Treatment of overactive K_ATP channels with glibenclamide in a zebrafish model and a clinical trial in humans with Cantu syndrome.

ABSTRACT: This study explores the efficacy of glibenclamide, a K_ATP channel inhibitor, for treating Cantú syndrome (CS), a genetic disorder characterized by hypertrichosis and cardiovascular abnormalities. Treatment with glibenclamide for Cantú syndrome has only been reported in a single case report. In this study, we tested this repurposed drug in both a zebrafish model and an open-label trial with CS patients. CS zebrafish embryos, created using CRISPR/Cas9, were treated with glibenclamide. Their cardiac function was assessed using high-speed imaging. In the trial part of the study, four adults with CS used 2.5 mg glibenclamide daily for 8 months. Hypertrichosis, cardiac function, and edema were evaluated and glucose levels were monitored continuously. In the zebrafish model of CS glibe

SUBMITTER: Kleinendorst L 

PROVIDER: S-EPMC12095479 | biostudies-literature | 2025 May

REPOSITORIES: biostudies-literature