ArticleScience translational medicine2020
Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3.
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In one paragraph
Article in Science translational medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed, 1 pooled it
3.4field-weighted citation impact, top 6% of its field
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
23 citing papers in PubMed, 1 synthesis or guideline pooled it, 50 citations in OpenAlex.
- Specific Biomarkers in Spinocerebellar Ataxia Type 3: A Systematic Review of Their Potential Uses in Disease Staging and Treatment Assessment.International journal of molecular sciences · 2024Pooled it
- Hereditary Ataxias: From Pathogenesis and Clinical Features to Neuroimaging, Fluid, and Digital Biomarkers-A Scoping Review.International journal of molecular sciences · 2026Review
- The role of neurofilament light chain and other fluid biomarkers in cerebellar ataxias.Brain communications · 2026Review
- Role of Repeat Tract Structure and the rs7158733 SNP in Spinocerebellar Ataxia 3.International journal of molecular sciences · 2025Article
- Progression of biological markers in spinocerebellar ataxia type 3: longitudinal analysis of prospective data from the ESMI cohort.The Lancet regional health. Europe · 2025Article
- The Natural History Study and Biomarker Collection of the Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA).Cerebellum (London, England) · 2025Review
- Blood DDIT4 and TRIM13 Transcript Levels Mark the Early Stages of Machado-Joseph Disease.Annals of neurology · 2025Article
- Biomarkers in Spinocerebellar Ataxias.Cerebellum (London, England) · 2025Review
- Evaluating Glial Fibrillary Acidic Protein and Neurofilament Light as Potential Biomarkers for Spinocerebellar Ataxia 7.International journal of molecular sciences · 2025Article
- Regional distribution of polymorphisms associated to the disease-causing gene of spinocerebellar ataxia type 3.Journal of neurology · 2024Article
- Sequence composition changes in short tandem repeats: heterogeneity, detection, mechanisms and clinical implications.Nature reviews. Genetics · 2024Review
- Clinical and genetic analyses of a Swedish patient series diagnosed with ataxia.Journal of neurology · 2024Article
- Disease progression of spinocerebellar ataxia types 1, 2, 3 and 6 before and after ataxia onset.Annals of clinical and translational neurology · 2023Article
- Blood levels of neurofilament light are associated with disease progression in a mouse model of spinocerebellar ataxia type 3.Disease models & mechanisms · 2023Article
- Blood neurofilament light chain levels are associated with disease progression in a transgenic SCA3 mouse model.bioRxiv : the preprint server for biology · 2023Article
- Tau and neurofilament light-chain as fluid biomarkers in spinocerebellar ataxia type 3.European journal of neurology · 2022Article
- Cross-species genetic screens identify transglutaminase 5 as a regulator of polyglutamine-expanded ataxin-1.The Journal of clinical investigation · 2022Article
- Plasma PolyQ-ATXN3 Levels Associate With Cerebellar Degeneration and Behavioral Abnormalities in a New AAV-Based SCA3 Mouse Model.Frontiers in cell and developmental biology · 2022Article
- Identification of the Prodromal Symptoms and Pre-Ataxic Stage in Cerebellar Disorders: The Next Challenge.International journal of environmental research and public health · 2021Article
- Ataxin-2 gene: a powerful modulator of neurological disorders.Current opinion in neurology · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
52 authors at 15 institutions in 6 countries.
Mercedes PrudencioDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0002-4894-4858
Hector Garcia-MorenoAtaxia Centre, Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London WC1N 3BG, UK.ORCID 0000-0002-0697-3271
Karen R Jansen-WestDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0002-1234-7103
Rana Hanna Al-ShaikhDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0003-0447-4089
Tania F GendronDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0002-7335-2627
Michael G HeckmanDivision of Biomedical Statistics and Informatics, Mayo Clinic, Jacksonville, FL 32224, USA.
