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Dysregulation of GDNF and GFRα1 expression in multifactorial Hirschsprung disease.

Created on 20 Jun 2026

Authors

Gunadi, Setiani Silvy Nurhidayah, Aleyy Adany, Farah Shahnaz Pravinovia, Siti Maisaroh, Kurnia Corie Tonda, Pramana Adhityo, Estelita Liana, Kristy Iskandar, Andi Dwihantoro

Published in

BMC research notes. Jun 19, 2026. Epub Jun 19, 2026.

Abstract

HSCR is caused by disruption of complex signaling pathways within the gene regulatory network (GRN) during enteric nervous system (ENS) development, including glial cell line-derived neurotrophic factor (GDNF) and GDNF family receptor alpha-1 (GFRα1). However, pathogenic variants in all GRN genes account for only ~ 80% cases; therefore, the epigenetic role remains to be elucidated. We compared GDNF and GFRα1 expression between HSCR patients and controls.
qPCR revealed an upregulated GDNF expression in both ganglionic (206.37-fold) and aganglionic (126.35-fold) HSCR compared to control colons (ΔCT 7.65 ± 2.19 vs. 15.34 ± 1.34; p = 0.0001; and ΔCT 8.35 ± 2.57 vs. 15.34 ± 1.34; p = 0.0001). qPCR also showed an upregulated GFRα1 expression in both ganglionic (29.66-fold) and aganglionic (18.44-fold) HSCR compared to control colons (ΔCT 8.44 ± 1.98 vs. 13.33 ± 1.36; p = 0.0001; and ΔCT 9.13 ± 1.46 vs. 13.33 ± 1.36; p = 0.0001). In addition, no significant differences were observed between ganglionic and aganglionic segments for either GDNF or GFRα1 (ΔCT 7.65 ± 2.19 vs. 8.35 ± 2.57; p = 0.43; and ΔCT 8.44 ± 1.98 vs. 9.13 ± 1.46; p = 0.29). Our study demonstrates global aberrant expression of GDNF and GFRα1 in HSCR patients. These findings underscore the complexity of HSCR as a multifactorial disorder and highlight the importance of integrated signaling networks in ENS development, providing a rationale for further mechanistic and translational studies.

PMID:
42321920
Bibliographic data and abstract were imported from PubMed on 20 Jun 2026.

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