Cell therapy with lentiviral transduced CSF2RA transgene in the treatment of hereditary pulmonary alveolar proteinosis
摘要:
The disclosure provides compositions and methods for cell therapy of hPAP in humans using gene corrected and in vitro differentiated patient autologous macrophage cells. The disclosure also provides compositions in the form of a cell product and related compositions and methods for making the cell product and for direct pulmonary transplantation of same.
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