Published in Gene Therapy Weekly, July 11th, 2002
CF is a genetic disease characterized in the lung by abnormal chloride and water transport from the pulmonary lining cells into the lumen. This anomaly causes the chronic formation of tenacious mucus, leading to infection and a vicious cycle of inflammatory lung damage that debilitates and leads to early death.
Moli1901 may normalize the mucus composition in the lung by activating an alternative chloride and water channel that is present in the lung lining cells of CF patients.
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