: 16 · 2020: 14 · 2019: 17 · 2018: 13 · 2017: 14 · 2016: 17 · 2015: 12 · 2014: 8 · 2013: 3
FWCI: 1.26
Type: journal article
Language: en
Open access: bronze
doi.org
Licence: www.elsevier.com/tdm/userlicense/1.0/ (tdm)
www.elsevier.com/legal/tdmrep-license (tdm)
www.elsevier.com/open-access/userlicense/1.0/ (vor, 3699 d)
Locations: Neurotherapeutics (publishedVersion)
PubMed Central (publishedVersion)
PubMed (publishedVersion)
Funders: National Institutes of Health
National Institute on Aging (P50AG005681)
National Institute of Neurological Disorders and Stroke (NIH/NINDS K08NS074194, NIH/NINDS R01NS078398, K08NS074194, R01NS078398)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Alzheimer's disease research and treatments
Health Sciences › Medicine › Clinical Neurology › Amyotrophic Lateral Sclerosis Research
Keywords: antisense oligonucleotides, neurodegenerative diseases, RNA-targeted therapeutics, splicing modulation, microRNA inhibition, mouse models of human disease, Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis
MeSH terms: Animals, Humans, RNA, Neuroprotective Agents, Neurodegenerative Diseases, Oligodeoxyribonucleotides, Antisense

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Antisense Oligonucleotides: Treating Neurodegeneration at the Level of RNA

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