Small noncoding differentially methylated copy-number variants, including lncRNA genes, cause a lethal lung developmental disorder

Genome Res. 2013 Jan;23(1):23-33. doi: 10.1101/gr.141887.112. Epub 2012 Oct 3.

Abstract

An unanticipated and tremendous amount of the noncoding sequence of the human genome is transcribed. Long noncoding RNAs (lncRNAs) constitute a significant fraction of non-protein-coding transcripts; however, their functions remain enigmatic. We demonstrate that deletions of a small noncoding differentially methylated region at 16q24.1, including lncRNA genes, cause a lethal lung developmental disorder, alveolar capillary dysplasia with misalignment of pulmonary veins (ACD/MPV), with parent-of-origin effects. We identify overlapping deletions 250 kb upstream of FOXF1 in nine patients with ACD/MPV that arose de novo specifically on the maternally inherited chromosome and delete lung-specific lncRNA genes. These deletions define a distant cis-regulatory region that harbors, besides lncRNA genes, also a differentially methylated CpG island, binds GLI2 depending on the methylation status of this CpG island, and physically interacts with and up-regulates the FOXF1 promoter. We suggest that lung-transcribed 16q24.1 lncRNAs may contribute to long-range regulation of FOXF1 by GLI2 and other transcription factors. Perturbation of lncRNA-mediated chromatin interactions may, in general, be responsible for position effect phenomena and potentially cause many disorders of human development.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Chromatin / metabolism
  • Chromosomes, Human, Pair 16 / genetics
  • CpG Islands
  • DNA Copy Number Variations*
  • DNA Methylation*
  • Enhancer Elements, Genetic
  • Fatal Outcome
  • Forkhead Transcription Factors / genetics
  • Forkhead Transcription Factors / metabolism
  • Gene Expression Regulation
  • Genomic Imprinting
  • HEK293 Cells
  • Humans
  • Infant, Newborn
  • Kruppel-Like Transcription Factors / metabolism
  • Nuclear Proteins / metabolism
  • Persistent Fetal Circulation Syndrome / diagnosis
  • Persistent Fetal Circulation Syndrome / genetics*
  • Promoter Regions, Genetic
  • RNA, Long Noncoding / genetics*
  • RNA, Long Noncoding / metabolism
  • Sequence Deletion
  • Transcription, Genetic
  • Zinc Finger Protein Gli2

Substances

  • Chromatin
  • FOXF1 protein, human
  • Forkhead Transcription Factors
  • GLI2 protein, human
  • Kruppel-Like Transcription Factors
  • Nuclear Proteins
  • RNA, Long Noncoding
  • Zinc Finger Protein Gli2

Associated data

  • GEO/GSE39258