The DOI matches its Crossref record; 161 of the 161 references it cites were found in a registry or an index.
Antisense Drugs Make Sense for Neurological Diseases
Open accessAbstract
The genetic basis for most inherited neurodegenerative diseases has been identified, yet there are limited disease-modifying therapies for these patients. A new class of drugs—antisense oligonucleotides (ASOs)—show promise as a therapeutic platform for treating neurological diseases. ASOs are designed to bind to the RNAs either by promoting degradation of the targeted RNA or by elevating expression by RNA splicing. Intrathecal injection into the cerebral spinal fluid results in broad distribution of antisense drugs and long-term effects. Approval of nusinersen in 2016 demonstrated that effective treatments for neurodegenerative diseases can be identified and that treatments not only slow disease progression but also improve some symptoms. Antisense drugs are currently in development for amyotrophic lateral sclerosis, Huntington's disease, Alzheimer's disease, Parkinson's disease, and Angelman syndrome, and several drugs are in late-stage research for additional neurological diseases. This review highlights the advances in antisense technology as potential treatments for neurological diseases.
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“The FDA has approved eight ASO drugs since 2016, and the approval of the ASO-based drug nusinersen provides a strong basis for using this technology to develop novel and effective therapies for neurodegenerative diseases”
From ApoE4 reduction: An emerging and promising therapeutic strategy for Alzheimer's disease (2022) · cites this paper as Bennett et al 2021
Experimental — accuracy not yet measuredThe cited source says what the claim attributes to it.
In the citing paper
“Currently, ASO targeting MAPT (Tau) for AD treatment is under Phase 1/2 clinical trial”
From ApoE4 reduction: An emerging and promising therapeutic strategy for Alzheimer's disease (2022) · cites this paper as Bennett et al 2021
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
Staging of brain pathology related to sporadic Parkinson’s disease
Braak, H., Tredici, K. D., Rüb, U., de Vos, R. A. I., Jansen Steur, E. N. H., & Braak, E. (2003) · Neurobiology of Aging · doi:10.1016/s0197-4580(02)00065-9 · Cited by 10,777
Amyloid oligomers exacerbate tau pathology in a mouse model of tauopathy
Selenica, M.-L. B., Brownlow, M., Jimenez, J. P., Lee, D. C., Pena, G., Dickey, C. A., Gordon, M. N., & Morgan, D. (2013) · Neurodegenerative Diseases · doi:10.1159/000337230 · Cited by 10,359
Polazzi, E., Mengoni, I., Caprini, M., Peña‐Altamira, E., Kurtys, E., & Monti, B. (2012) · Neurosignals · doi:10.1159/000337115 · Cited by 9,023
MACDONALD, M. (1993) · Cell · doi:10.1016/0092-8674(93)90585-e · Cited by 8,366
Rosen, D. R., Siddique, T., Patterson, D., Figlewicz, D. A., Sapp, P., Hentati, A., Donaldson, D., Goto, J., O'Regan, J. P., Deng, H.-X., et al. (1993) · Nature · doi:10.1038/362059a0 · Cited by 7,062
Ubiquitinated TDP-43 in Frontotemporal Lobar Degeneration and Amyotrophic Lateral Sclerosis
Neumann, M., Sampathu, D. M., Kwong, L. K., Truax, A. C., Micsenyi, M. C., Chou, T. T., Bruce, J., Schuck, T., Grossman, M., Clark, C. M., et al. (2006) · Science · doi:10.1126/science.1134108 · Cited by 6,576
Iliff, J. J., Wang, M., Liao, Y., Plogg, B. A., Peng, W., Gundersen, G. A., Benveniste, H., Vates, G. E., Deane, R., Goldman, S. A., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3003748 · Cited by 5,824 · cited 1×
“The exchange of CSF and interstitial fluid is facilitated by convective influx of CSF along the periarterial space into the brain parenchyma through the glymphatic system, with these convective forces driving movement of macromolecules through the parenchyma (52).”
