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Record W4406178968 · doi:10.7554/elife.102879

Identification of neurodevelopmental organization of the cell populations of juvenile Huntington’s disease using dorso-ventral HD organoids and HD mouse embryos

2025· preprint· en· W4406178968 on OpenAlexaff
Karolina Świtońska-Kurkowska, Jakub Kubiś, Joanna Delimata-Raczek, Bart Krist, Magda Surdyka, Żaneta Kalinowska-Pośka, Piotr Piasecki, Luiza Handschuh, Jan Podkowiński, Magdalena Rakoczy, Anna Samelak-Czajka, Michael R. Hayden, Nicholas S. Caron, Maciej Figiel

Bibliographic record

VenueeLife · 2025
Typepreprint
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicPluripotent Stem Cells Research
Canadian institutionsUniversity of British Columbia
Fundersnot available
KeywordsOrganoidHuntington's diseaseBiologyJuvenileEmbryoIdentification (biology)DiseaseNeuroscienceEmbryonic stem cellGeneticsPathologyMedicineGene

Abstract

fetched live from OpenAlex

Abstract Huntington’s disease (HD) is a polyglutamine neurodegenerative disease involving pathogenesis within the striatum and cerebral cortex and a neurodevelopmental component, particularly in juvenile HD form (JOHD). We established a fused HD dorsal-ventral system, imitating the cortex and striatum interaction in a single organoid to discover neurodevelopmental impairments at the level of cell populations. We found a range of early pathogenic phenotypes indicating that brain development in HD is affected by impaired neurogenesis. The phenotypes occurred already in early-stage 60-day organoids and the brain of humanized mouse embryos, at time of the beginning of the neurogenesis and choroid plexus development. We demonstrated that HD organoids and HD mouse embryonic brains had gene expression profiles of impaired maturation of neurons and increased expression of genes responsible for proliferation compared to genes responsible for differentiation in control organoids. By using scRNA-seq, the choroid plexus population was highly abundant in HD organoids and embryonic brains. Cortical and choroid plexus cell populations in HD organoids and brains co-expressed genes responsible for HTT function (mitotic spindle and cilia). The impaired maturation and the increased occurrence of the choroid plexus populations were mitigated in our compensatory model, mosaic dorsal/ventral (D/V) or V/D HD/control organoids. Finally, we found that TTR protein, a choroid plexus marker, is elevated in the adult HD mouse serum, indicating that TTR may be a promising marker for detecting HD. In summary, the fused dorso-ventral HD organoids identify a spectrum of neurodevelopmental features, including increased proliferation and delayed cell maturation. We demonstrate that the choroid plexus population is characteristic of aberrant HD neurodevelopment, and contains TTR marker, which can be translated as a blood marker in HD.

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.

How this classification was reachedexpand

Full frame machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Bench or experimental · Consensus signal: Bench or experimental
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.001
Threshold uncertainty score0.003

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0010.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0010.000

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 head0.015
GPT teacher head0.266
Teacher spread0.251 · 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

Classification

machine, unvalidated

Machine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designBench or experimental
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations0
Published2025
Admission routes1
Has abstractyes

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