Research ArticleLung Disease

Pulmonary transplantation of macrophage progenitors as effective and long-lasting therapy for hereditary pulmonary alveolar proteinosis

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Science Translational Medicine  20 Aug 2014:
Vol. 6, Issue 250, pp. 250ra113
DOI: 10.1126/scitranslmed.3009750

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Macrophages Treat Rare Lung Disease

Innate immune cell transplant into the lung could be an effective treatment for a rare lung disease. Happle et al. report that transplanting macrophage progenitors into lungs of a mouse model of hereditary pulmonary alveolar proteinosis (herPAP) improved lung function for up to 9 months after transplant. herPAP is caused by mutations in the granulocyte-macrophage colony-stimulating factor receptor genes, resulting in disturbed alveolar macrophage differentiation and life-threatening respiratory problems. A single transplantation of macrophage progenitors into a mouse model of herPAP resulted in differentiation into functional alveolar macrophages. If these data hold true in humans, this could not only provide a new treatment modality for herPAP but also serve as a proof of principle for other genetic diseases.