Long Non-coding RNA BGas Regulates the Cystic Fibrosis Transmembrane Conductance Regulator

Mol Ther. 2016 Aug;24(8):1351-7. doi: 10.1038/mt.2016.112. Epub 2016 May 30.

Abstract

Cystic fibrosis (CF) is a life-shortening genetic disease. The root cause of CF is heritable recessive mutations that affect the cystic fibrosis transmembrance conductance regulator (CFTR) gene and the subsequent expression and activity of encoded ion channels at the cell surface. We show that CFTR is regulated transcriptionally by the actions of a novel long noncoding RNA (lncRNA), designated as BGas, that emanates from intron 11 of the CFTR gene and is expressed in the antisense orientation relative to the protein coding sense strand. We find that BGas functions in concert with several proteins including HMGA1, HMGB1, and WIBG to modulate the local chromatin and DNA architecture of intron 11 of the CFTR gene and thereby affects transcription. Suppression of BGas or its associated proteins results in a gain of both CFTR expression and chloride ion function. The observations described here highlight a previously underappreciated mechanism of transcriptional control and suggest that BGas may serve as a therapeutic target for specifically activating expression of CFTR.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Cystic Fibrosis / genetics*
  • Cystic Fibrosis / metabolism
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics*
  • DNA-Binding Proteins / metabolism
  • Gene Expression Regulation*
  • Genetic Loci
  • Humans
  • Models, Biological
  • Protein Binding
  • RNA, Antisense / genetics*
  • RNA, Long Noncoding*

Substances

  • DNA-Binding Proteins
  • RNA, Antisense
  • RNA, Long Noncoding
  • Cystic Fibrosis Transmembrane Conductance Regulator