BackgroundPoewe, W., Seppi, K., Tanner, C. M., Halliday, G. M., Brundin, P., Volkmann, J., Schrag, A.-E., & Lang, A. E. (2017) · Nature Reviews Disease Primers · doi:10.1038/nrdp.2017.13 · Cited by 5,415
DeJesus-Hernandez, M., Mackenzie, I. R., Boeve, B. F., Boxer, A. L., Baker, M., Rutherford, N. J., Nicholson, A. M., Finch, N. A., Flynn, H., Adamson, J., et al. (2011) · Neuron · doi:10.1016/j.neuron.2011.09.011 · Cited by 4,936 · cited 9×
“RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention.”
BackgroundInfluentialA Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD
Renton, A. E., Majounie, E., Waite, A., Simón-Sánchez, J., Rollinson, S., Gibbs, J. R., Schymick, J. C., Laaksovirta, H., van Swieten, J. C., Myllykangas, L., et al. (2011) · Neuron · doi:10.1016/j.neuron.2011.09.010 · Cited by 4,409
Research works printed but not linked: 1
- Zhu Q, 2020, Neurosci, 23, 615
Cited by
Sorted by citations
Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies
Baughn, M. W., Melamed, Z., López‐Erauskin, J., Beccari, M. S., Ling, K., Zuberi, A. R., Presa, M. F., Gonzalo-Gil, E., Maimon, R., Vazquez‐Sanchez, S., et al. (2023) · Science · doi:10.1126/science.abq5622 · Cited by 252
Self-citationReexamining assumptions about miRNA-guided gene silencing
Kilikevicius, A., Meister, G., & Corey, D. R. (2022) · Nucleic Acids Research · doi:10.1093/nar/gkab1256 · Cited by 194
“Spinraza, an ASO that affects splicing of the survival motor neuron 2 (SMN2) RNA has proven to be a remarkably effective treatment for spinal muscular atrophy (84).”
S. aureus drives itch and scratch-induced skin damage through a V8 protease-PAR1 axis
Deng, L., Costa, F. G., Blake, K. J., Choi, S., Chandrabalan, A., Yousuf, M. S., Shiers, S. I., Dubreuil, D., Vega‐Mendoza, D., Rolland, C., et al. (2023) · Cell · doi:10.1016/j.cell.2023.10.019 · Cited by 165
Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic
Rook, M. E., & Southwell, A. L. (2022) · BioDrugs · doi:10.1007/s40259-022-00519-9 · Cited by 123
Therapeutics in the Pipeline Targeting α-Synuclein for Parkinson's Disease
Jasutkar, H. G., Oh, S. E., & Mouradian, M. M. (2022) · Pharmacological Reviews · doi:10.1124/pharmrev.120.000133 · Cited by 104
Metadata
- Authors
- Bennett, C. F., Kordasiewicz, H. B., & Cleveland, D. W.
- Affiliations
- Ionis Pharmaceuticals (United States) (US)
- Ludwig Cancer Research (BE)
- Venue
- Annual Review of Pharmacology and Toxicology
- Publisher
- Annual Reviews
- Published
- 2021-01-06
- Volume
- 61
- Issue
- 1
- Pages
- 831-852
- ISSN
- 0362-1642 (ISSN-L)
- 1545-4304
- PMC
- PMC8682074
- OpenAlex
- W3091999396
- Cited by
- 108
- References
- 161
- Citations by year
- 2026: 15 · 2025: 11 · 2024: 29 · 2023: 24 · 2022: 19 · 2021: 10
- FWCI
- 1.26
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- The Annual Review of Pharmacology and Toxicology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- 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 › Clinical Neurology › Amyotrophic Lateral Sclerosis Research
- Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
- Keywords
- antisense oligonucleotides, neurodegenerative diseases, RNA degradation, intrathecal drug delivery, nusinersen, amyotrophic lateral sclerosis, Huntington's disease, Alzheimer's disease, Parkinson's disease, Angelman syndrome, disease-modifying therapy
- MeSH terms
- Pharmaceutical Preparations, Humans, Parkinson Disease, RNA, Oligonucleotides, Antisense, Neurodegenerative Diseases
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 ›
Are you an author, or do you see a problem with this page? Request a review
