What is new about the genetic background of Hirschsprung disease?

Clin Genet. 2020 Jan;97(1):114-124. doi: 10.1111/cge.13615. Epub 2019 Aug 5.

Abstract

Hirschsprung disease (HSCR) is a rare congenital disorder caused by an incorrect enteric nervous system development due to a failure in migration, proliferation, differentiation and/or survival of enteric neural crest cells. HSCR is a complex genetic disease, where alterations at different molecular levels are required for the manifestation of the disease. In addition, a wide spectrum of mutations affecting many different genes cause HSCR, although the occurrence and severity of HSCR from many cases still remain unexplained. This review summarizes the current knowledge about molecular genetic basis of HSCR.

Keywords: Hirschsprung disease; additive model; genetics; polygenic disease.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Hirschsprung Disease / genetics*
  • Hirschsprung Disease / pathology
  • Humans
  • Mutation / genetics
  • Nervous System / metabolism
  • Nervous System / pathology*
  • Nervous System Diseases / genetics*
  • Nervous System Diseases / pathology
  • Neural Crest / growth & development
  • Neural Crest / pathology
  • Proto-Oncogene Proteins c-ret / genetics*
  • Signal Transduction / genetics

Substances

  • Proto-Oncogene Proteins c-ret
  • RET protein, human