Evidence map›Paper›PMID 40174224›Full record

ArticleThe New England journal of medicine2025

The Genetic Architecture of Congenital Diarrhea and Enteropathy.

Zeenat Gaibee, Neil Warner, Katlynn Bugda Gwilt, Wenjuan Li, Rei Guan, Michael Yourshaw, Ryder Whittaker Hawkins, Christiane Zorbas, Jonathan St-Germain, Mahdi Tabatabaie and 24 more

Abstract read
In one paragraph

Article in The New England journal of medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

9 citing papers in PubMed.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

34 authors.

Zeenat GaibeeDivision of Gastroenterology, Hepatology, and Nutrition, Hospital for Sick Children, Toronto.
Neil WarnerCell Biology Program, Research Institute, Hospital for Sick Children, Toronto.
Katlynn Bugda GwiltDivision of Gastroenterology, Hepatology, and Nutrition, Boston Children's Hospital and Harvard Medical School, Boston.
Wenjuan LiCell Biology Program, Research Institute, Hospital for Sick Children, Toronto.
Rei GuanCell Biology Program, Research Institute, Hospital for Sick Children, Toronto.
Michael YourshawDepartment of Human Genetics, University of California, Los Angeles, Los Angeles.
Ryder Whittaker HawkinsCell Biology Program, Research Institute, Hospital for Sick Children, Toronto.
Christiane ZorbasRNA Molecular Biology, Fonds de la Recherche Scientifique, Université Libre de Bruxelles, BioPark Campus, Gosselies, Belgium.
Jonathan St-GermainPrincess Margaret Cancer Centre, University Health Network, and Department of Medical Biophysics, University of Toronto, Toronto.
Mahdi TabatabaiePrincess Margaret Cancer Centre, University Health Network, and Department of Medical Biophysics, University of Toronto, Toronto.
Suli MaoDepartment of Cell and Developmental Biology, Epithelial Biology Center, Vanderbilt University School of Medicine, Nashville.
Vered PinskDivision of Pediatrics, Pediatric Gastroenterology Unit, Soroka University Medical Center and Faculty of Health Sciences, Ben-Gurion University of the Negev, Be'er Sheva, Israel.
Baruch YerushalmiDivision of Pediatrics, Pediatric Gastroenterology Unit, Soroka University Medical Center and Faculty of Health Sciences, Ben-Gurion University of the Negev, Be'er Sheva, Israel.
Lee-Kai WangDepartment of Human Genetics, University of California, Los Angeles, Los Angeles.
Stanley F NelsonDepartment of Human Genetics, University of California, Los Angeles, Los Angeles.
Laura WozniakDepartment of Pediatrics, Cedars-Sinai Medical Center, Los Angeles.
Dror S ShouvalInstitute of Gastroenterology, Nutrition, and Liver Diseases, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.
Manar MatarInstitute of Gastroenterology, Nutrition, and Liver Diseases, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.
Amit AssaJuliet Keidan Institute of Pediatric Gastroenterology and Nutrition, Shaare Zedek Medical Center, Hebrew University of Jerusalem, Jerusalem.
Nathaniel FrostCell Biology Program, Research Institute, Hospital for Sick Children, Toronto.
Lissette JimenezDivision of Gastroenterology, Hepatology, and Nutrition, Boston Children's Hospital and Harvard Medical School, Boston.
Sari AcraDivision of Pediatric Gastroenterology, Hepatology, and Nutrition, Vanderbilt University Medical Center, Nashville.
Thomas WaltersDivision of Gastroenterology, Hepatology, and Nutrition, Hospital for Sick Children, Toronto.
Stephen MouatDepartment of Paediatric Gastroenterology and Hepatology, Starship Children's Health, Te Toka Tumai Auckland, Auckland, New Zealand.
Michael LiCenter for Computational Medicine, Research Institute, Hospital for Sick Children, Toronto.
Denis L J LafontaineRNA Molecular Biology, Fonds de la Recherche Scientifique, Université Libre de Bruxelles, BioPark Campus, Gosselies, Belgium.
Matthew TyskaDepartment of Cell and Developmental Biology, Epithelial Biology Center, Vanderbilt University School of Medicine, Nashville.
Brian RaughtPrincess Margaret Cancer Centre, University Health Network, and Department of Medical Biophysics, University of Toronto, Toronto.
Yaron AvitzurDivision of Gastroenterology, Hepatology, and Nutrition, Hospital for Sick Children, Toronto.
Wayne I LencerDivision of Gastroenterology, Hepatology, and Nutrition, Boston Children's Hospital and Harvard Medical School, Boston.
James R GoldenringDepartment of Cell and Developmental Biology, Epithelial Biology Center, Vanderbilt University School of Medicine, Nashville.
Martín G MartínDepartment of Pediatrics, Division of Gastroenterology and Nutrition, Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, Mattel Children's Hospital, and the David Geffen School of Medicine, University of California, Los Angeles, Los Angeles.
Jay R ThiagarajahDivision of Gastroenterology, Hepatology, and Nutrition, Boston Children's Hospital and Harvard Medical School, Boston.
Aleixo M MuiseDivision of Gastroenterology, Hepatology, and Nutrition, Hospital for Sick Children, Toronto.ORCID 0000-0001-9624-3346

