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Tuesday, February 17, 2015

Gene correction of human induced pluripotent stem cells repairs the...

Hereditary pulmonary alveolar proteinosis caused by granulocyte-macrophage colony-stimulating factor receptor -chain deficiency is a rare, life-threatening lung disease characterized by accumulation of proteins and phospholipids in the alveolar spaces. The disease is caused by a functional insufficiency of alveolar macrophages, which require GM-CSF signaling for terminal differentiation and effective degradation of alveolar proteins and phospholipids.

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