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Record W2076265228 · doi:10.1186/s13023-015-0248-3

Defects in fatty acid amide hydrolase 2 in a male with neurologic and psychiatric symptoms

2015· article· en· W2076265228 on OpenAlexafffund
Sandra Sirrs, Clara DM van Karnebeek, Xiaoxue Peng, Casper Shyr, Maja Tarailo‐Graovac, Rupasri Mandal, Daniel Testa, Devin Dubin, Gregory Carbonetti, Steven E. Glynn, Bryan Sayson, Wendy P. Robinson, David S. Wishart, Colin J.D. Ross, Wyeth W. Wasserman, Trevor A. Hurwitz, Graham Sinclair, Martin Kaczocha

Bibliographic record

VenueOrphanet Journal of Rare Diseases · 2015
Typearticle
Languageen
FieldMedicine
TopicCannabis and Cannabinoid Research
Canadian institutionsUniversity of British Columbia HospitalSpinal Cord Injury BCPancreas Centre (Canada)British Columbia Centre of Excellence for Women's HealthWorld Wildlife Fund CanadaUniversity of AlbertaChild and Family Research InstituteBC Children's HospitalUniversity of British Columbia
FundersNational Institute on Drug AbuseGenome British ColumbiaMichael Smith Health Research BCChildren's Hospital FoundationNational Institute of General Medical SciencesRadboud UniversiteitCanadian Institutes of Health ResearchGenome Canada
KeywordsMissense mutationFatty acid amide hydrolaseExome sequencingSanger sequencingAtaxiaCompound heterozygosityAutismBiologyMutationMedicineGeneticsPsychiatryGeneCannabinoid receptor

Abstract

fetched live from OpenAlex

BACKGROUND: Fatty acid amide hydrolase 2 (FAAH2) is a hydrolase that mediates the degradation of endocannabinoids in man. Alterations in the endocannabinoid system are associated with a wide variety of neurologic and psychiatric conditions, but the phenotype and biochemical characterization of patients with genetic defects of FAAH2 activity have not previously been described. We report a male with autistic features with an onset before the age of 2 years who subsequently developed additional features including anxiety, pseudoseizures, ataxia, supranuclear gaze palsy, and isolated learning disabilities but was otherwise cognitively intact as an adult. METHODS AND RESULTS: Whole exome sequencing identified a rare missense mutation in FAAH2, hg19: g.57475100G > T (c.1372G > T) resulting in an amino acid change (p.Ala458Ser), which was Sanger confirmed as maternally inherited and absent in his healthy brother. Alterations in lipid metabolism with abnormalities of the whole blood acyl carnitine profile were found. Biochemical and molecular modeling studies confirmed that the p.Ala458Ser mutation results in partial inactivation of FAAH2. Studies in patient derived fibroblasts confirmed a defect in FAAH2 activity resulting in altered levels of endocannabinoid metabolites. CONCLUSIONS: We propose that genetic alterations in FAAH2 activity contribute to neurologic and psychiatric disorders in humans.

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: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.013
Threshold uncertainty score0.399

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 head0.012
GPT teacher head0.264
Teacher spread0.252 · 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 designObservational
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

Citations30
Published2015
Admission routes2
Has abstractyes

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