Matthew R SpiegelDivision of Biomedical Statistics and Informatics, Mayo Clinic, Jacksonville, FL 32224, USA.
Yari CarlomagnoDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0001-5530-7737
Lillian M DaughrityDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0003-4644-7265
Yuping SongDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
Judith A DunmoreDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0003-0190-0089
Natalie ByronDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
Björn OskarssonDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0002-1725-9866
Katharine A NicholsonSean M. Healey and AMG Center for ALS, Massachusetts General Hospital (MGH), Boston, MA 02114, USA.
Nathan P StaffDepartment of Neurology, Mayo Clinic, Rochester, MN 55905, USA.ORCID 0000-0001-6760-3859
Sorina GorcencoLund University, Skåne University Hospital, Department of Clinical Sciences Lund, Neurology, Lund 22185, Sweden.ORCID 0000-0002-7776-6691
Andreas PuschmannLund University, Skåne University Hospital, Department of Clinical Sciences Lund, Neurology, Lund 22185, Sweden.ORCID 0000-0002-3201-8198
João LemosCoimbra University Hospital Centre, Coimbra University, Coimbra 3000-075, Portugal.ORCID 0000-0002-9128-7064
Cristina JanuárioCoimbra University Hospital Centre, Coimbra University, Coimbra 3000-075, Portugal.ORCID 0000-0001-5402-3978
Mark S LeDouxUniversity of Memphis and Veracity Neuroscience LLC, Memphis, TN 38152, USA.
Joseph H FriedmanDepartment of Neurology, Warren Alpert Medical School of Brown University, Providence, RI 02906, USA.
James PolkeAtaxia Centre, Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London WC1N 3BG, UK.
Robin LabrumAtaxia Centre, Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London WC1N 3BG, UK.
Vikram ShakkottaiDepartment of Neurology, University of Michigan, Ann Arbor, MI 48109, USA.ORCID 0000-0003-2742-6469
Hayley S McLoughlinDepartment of Neurology, University of Michigan, Ann Arbor, MI 48109, USA.ORCID 0000-0003-4279-2758
Henry L PaulsonDepartment of Neurology, University of Michigan, Ann Arbor, MI 48109, USA.ORCID 0000-0002-0382-7535
Takuya KonnoDepartment of Neurology, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.ORCID 0000-0002-2685-0025
Osamu OnoderaDepartment of Neurology, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.ORCID 0000-0003-3354-5472
Takeshi IkeuchiDepartment of Molecular Genetics, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.ORCID 0000-0001-8828-8085
Mari TadaDepartment of Pathology, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.ORCID 0000-0003-1485-0703
Akiyoshi KakitaDepartment of Pathology, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.
John D FryerNeuroscience Graduate Program, Mayo Clinic Graduate School of Biomedical Sciences, Jacksonville, FL 32224, USA.
Christin KarremoLund University, Skåne University Hospital, Department of Clinical Sciences Lund, Neurology, Lund 22185, Sweden.
Inês GomesCoimbra University Hospital Centre, Coimbra University, Coimbra 3000-075, Portugal.ORCID 0000-0003-4166-640X
John N CavinessDepartment of Neurology, Mayo Clinic, Scottsdale, AZ 85259, USA.
Mark R PittelkowDepartment of Dermatology, Mayo Clinic, Scottsdale, AZ 85259, USA.ORCID 0000-0001-7913-0294
Jan AaslyNorwegian University of Science and Technology, 7006 Trondheim, Norway.ORCID 0000-0002-0558-7686
Ronald F PfeifferDepartment of Neurology, Oregon Health & Science University, Portland, OR 97239, USA.
Venka VeerappanDepartment of Neurology, Oregon Health & Science University, Portland, OR 97239, USA.
Eric R EggenbergerDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
William D FreemanDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0003-2326-0633
Josephine F HuangDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0002-2407-1343
Ryan J UittiDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
Klaas J WierengaDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
Iris V Marin CollazoDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0002-0858-3787
Philip W TiptonDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0003-4084-2248
Jay A van GerpenUniversity of Alabama at Birmingham, Birmingham, AL 35233, USA.
