Microvillus inclusion disease (MVID) is a rare congenital enteropathy in infants causing severe diarrhea and inability to absorb food. Previously fatal, advances in parenteral nutrition have made it manageable but require daily treatments. Investigating the root cause, researchers created organoids from patient cells, revealing dysfunctional electrolyte transport as the cause of fluid loss. A chloride channel blocker reduced fluid secretion, and a gamma-secretase inhibitor showed promise in restoring microvilli function. Clinical trials are underway for potential treatments. The research represents a significant step towards understanding and potentially reversing the disease itself.
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From organoids to new treatments
