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Oligonucleotide therapies for disorders of the nervous system
Open accessAbstract
Oligonucleotide therapies are currently experiencing a resurgence driven by advances in backbone chemistry and discoveries of novel therapeutic pathways that can be uniquely and efficiently modulated by the oligonucleotide drugs. A quarter of a century has passed since oligonucleotides were first applied in living mammalian brain to modulate gene expression. Despite challenges in delivery to the brain, multiple oligonucleotide-based compounds are now being developed for treatment of human brain disorders by direct delivery inside the blood brain barrier (BBB). Notably, the first new central nervous system (CNS)-targeted oligonucleotide-based drug (nusinersen/Spinraza) was approved by US Food and Drug Administration (FDA) in late 2016 and several other compounds are in advanced clinical trials. Human testing of brain-targeted oligonucleotides has highlighted unusual pharmacokinetic and pharmacodynamic properties of these compounds, including complex active uptake mechanisms, low systemic exposure, extremely long half-lives, accumulation and gradual release from subcellular depots. Further work on oligonucleotide uptake, development of formulations for delivery across the BBB and relevant disease biology studies are required for further optimization of the oligonucleotide drug development process for brain applications.
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“it prevents the transfer of most of small-molecule drugs and macromolecules (e.g., peptides, gene drugs, and protein drugs) into the brain (1–6).”
From Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection (2020) · cites this paper as [5]
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“The treatment of central nervous system (CNS) diseases, such as neurodegenerative disorders, brain tumors, brain infections, and stroke, is severely constrained by the blood-brain barrier (BBB) because it prevents the transfer of most of small-molecule drugs and macromolecules (e.g., peptides, gene drugs, and protein drugs) into the brain (1–6).”
From Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection (2020) · cites this paper as [5]
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“Some ASOs were clinically approved for certain neuromuscular diseases and are currently in trials for other conditions.184,185”
From MiR-128-3p – a gray eminence of the human central nervous system (2024) · cites this paper as [184]
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“The U.S. Food and Drug Administration approved the first CNS-related oligonucleotide-based drug, Nusinersen, for treating spinal muscular atrophy caused by a survival motor neuron (SMN) protein deficiency.”
From MiR-128-3p – a gray eminence of the human central nervous system (2024) · cites this paper as [184]
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“Nusinersen is designed to modify the splicing of the SMN2 (Survival of motor neuron 2, centromeric) gene, increasing the production of functional SMN protein.184,186,187”
From MiR-128-3p – a gray eminence of the human central nervous system (2024) · cites this paper as [184]
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Metadata
- Authors
- Khorkova, O., & Wahlestedt, C.
- Affiliations
- OPKO Health (United States) (US)
- University of Miami (US)
- Corresponding author
- Wahlestedt, Claes
- Venue
- Nature Biotechnology
- Published
- 2017-02-27
- Volume
- 35
- Issue
- 3
- Pages
- 249-263
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- PMC
- PMC6043900
- OpenAlex
- W2593574576
- Cited by
- 179
- References
- 118
- Citations by year
- 2026: 10 · 2025: 17 · 2024: 21 · 2023: 18 · 2022: 23 · 2021: 17 · 2020: 23 · 2019: 20 · 2018: 20 · 2017: 10
- FWCI
- 8.16
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institute on Alcohol Abuse and Alcoholism (R01AA023781)
- National Institute on Aging (RC2AG036596)
- National Institute of Mental Health (R01MH083733)
- National Institute on Drug Abuse (R01DA025983, R21DA035592)
- National Institute of Neurological Disorders and Stroke (R01NS063974, P50NS071674, R01NS081208)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
- Keywords
- oligonucleotide therapeutics, central nervous system disorders, blood-brain barrier, nusinersen, antisense oligonucleotides, oligonucleotide delivery, pharmacokinetics, pharmacodynamics, oligonucleotide uptake, drug formulation, neurodegenerative diseases
- MeSH terms
- Animals, Brain Diseases, Humans, Oligonucleotides, Genetic Therapy, Treatment Outcome, Evidence-Based Medicine
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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