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Record W2957790560 · doi:10.1186/s40478-019-0724-4

C9orf72 intermediate expansions of 24–30 repeats are associated with ALS

2019· review· en· W2957790560 on OpenAlex
Alfredo Iacoangeli, Ahmad Al Khleifat, William Sproviero, Aleksey Shatunov, Sarah Opie-Martin, Karen Morrison, Pamela J. Shaw, Christopher E. Shaw, Isabella Fogh, Richard Dobson, Stephen Newhouse, Ammar Al‐Chalabi

Why this work is in the frame

A frame that forgets how it found something cannot be audited. These are the routes that admitted this work.

fundA Canadian funder is recorded on the work.
no affNo Canadian affiliation: this work is invisible to an affiliation-only frame.
No Canadian affiliation. An affiliation-only frame, the usual design, would never have seen this work. It is one of the works that make the case for inverting the frame.

Bibliographic record

VenueActa Neuropathologica Communications · 2019
Typereview
Languageen
FieldMedicine
TopicAmyotrophic Lateral Sclerosis Research
Canadian institutionsnot available
FundersEconomic and Social Research CouncilNational Institute of Biomedical Imaging and BioengineeringCanadian Institutes of Health ResearchHorizon 2020 Framework ProgrammeGenentechNational Institutes of HealthIXICOH. Lundbeck A/SKing's College LondonServierEU Joint Programme – Neurodegenerative Disease ResearchNational Institute on AgingNational Institute for Health and Care ResearchNorthern California Institute for Research and EducationEisaiMotor Neurone Disease AssociationSouth London and Maudsley NHS Foundation TrustPfizerBiogenBioClinicaF. Hoffmann-La RocheWellcome TrustUniversity of Southern CaliforniaEli Lilly and CompanyU.S. Department of DefenseMedical Research CouncilMeso Scale DiagnosticsAlzheimer's Disease Neuroimaging InitiativeNovartis Pharmaceuticals CorporationBristol-Myers SquibbNIHR Sheffield Biomedical Research CentreAlzheimer's AssociationFoundation for the National Institutes of Health
KeywordsC9orf72NeurologyMedicineBiologyNeuroscienceComputational biologyTrinucleotide repeat expansionGeneticsGene

Abstract

fetched live from OpenAlex

The expansion of a hexanucleotide repeat GGGGCC in C9orf72 is the most common known cause of ALS accounting for ~ 40% familial cases and ~ 7% sporadic cases in the European population. In most people, the repeat length is 2, but in people with ALS, hundreds to thousands of repeats may be observed. A small proportion of people have an intermediate expansion, of the order of 20 to 30 repeats in size, and it remains unknown whether intermediate expansions confer risk of ALS in the same way that massive expansions do. We investigated the association of this intermediate repeat with ALS by performing a meta-analysis of four previously published studies and a new British/Alzheimer’s Disease Neuroimaging Initiative dataset of 1295 cases and 613 controls. The final dataset comprised 5071 cases and 3747 controls. Our meta-analysis showed association between ALS and intermediate C9orf72 repeats of 24 to 30 repeats in size (random-effects model OR = 4.2, 95% CI = 1.23–14.35, p-value = 0.02). Furthermore, we showed a different frequency of the repeat between the northern and southern European populations (Fisher’s exact test p-value = 5 × 10− 3). Our findings provide evidence for the association between intermediate repeats and ALS (p-value = 2 × 10− 4) with direct relevance for research and clinical practice by showing that an expansion of 24 or more repeats should be considered pathogenic.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.001
metaresearch head score (Gemma)0.003
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Other design · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.931
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.003
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0030.001
Bibliometrics0.0000.001
Science and technology studies0.0000.001
Scholarly communication0.0000.000
Open science0.0020.002
Research integrity0.0010.002
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.255
GPT teacher head0.403
Teacher spread0.148 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it