The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration
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Abstract
Antisense oligonucleotides (ASOs) have emerged as a new class of drugs to treat a wide range of diseases, including neurological indications. Spinraza, an ASO that modulates splicing of SMN2 RNA, has shown profound disease modifying effects in Spinal Muscular Atrophy (SMA) patients, energizing efforts to develop ASOs for other neurological diseases. While SMA specifically affects spinal motor neurons, other neurological diseases affect different central nervous system (CNS) regions, neuronal and non-neuronal cells. Therefore, it is important to characterize ASO distribution and activity in all major CNS structures and cell types to have a better understanding of which neurological diseases are amenable to ASO therapy. Here we present for the first time the atlas of ASO distribution and activity in the CNS of mice, rats, and non-human primates (NHP), species commonly used in preclinical therapeutic development. Following central administration of an ASO to rodents, we observe widespread distribution and target RNA reduction throughout the CNS in neurons, oligodendrocytes, astrocytes and microglia. This is also the case in NHP, despite a larger CNS volume and more complex neuroarchitecture. Our results demonstrate that ASO drugs are well suited for treating a wide range of neurological diseases for which no effective treatments are available.
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ASOs are not well suited for deep brain structure targets.
“numerous studies suggest that ASOs are not well suited for deep brain structure targets [16,47,52].”
From Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid–siRNA conjugate administered into cerebrospinal fluid (2025) · cites this paper as [52]
The Discussion (page not provided) reports less ASO accumulation and RNA knockdown in some deep structures, including the striatum, caudate, putamen, and globus pallidus. It says higher doses are needed to achieve the desired suppression there, not that ASOs are generally unsuited to deep-brain targets; the Discussion also reports widespread activity across most CNS regions. (LLM, conf=0.88)
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- Authors
- Jafar-nejad, P., Powers, B., Soriano, A., Zhao, H., Norris, D. A., Matson, J., DeBrosse-Serra, B., Watson, J., Narayanan, P., Chun, S. J., Mazur, C., Kordasiewicz, H., Swayze, E. E., & Rigo, F.
- Affiliations
- Ionis Pharmaceuticals Inc. Carlsbad, CA 92010, USA
- Ionis Pharmaceuticals (United States) (US)
- Corresponding author
- Rigo, Frank
- ORCID
- Venue
- Nucleic Acids Research
- Publisher
- Oxford University Press (OUP)
- Published
- 2020-12-24
- Volume
- 49
- Issue
- 2
- Pages
- 657-673
- ISSN
- 0305-1048 (ISSN-L)
- 1362-4962
- PMC
- PMC7826274
- OpenAlex
- W3114205754
- Cited by
- 128
- References
- 56
- Citations by year
- 2026: 32 · 2025: 23 · 2024: 24 · 2023: 21 · 2022: 22 · 2021: 5 · 2020: 1
- FWCI
- 3.97
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Nucleic Acids Research (publishedVersion, cc-by-nc)
- bioRxiv (Cold Spring Harbor Laboratory) (acceptedVersion)
- Europe PMC (PubMed Central) (publishedVersion, cc-by-nc)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Keywords
- antisense oligonucleotides, RNase H, central nervous system distribution, spinal muscular atrophy, SMN2 splicing, oligodendrocytes, astrocytes, microglia, neurons, non-human primates, rodents, centralized management, neurological disease therapy
- MeSH terms
- Animals, Central Nervous System, Female, Injections, Intraventricular, Injections, Spinal, Macaca fascicularis, Male, Neuroglia, Neurons, Organ Specificity, Primates, Tissue Distribution, Oligonucleotides, Antisense, Ribonuclease H, Rats, Sprague-Dawley, In Situ Hybridization, Mice, Rats, RNA, Long Noncoding
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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