Evidence map›Paper›PMID 37664882›Full record

ArticleDisease models & mechanisms2023

Blood levels of neurofilament light are associated with disease progression in a mouse model of spinocerebellar ataxia type 3.

David Mengel, Isabel G Wellik, Kristen H Schuster, Sabrina I Jarrah, Madeleine Wacker, Naila S Ashraf, Gülin Öz, Matthis Synofzik, Maria do Carmo Costa, Hayley S McLoughlin

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Disease models & mechanisms, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.6field-weighted citation impact, top 36% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed, 4 citations in OpenAlex.

  1. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors at 3 institutions in 2 countries.

David MengelResearch Division Translational Genomics of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research and Center of Neurology, University of Tübingen, Tübingen 72076,Germany.
Isabel G WellikDepartment of Neurology, University of Michigan, Ann Arbor, MI 48109-2200, USA.ORCID 0000-0001-5780-1165
Kristen H SchusterDepartment of Neurology, University of Michigan, Ann Arbor, MI 48109-2200, USA.ORCID 0000-0002-8896-3623
Sabrina I JarrahDepartment of Neurology, University of Michigan, Ann Arbor, MI 48109-2200, USA.
Madeleine WackerResearch Division Translational Genomics of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research and Center of Neurology, University of Tübingen, Tübingen 72076,Germany.
Naila S AshrafDepartment of Neurology, University of Michigan, Ann Arbor, MI 48109-2200, USA.
Gülin ÖzCenter for Magnetic Resonance Research, Department of Radiology, Medical School, University of Minnesota, Minneapolis, MN 55455, USA.ORCID 0000-0002-5769-183X
Matthis SynofzikResearch Division Translational Genomics of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research and Center of Neurology, University of Tübingen, Tübingen 72076,Germany.ORCID 0000-0002-2280-7273
Maria do Carmo CostaDepartment of Neurology, University of Michigan, Ann Arbor, MI 48109-2200, USA.ORCID 0000-0001-8466-4864
Hayley S McLoughlinDepartment of Neurology, University of Michigan, Ann Arbor, MI 48109-2200, USA.ORCID 0000-0003-4279-2758
University of Michigan · USGerman Center for Neurodegenerative Diseases · DEUniversity of Minnesota · US

Funding

Preclinical 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
$2.1M
Defining Biomarkers for Spinocerebellar Ataxia Type 3R21NS111154 · NINDS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI MCLOUGHLIN, HAYLEY SARAH, OZ, GULIN · 2019 to 2019
$435k
NINDS NIH HHS R21 NS111154NINDS NIH HHS U01 NS106670
6 · The paper itself

Abstract

Increased neurofilament light (NfL; NEFL) protein in biofluids is reflective of neurodegeneration and has gained interest as a biomarker across neurodegenerative diseases. In spinocerebellar ataxia type 3 (SCA3), the most common dominantly inherited ataxia, patients exhibit progressive NfL increases in peripheral blood when becoming symptomatic, and NfL remains stably elevated throughout further disease course. However, progressive NfL changes are not yet validated in relevant preclinical SCA3 animal models, hindering its application as a biomarker during therapeutic development. We used ultra-sensitive single-molecule array (Simoa) to measure blood NfL over disease progression in YACQ84 mice, a model of SCA3, assessing relationships with measures of disease severity including age, CAG repeat size and magnetic resonance spectroscopy. YACQ84 mice exhibited plasma NfL increases that were concomitant with ataxia-related motor deficits as well as increased serum NfL, which correlated with previously established neurometabolite abnormalities, two relevant measures of disease in patients with SCA3. Our findings establish the progression of NfL increases in the preclinical YACQ84 mouse, further supporting the utility of blood NfL as a peripheral neurodegeneration biomarker and informing on coinciding timelines of different measures of SCA3 pathogenesis.

Indexed as

Machado-Joseph DiseaseAnimalsAtaxiaDisease Models, AnimalDisease ProgressionIntermediate FilamentsMiceAtaxiaATXN3Blood biomarkerNeurochemicalNeurodegenerationPolyglutamine

Identifiers

PMID37664882
PMCPMC10499033
OpenAlexW4386407997

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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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.