CSF2RA gene-corrected macrophages

CSF2RA gene-corrected macrophages is an experimental autologous cell and gene therapy designed to treat hereditary pulmonary alveolar proteinosis (hPAP). The treatment involves harvesting a patient's own bone marrow-derived CD34+ hematopoietic progenitor cells, which are then differentiated into macrophages ex vivo and genetically modified using a lentiviral vector to express a functional version of the CSF2RA gene. This gene encodes the alpha chain of the granulocyte-macrophage colony-stimulating factor (GM-CSF) receptor. The gene-corrected macrophages are then transplanted directly into the patient's lung segments via bronchoscopic instillation (pulmonary macrophage transplantation, or PMT). Once in the lungs, these macrophages are intended to restore normal GM-CSF signaling, enabling the clearance of excess pulmonary surfactant from the alveoli and reversing the respiratory failure characteristic of hPAP.

Other names

PMT therapypulmonary macrophage transplantationlentiviral CSF2RA gene-corrected macrophagesautologous CSF2RA-corrected macrophages