Identification of rare de novo epigenetic variations in congenital disorders

Nat Commun. 2018 May 25;9(1):2064. doi: 10.1038/s41467-018-04540-x.

Abstract

Certain human traits such as neurodevelopmental disorders (NDs) and congenital anomalies (CAs) are believed to be primarily genetic in origin. However, even after whole-genome sequencing (WGS), a substantial fraction of such disorders remain unexplained. We hypothesize that some cases of ND-CA are caused by aberrant DNA methylation leading to dysregulated genome function. Comparing DNA methylation profiles from 489 individuals with ND-CAs against 1534 controls, we identify epivariations as a frequent occurrence in the human genome. De novo epivariations are significantly enriched in cases, while RNAseq analysis shows that epivariations often have an impact on gene expression comparable to loss-of-function mutations. Additionally, we detect and replicate an enrichment of rare sequence mutations overlapping CTCF binding sites close to epivariations, providing a rationale for interpreting non-coding variation. We propose that epivariations contribute to the pathogenesis of some patients with unexplained ND-CAs, and as such likely have diagnostic relevance.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Case-Control Studies
  • Child
  • Child, Preschool
  • Cohort Studies
  • Congenital Abnormalities / genetics*
  • DNA Methylation / genetics
  • Datasets as Topic
  • Epigenesis, Genetic*
  • Epigenomics / methods
  • Genome, Human / genetics*
  • Humans
  • Infant
  • Infant, Newborn
  • Loss of Function Mutation / genetics
  • Male
  • Middle Aged
  • Neurodevelopmental Disorders / genetics*
  • Sequence Analysis, DNA
  • Sequence Analysis, RNA
  • Young Adult