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Record W4388518164 · doi:10.1093/hmg/ddad188

Clinical features, functional consequences, and rescue pharmacology of missense <i>GRID1</i> and <i>GRID2</i> human variants

2023· article· en· W4388518164 on OpenAlexaff
James P. Allen, Kathryn B. Garber, Riley E. Perszyk, Cara T Khayat, Steven A. Kell, Maki Kaneko, Catherine Quindipan, Sulagna C. Saitta, Roger L. Ladda, Stacy Hewson, Michal Inbar‐Feigenberg, Chitra Prasad, Asuri N. Prasad, Leah Olewiler, Weiyi Mu, Liana S. Rosenthal, Marcello Scala, Pasquale Striano, Federico Zara, Tyler W. McCullock, Robin‐Tobias Jauss, Johannes R. Lemke, David M. MacLean, Cheng Zhu, Hongjie Yuan, Scott J. Myers, Stephen F. Traynelis

Bibliographic record

VenueHuman Molecular Genetics · 2023
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicGenetics and Neurodevelopmental Disorders
Canadian institutionsWestern UniversityChildren's Hospital of Western OntarioUniversity of TorontoSickKids FoundationCanada Research ChairsHospital for Sick Children
FundersNational Center for Advancing Translational SciencesNational Institute of Allergy and Infectious DiseasesNational Institute of Neurological Disorders and StrokeNational Institute of General Medical SciencesEmory UniversityNational Institute on AgingNational Cancer InstituteNational Institutes of HealthSage TherapeuticsEunice Kennedy Shriver National Institute of Child Health and Human DevelopmentSimons Foundation Autism Research Initiative
KeywordsBiologyMissense mutationPhenotypeReceptorGeneTransmembrane domainGeneticsMutationCell biologyNeuroscience

Abstract

fetched live from OpenAlex

GRID1 and GRID2 encode the enigmatic GluD1 and GluD2 proteins, which form tetrameric receptors that play important roles in synapse organization and development of the central nervous system. Variation in these genes has been implicated in neurodevelopmental phenotypes. We evaluated GRID1 and GRID2 human variants from the literature, ClinVar, and clinical laboratories and found that many of these variants reside in intolerant domains, including the amino terminal domain of both GRID1 and GRID2. Other conserved regions, such as the M3 transmembrane domain, show different intolerance between GRID1 and GRID2. We introduced these variants into GluD1 and GluD2 cDNA and performed electrophysiological and biochemical assays to investigate the mechanisms of dysfunction of GRID1/2 variants. One variant in the GRID1 distal amino terminal domain resides at a position predicted to interact with Cbln2/Cbln4, and the variant disrupts complex formation between GluD1 and Cbln2, which could perturb its role in synapse organization. We also discovered that, like the lurcher mutation (GluD2-A654T), other rare variants in the GRID2 M3 domain create constitutively active receptors that share similar pathogenic phenotypes. We also found that the SCHEMA schizophrenia M3 variant GluD1-A650T produced constitutively active receptors. We tested a variety of compounds for their ability to inhibit constitutive currents of GluD receptor variants and found that pentamidine potently inhibited GluD2-T649A constitutive channels (IC50 50 nM). These results identify regions of intolerance to variation in the GRID genes, illustrate the functional consequences of GRID1 and GRID2 variants, and suggest how these receptors function normally and in disease.

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 categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Bench or experimental · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.538
Threshold uncertainty score1.000

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 studies0.0000.001
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 head0.027
GPT teacher head0.313
Teacher spread0.286 · 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.

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

Citations13
Published2023
Admission routes1
Has abstractyes

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