Marka van BlitterswijkDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0002-3054-7053
Guojun BuDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.ORCID 0000-0003-3491-1016
Zbigniew K WszolekDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA. wszolek.zbigniew@mayo.edu p.giunti@ucl.ac.uk petrucelli.leonard@mayo.edu.ORCID 0000-0001-5487-1053
Paola GiuntiAtaxia Centre, Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London WC1N 3BG, UK. wszolek.zbigniew@mayo.edu p.giunti@ucl.ac.uk petrucelli.leonard@mayo.edu.ORCID 0000-0003-3508-4788
Leonard PetrucelliDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA. wszolek.zbigniew@mayo.edu p.giunti@ucl.ac.uk petrucelli.leonard@mayo.edu.ORCID 0000-0003-2959-129X
Mayo Clinic in Florida · USNiigata University · JPUniversity College London Hospitals NHS Foundation Trust · GBLund University · SEUniversity of Coimbra · PTUniversity of Michigan · USMayo Clinic in Arizona · USOregon Health & Science University · USBrown University · USMassachusetts General Hospital · USMayo Clinic · USNorwegian University of Science and Technology · NOUniversity of Alabama at Birmingham · USUniversity of Memphis · USWinnMed · US
Funding
Project 3: Neuropathology of the multi-proteinopathy of c9FTD/ALS.P01NS084974 · NINDS · MAYO CLINIC JACKSONVILLE · PI PETRUCELLI, LEONARD · 2014 to 2024
$17.6MViral Vectors CoreP01NS074969 · NINDS · WASHINGTON UNIVERSITY · PI HOLTZMAN, DAVID M. · 2012 to 2021
$13.0MExpanding insights into FTD disease mechanismsR35NS097273 · NINDS · MAYO CLINIC JACKSONVILLE · PI PETRUCELLI, LEONARD · 2017 to 2024
$10.6MValidation of r(G4C2)exp-binding small molecules in vivo and c9FTD/ALS biomarker developmentP01NS099114 · NINDS · UNIVERSITY OF FLORIDA · PI ROTHSTEIN, JEFFREY D · 2017 to 2021
$6.5MPreclinical Development of Antisense Oligonucleotide Therapy for Spinocerebellar Ataxia Type 3U01NS106670 · NINDS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI MCLOUGHLIN, HAYLEY SARAH, PAULSON, HENRY L · 2018 to 2021
$2.1MSilencing C9or72 with rAAV Mediated RNAiR01NS088689 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI BROWN, ROBERT H., MUELLER, CHRISTIAN · 2014 to 2017
$1.6MRNA foci in C9FTD/ALSR21NS084528 · NINDS · MAYO CLINIC JACKSONVILLE · PI PETRUCELLI, LEONARD · 2013 to 2014
$430kMINORITY PREDOCTORAL FELLOWSHIP PROGRAMF31NS010667 · NINDS · HARVARD UNIVERSITY (MEDICAL SCHOOL) · PI GARCIA, JESUS A · 1997 to 2000
$36kMedical Research Council MR/N028767/1NINDS NIH HHS F31 NS010667NINDS NIH HHS P01 NS074969NINDS NIH HHS P01 NS084974NINDS NIH HHS P01 NS099114NINDS NIH HHS R01 NS088689NINDS NIH HHS R21 NS084528NINDS NIH HHS R35 NS097273NINDS NIH HHS U01 NS106670
Abstract
Spinocerebellar ataxia type 3 (SCA3), caused by a CAG repeat expansion in the ataxin-3 gene (
Indexed as
Machado-Joseph DiseaseAllelesAtaxin-3HumansNeuronsRepressor ProteinsAtaxin-3Repressor Proteins
Identifiers
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.