Funding

COngenital Diarrhea and Enteropathy (PediCODE) Consortium and BioRepositoryRC2DK118640 · NIDDK · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI James Richard Goldenring, Izumi Kaji · 2019 to 2026
$14.8M
Identifying Immune and Epithelial Network Signatures in Very Early Onset Inflammatory Bowel DiseaseRC2DK122532 · NIDDK · BOSTON CHILDREN'S HOSPITAL · PI KLEIN, CHRISTOPH, MUISE, ALEIXO M · 2020 to 2024
$9.1M
Molecular basis of brush border assemblyR01DK095811 · NIDDK · VANDERBILT UNIVERSITY · PI MATTHEW J TYSKA · 2013 to 2026
$4.9M
Role of I-BAR proteins in enterocyte differentiationR01DK111949 · NIDDK · VANDERBILT UNIVERSITY · PI MATTHEW J TYSKA · 2017 to 2026
$4.2M
Myosin-2 function in the enterocyte terminal webR01DK125546 · NIDDK · VANDERBILT UNIVERSITY · PI TYSKA, MATTHEW J · 2021 to 2024
$1.4M
NIDDK NIH HHS R01 DK095811NIDDK NIH HHS R01 DK111949NIDDK NIH HHS R01 DK125546NIDDK NIH HHS RC2 DK118640NIDDK NIH HHS RC2DK118640NIDDK NIH HHS RC2 DK122532NIDDK NIH HHS RC2DK122532
6 · The paper itself

Abstract

backgroundNext-generation sequencing has enabled precision therapeutic approaches that have improved the lives of children with rare diseases. Congenital diarrhea and enteropathies (CODEs) are associated with high morbidity and mortality. Although treatment of these disorders is largely supportive, emerging targeted therapies based on genetic diagnoses include specific diets, pharmacologic treatments, and surgical interventions.

methodsWe analyzed the exomes or genomes of infants with suspected monogenic congenital diarrheal disorders. Using cell and zebrafish models, we tested the effects of variants in newly implicated genes.

resultsIn our case series of 129 infant probands with suspected monogenic congenital diarrheal disorders, we identified causal variants, including a new founder

conclusionsWe have characterized the broad genetic architecture of CODE disorders in a large case series of patients and identified three novel genes associated with CODEs. (Funded by the National Institutes of Health and others.).

Indexed as

Basic Helix-Loop-Helix ProteinsDiarrhea, InfantileNerve Tissue ProteinsAnimalsAnimals, Genetically ModifiedCell LineDisease Models, AnimalExomeFemaleHumansInfantMaleMutationZebrafishZebrafish ProteinsBasic Helix-Loop-Helix ProteinsNerve Tissue ProteinsNEUROG3 protein, humanZebrafish Proteins

Identifiers

PMID40174224
PMCPMC11968080

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.