학술논문

Endophenotype effect sizes support variant pathogenicity in monogenic disease susceptibility genes
Document Type
article
Source
Nature Communications. 13(1)
Subject
Epidemiology
Biological Sciences
Health Sciences
Genetics
Clinical Research
Aetiology
2.1 Biological and endogenous factors
Metabolic and endocrine
Good Health and Well Being
Disease Susceptibility
Endophenotypes
Humans
Long QT Syndrome
Virulence
NHLBI Trans-Omics for Precision Medicine (TOPMed) Consortium
Language
Abstract
Accurate and efficient classification of variant pathogenicity is critical for research and clinical care. Using data from three large studies, we demonstrate that population-based associations between rare variants and quantitative endophenotypes for three monogenic diseases (low-density-lipoprotein cholesterol for familial hypercholesterolemia, electrocardiographic QTc interval for long QT syndrome, and glycosylated hemoglobin for maturity-onset diabetes of the young) provide evidence for variant pathogenicity. Effect sizes are associated with pathogenic ClinVar assertions (P