Evidence map›Paper›PMID 39241757›Full record

ReviewAmerican journal of human genetics2024

Implementation of a dyadic nomenclature for monogenic diseases.

Courtney Thaxton, Leslie G Biesecker, Marina DiStefano, Melissa Haendel, Ada Hamosh, Emma Owens, Sharon E Plon, Heidi L Rehm, Jonathan S Berg

Abstract readReview
In one paragraph

Review in American journal of human genetics, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing 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

14 citing papers in PubMed.

  1. Evidence-based classification of genes implicated in craniosynostosis disorders using the ClinGen curation framework.Genetics in medicine : official journal of the American College of Medical Genetics · 2026
    Article
  2. Review
  3. Identification of biallelic loss-of-functionJournal of medical genetics · 2026
    Article
  4. Review
  5. Article
  6. Article
  7. Article
  8. Evidence-based classification of genes implicated in skeletal disorders using the ClinGen curation framework.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2026
    Article
  9. Article
  10. Article
  11. Article
  12. Article
  13. Towards a WHO classification of genetic tumour syndromes.Medizinische Genetik : Mitteilungsblatt des Berufsverbandes Medizinische Genetik e.V · 2025
    Article
  14. Article
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

9 authors.

Courtney ThaxtonDepartment of Genetics, University of North Carolina, Chapel Hill, NC 27599, USA. Electronic address: courtney_thaxton@med.unc.edu.
Leslie G BieseckerCenter for Precision Health Research, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Marina DiStefanoMedical and Population Genetics, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Melissa HaendelDepartment of Genetics, University of North Carolina, Chapel Hill, NC 27599, USA.
Ada HamoshMcKusick-Nathans Department of Genetic Medicine, Johns Hopkins University, Baltimore, MD 21205, USA.
Emma OwensDepartment of Genetics, University of North Carolina, Chapel Hill, NC 27599, USA.
Sharon E PlonDepartment of Pediatrics/Hematology-Oncology, Baylor College of Medicine, Houston, TX 77030, USA; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Heidi L RehmMedical and Population Genetics, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Jonathan S BergDepartment of Genetics, University of North Carolina, Chapel Hill, NC 27599, USA. Electronic address: jonathan_berg@med.unc.edu.

Funding

Baylor College of Medicine/Stanford University Clinical Genome Resource (CLINGEN)U24HG009649 · NHGRI · BAYLOR COLLEGE OF MEDICINE · PI TERI Ellen KLEIN, Aleksandar Milosavljevic · 2021 to 2026
$31.5M
The Clinical Genome Resource – Advancing genomic medicine through biocuration and expert assessment of genes and variants at scaleU24HG009650 · NHGRI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI JONATHAN S BERG, Jessica Ezzell Hunter · 2021 to 2026
$30.0M
ClinGen AI Data Delivery SupplementU24HG006834 · NHGRI · BROAD INSTITUTE, INC. · PI Marina DiStefano, CHRISTA LESE MARTIN · 2021 to 2026
$26.7M
Resource ProjectU41HG006627 · NHGRI · JOHNS HOPKINS UNIVERSITY · PI HAMOSH, ADA · 2012 to 2021
$18.0M
Rare & Mosaic Disorders - Clinical ResearchZIAHG200388 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI BIESECKER, LESLIE · 2013 to 2025
$17.3M
The Monarch Initiative: Linking Diseases to Model Organism ResourcesR24OD011883 · OD · UNIV OF NORTH CAROLINA CHAPEL HILL · PI HAENDEL, MELISSA A, MUNGALL, CHRISTOPHER J · 2012 to 2024
$16.0M
Improvements to the LinkML framework to support the Phenomics First open science resourceRM1HG010860 · NHGRI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI HAENDEL, MELISSA A, MUNGALL, CHRISTOPHER J · 2020 to 2024
$10.3M
Online Mendelian Inheritance in Man (OMIM)U24HG006627 · NHGRI · JOHNS HOPKINS UNIVERSITY · PI HAMOSH, ADA · 2022 to 2025
$7.7M
Clinical and Molecular Studies of MalformationsZ01HG200328 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI BIESECKER, LESLIE G · 2005 to 2008
$5.5M
GeneMatcher, VariantMatcher and PhenoDB, implementation of new features and connectionsU24HG012184 · NHGRI · JOHNS HOPKINS UNIVERSITY · PI Nara Sobreira · 2022 to 2026
$3.0M
Intramural NIH HHS Z01 HG200328Intramural NIH HHS ZIA HG200388NHGRI NIH HHS RM1 HG010860NHGRI NIH HHS U24 HG006627NHGRI NIH HHS U24 HG006834NHGRI NIH HHS U24 HG009649NHGRI NIH HHS U24 HG009650NHGRI NIH HHS U24 HG012184NHGRI NIH HHS U41 HG006627NIH HHS R24 OD011883
6 · The paper itself

Abstract

A core task when establishing the strength of evidence for a gene's role in a monogenic disorder is determining the appropriate disease entity to curate. Establishing this concept determines which evidence can be applied and quantified toward the final gene-disease validity, variant pathogenicity, or actionability classification. Genes with implications in more than one phenotype can necessitate a process of lumping and splitting, disease reorganization, and updates to disease nomenclature. Reappraisal of the names that are used as labels for disease entities is therefore a necessary and perpetual process. The Clinical Genome Resource (ClinGen), in collaboration with representatives from Monarch Disease Ontology (Mondo) and Online Inheritance in Man (OMIM), formed the Disease Naming Advisory Committee (DNAC) to develop guidance for groups faced with the need to establish the "curated disease entity" for gene-phenotype validity and variant pathogenicity and to update disease names for clinical use when necessary. The objective of this group was to harmonize guidance for disease naming across these nosologic entities and among ClinGen curation groups in collaboration with other disease-related professional groups. Here, we present the initial guidance developed by the DNAC with representative examples provided by the ClinGen expert panels and working groups that warranted nomenclature updates. We also discuss the broader implications of these efforts and their benefits for harmonization of gene-disease validity curation. Overall, this work sheds light on current inconsistencies and/or discrepancies and is designed to engage the broader community on how ClinGen defines monogenic disorders using a consistent approach for disease naming.

Indexed as

Genetic Diseases, InbornTerminology as TopicDatabases, GeneticHumansPhenotype

Identifiers

PMID39241757
PMCPMC11393707

What OpenQuestion holds

Textmetadata
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 OpenQuestion graph.