Antisense oligonucleotides for neurodegeneration
Open accessAbstract
Promising clinical results for Huntington's disease give hope for other diseases
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
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?
ASO injection is a current standard of care for spinal muscular atrophy and is in five ongoing Phase I trials in ALS, Parkinson's, and Alzheimer's diseases and in Phase III trials for inherited ALS and Huntington's disease.
“ASO injection is a current standard of care for the fatal childhood disease spinal muscular atrophy15 and is in five ongoing Phase I trials in ALS, Parkinson’s, and Alzheimer’s diseases and in Phase III trials for inherited ALS from a mutation in superoxide dismutase16,17 and for Huntington’s disease18.”
From Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB (2021) · cites this paper as [18]
The Abstract reports an approved ASO therapy for spinal muscular atrophy and ongoing trials in ALS, Parkinson’s, and Alzheimer’s disease. The clinical-trials section identifies a phase 3 Huntington’s disease trial, but places the SOD1-mutant ALS trial in phase 1/2a, not phase 3; it also does not establish a total of five ongoing phase I trials. The copy provides no page numbers for these passages. (LLM, conf=0.88)
Experimental — accuracy not yet measuredThe FDA has approved eight ASO drugs since 2016, and the approval of nusinersen provides a strong basis for using ASO technology to develop novel therapies for neurodegenerative diseases.
“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 Leavitt & Tabrizi 2020
Split into 2 atoms — 1 confirmed, 1 partially supported, 0 disputed, 0 misattributed, 0 unverifiable. Expand the claim to see per-atom verdicts.
Experimental — accuracy not yet measured
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
No references could be resolved yet.
Research works printed but not linked: 13
- S.J. Tabrizi SJ, R. Ghosh R, B.R. Leavitt, Neuron 102, 899 (2019).
- C. Rinaldi, M.J.A. Wood, Nat. Rev. Neurol. 14, 9 (2018).
- G.P. Bates et al., Nat. Rev. Dis. Prim. 1, 15005 (2015).
- H.B. Kordasiewicz et al., Neuron 74, 1031 (2012).
- S.J. Tabrizi SJ, et al., N Eng. J. Med. 380, 2307 (2019).
- N. Iwamoto et al., Nat. Biotech. 35, 845 (2017).
- T.M. Miller et al., Lancet Neurology 12, 435 (2013).
- H.T. Zhao et al., Mol. Ther. Nucleic Acids 8, 508 (2017).
- S.L. DeVos et al., J. Neuroscience 33, 12887 (2013).
- G.J. Raymond et al., JCI Insight 4, 131175 (2019).
- P. Gonzalez-Alegre Hum. Mol. Genet. 28, R80 (2019).
- L.A. Becker et al., Nature 544, 367 (2017).
- J. Jiang et al., Neuron. 90, 535 (2016). ACKNOWLEDGMENTS (OPTIONAL) Both authors have undertaken paid consultancy work for Ionis pharmaceuticals, Takeda pharmaceuticals, and F Hoffman-La Roche.
Cited by
Sorted by citations
Jack, C. R., Arani, A., Borowski, B. J., Cash, D. M., Crawford, K., Das, S. R., DeCarli, C., Fletcher, E., Fox, N. C., Gunter, J. L., et al. (2024) · Alzheimer's & Dementia · doi:10.1002/alz.14166 · Cited by 82 · see citing works
Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB
Maimon, R., Chillon-Marinas, C., Snethlage, C. E., Singhal, S. M., McAlonis-Downes, M., Ling, K., Rigo, F., Bennett, C. F., Da Cruz, S., Hnasko, T. S., et al. (2021) · Nature Neuroscience · doi:10.1038/s41593-021-00864-y · Cited by 62 · see citing works
ApoE4 reduction: An emerging and promising therapeutic strategy for Alzheimer's disease
Li, Y., Macyczko, J. R., Liu, C.-C., & Bu, G. (2022) · Neurobiology of Aging · doi:10.1016/j.neurobiolaging.2022.03.011 · Cited by 61 · see citing works
- Authors
- Leavitt, B. R., & Tabrizi, S. J.
- Affiliations
- University of British Columbia Hospital (CA)
- UK Dementia Research Institute (GB)
- Venue
- Science
- Published
- 2020-03-27
- Volume
- 367
- Issue
- 6485
- Pages
- 1428-1429
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- OpenAlex
- W3013988004
- Cited by
- 96
- References
- 0
- Citations by year
- 2026: 4 · 2025: 16 · 2024: 16 · 2023: 10 · 2022: 14 · 2021: 23 · 2020: 13
- Type
- journal article
- Language
- en
- Open access
- green
- discovery.ucl.ac.uk
- Locations
- Science (publishedVersion)
- PubMed (publishedVersion)
- UCL Discovery (University College London) (submittedVersion)
- Topics
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- antisense oligonucleotides, neurodegeneration, Huntington's disease, gene therapy, clinical trials
- MeSH terms
- Huntingtin Protein, Clinical Trials as Topic, Gene Expression Regulation, Humans, Huntington Disease, RNA Precursors, RNA, Messenger, Oligonucleotides, Antisense, Neurodegenerative Diseases, RNA Interference
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
