The National Heart, Lung, and Blood Institute (NHLBI) awarded Duke University a $762,974 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to investigate alveolar injury and repair in Pompe disease. The research aims to determine if Pompe disease disrupts alveolar repair following lung injury, elucidate the impact of Pompe on autophagy and cellular homeostasis in alveolar type 2 cells, and evaluate whether gene therapy can treat alveolar pathology. This 4-year project will leverage Duke's expertise in Pompe disease, respiratory physiology, alveolar stem cells, and genetic/epigenetic analysis. The findings are expected to provide clinically relevant insights to inform future therapeutic interventions for respiratory complications in Pompe disease.
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