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Record W4401078278 · doi:10.1016/j.neuron.2024.06.002

Rapid iPSC inclusionopathy models shed light on formation, consequence, and molecular subtype of α-synuclein inclusions

2024· article· en· W4401078278 on OpenAlexaff
Isabel Lam, Alain Ndayisaba, Amanda J. Lewis, YuHong Fu, Giselle T. Sagredo, Anastasia Kuzkina, Ludovica Zaccagnini, Meral Celikag, Jackson Sandoe, Ricardo L. Sanz, Aazam Vahdatshoar, Timothy D. Martin, Nader Morshed, Toru Ichihashi, Arati Tripathi, Nagendran Ramalingam, Charlotte Oettgen-Suazo, Theresa Bartels, Manel Boussouf, Max Schäbinger, Erinc Hallacli, Xin Jiang, Amrita Verma, Challana Tea, Zichen Wang, Hiroyuki Hakozaki, Xiao Yu, Kelly Hyles, Chansaem Park, Xinyuan Wang, Thorold W. Theunissen, Haoyi Wang, Rudolf Jaenisch, Susan Lindquist, Beth Stevens, Nadia Stefanova, Gregor K. Wenning, Wilma D. J. van de Berg, Kelvin C. Luk, Rosario Sánchez‐Pernaute, Juan Carlos Gómez‐Esteban, Daniel Felsky, Yasujiro Kiyota, Nidhi Sahni, S. Stephen Yi, Chee Yeun Chung, Henning Stahlberg, Isidró Ferrer, Johannes Schöneberg, Stephen J. Elledge, Ulf Dettmer, Glenda M. Halliday, Tim Bartels, Vikram Khurana

Bibliographic record

VenueNeuron · 2024
Typearticle
Languageen
FieldMedicine
TopicParkinson's Disease Mechanisms and Treatments
Canadian institutionsUniversity of TorontoCentre for Addiction and Mental Health
FundersNational Institute of Neurological Disorders and StrokeNational Institute of General Medical SciencesNational Institute on AgingNational Health and Medical Research CouncilMichael J. Fox Foundation for Parkinson's ResearchAmerican Parkinson Disease AssociationStanley Center for Psychiatric Research, Broad InstituteBrigham Research InstituteNational Institutes of HealthAmerican Academy of NeurologyDeutscher Akademischer AustauschdienstNew York Stem Cell FoundationNeuroscience Research AustraliaPharmaceutical Research and Manufacturers of America FoundationFoellinger Foundation
KeywordsNeurodegenerationBiologyNeuroprotectionProteomeTransgeneCell biologyRHOANeuroscienceGeneticsGeneSignal transductionPathologyMedicine

Abstract

fetched live from OpenAlex

The heterogeneity of protein-rich inclusions and its significance in neurodegeneration is poorly understood. Standard patient-derived iPSC models develop inclusions neither reproducibly nor in a reasonable time frame. Here, we developed screenable iPSC "inclusionopathy" models utilizing piggyBac or targeted transgenes to rapidly induce CNS cells that express aggregation-prone proteins at brain-like levels. Inclusions and their effects on cell survival were trackable at single-inclusion resolution. Exemplar cortical neuron α-synuclein inclusionopathy models were engineered through transgenic expression of α-synuclein mutant forms or exogenous seeding with fibrils. We identified multiple inclusion classes, including neuroprotective p62-positive inclusions versus dynamic and neurotoxic lipid-rich inclusions, both identified in patient brains. Fusion events between these inclusion subtypes altered neuronal survival. Proteome-scale α-synuclein genetic- and physical-interaction screens pinpointed candidate RNA-processing and actin-cytoskeleton-modulator proteins like RhoA whose sequestration into inclusions could enhance toxicity. These tractable CNS models should prove useful in functional genomic analysis and drug development for proteinopathies.

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 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)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation 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 score0.137
Threshold uncertainty score0.368

Codex and Gemma teacher scores by category

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

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 head0.017
GPT teacher head0.255
Teacher spread0.237 · 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 teacher head, 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

Citations29
Published2024
Admission routes1
Has abstractyes

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