A Potential Treatment of Congenital Sodium Diarrhea in Patients With Activating GUCY2C Mutations.

Anke H M van VugtMarcel J C BijveldsHugo R de JongeKelly F MeijsenTanja RestinManuel B BryantAntje BallauffBart KootThomas MüllerRoderick H J HouwenAndreas R JaneckeSabine Middendorp

Published in: Clinical and translational gastroenterology (2021)

We reported in this study that the GCC inhibitor SSP2518 normalizes cGMP levels in intestinal organoids derived from patients with GCC gain-of-function mutations and markedly reduces cystic fibrosis transmembrane conductance regulator-dependent chloride secretion, the driver of persistent diarrhea.

Keyphrases

cystic fibrosis
irritable bowel syndrome
nitric oxide
pseudomonas aeruginosa
clostridium difficile
lung function
combination therapy
replacement therapy
induced pluripotent stem cells