Oligonucleotide therapies for disorders of the nervous system

Khorkova, Olga; Wahlestedt, Claes

doi:10.1038/nbt.3784

Cited by 179RNA Interference and Gene DeliveryBarrier Structure and Function StudiesNeurogenetic and Muscular Disorders Research

Oligonucleotide therapies for disorders of the nervous system

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Khorkova, Olga, Wahlestedt, Claes·2017·Nature Biotechnology

DOI 10.1038/nbt.3784PMC PMC6043900

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Abstract

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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The blood-brain barrier prevents the transfer of most small-molecule drugs and macromolecules into the brain.
“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]
The Abstract (page unavailable) says oligonucleotides do not readily cross the blood-brain barrier and that fewer than 1% of systemically delivered oligonucleotides reach the brain. “Assisted delivery and CNS targeting of ODNs” (page unavailable) also mentions small molecules with poor BBB permeability and intranasal delivery of peptides or proteins. The paper does not establish the broader claim that the BBB prevents transfer of most small-molecule drugs and macromolecules. (LLM, conf=0.84)
Experimental — accuracy not yet measured
Treatment of central nervous system diseases is severely constrained by the blood-brain barrier.
“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]
The paper’s introductory passage says that the BBB is a substantial obstacle to treating CNS disorders with oligonucleotides: fewer than 1% of systemically delivered oligonucleotides reach the brain. Its assisted-delivery discussion also mentions small molecules with poor BBB permeability, peptides, and proteins. It does not establish the citing sentence’s broader assertion that the BBB prevents most small-molecule drugs and macromolecules from entering the brain across the listed CNS diseases. (LLM, conf=0.84)
Experimental — accuracy not yet measured

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[67]Wahlestedt C Yee F Yoo H Koob GF Heilig M Molecular Neurobiology Proceedings of the second NIMH Conference Zalcman S Scheller R Tsien R 1992
[118]DeVos SL Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy Sci Transl Med 9 374 2017 PMC5792300 28123067

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Authors: Khorkova, O., & Wahlestedt, C.
Affiliations: OPKO Health (United States) (US)
University of Miami (US)
Corresponding author: Wahlestedt, Claes
ORCID: Khorkova, Olga
Wahlestedt, Claes
Venue: Nature Biotechnology
Publisher: Springer Science and Business Media LLC
Published: 2017-02-27
Volume: 35
Issue: 3
Pages: 249-263
ISSN: 0733-222X (ISSN-L)
1087-0156
1546-1696
DOI: 10.1038/nbt.3784
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 › 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

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Oligonucleotide therapies for disorders of the nervous system

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