iPSCs derived from esophageal atresia patients reveal SOX2 dysregulation at the anterior foregut stage

Dis Model Mech. 2022 Nov 1;15(11):dmm049541. doi: 10.1242/dmm.049541. Epub 2022 Nov 28.

Abstract

A series of well-regulated cellular and molecular events result in the compartmentalization of the anterior foregut into the esophagus and trachea. Disruption of the compartmentalization process leads to esophageal atresia/tracheoesophageal fistula (EA/TEF). The cause of EA/TEF remains largely unknown. Therefore, to mimic the early development of the esophagus and trachea, we differentiated induced pluripotent stem cells (iPSCs) from EA/TEF patients, and iPSCs and embryonic stem cells from healthy individuals into mature three-dimensional esophageal organoids. CXCR4, SOX17 and GATA4 expression was similar in both patient-derived and healthy endodermal cells. The expression of the key transcription factor SOX2 was significantly lower in the patient-derived anterior foregut. We also observed an abnormal expression of NKX2.1 (or NKX2-1) in the patient-derived mature esophageal organoids. At the anterior foregut stage, RNA sequencing revealed the critical genes GSTM1 and RAB37 to be significantly lower in the patient-derived anterior foregut. We therefore hypothesize that a transient dysregulation of SOX2 and the abnormal expression of NKX2.1 in patient-derived cells could be responsible for the abnormal foregut compartmentalization.

Keywords: Anterior foregut; Esophageal atresia/tracheoesophageal fistula; Esophageal organoids; iPSCs.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Esophageal Atresia* / complications
  • Esophageal Atresia* / genetics
  • Humans
  • Induced Pluripotent Stem Cells* / metabolism
  • SOXB1 Transcription Factors / genetics
  • Tracheoesophageal Fistula* / etiology
  • Tracheoesophageal Fistula* / metabolism

Substances

  • SOX2 protein, human
  • SOXB1 Transcription Factors

Supplementary concepts

  • Esophageal atresia with or without tracheoesophageal fistula