Cell therapy with lentiviral transduced CSF2RA transgene in the treatment of hereditary pulmonary alveolar proteinosis
AssigneesChildren's Hospital Medical Center, Medizinische Hochschule Hannover
InventorsBruce C. Trapnell, Takuji Suzuki, Thomas Moritz, Nico Lachmann, Axel Schambach
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Abstract
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.
Claims28
A self-inactivating lentiviral vector comprising a human CSF2RA cDNA, the human CSF2RA cDNA comprising the sequence identified by nucleotides 2084 to 3299 of SEQ ID NO:1.
A method for treating hPAP in a human subject in need thereof, the method comprising administering to a lung segment of the subject a therapeutic amount of a composition comprising non-naturally occurring in vitro differentiated human macrophage cells derived from CD34+ cells transduced with a self-inactivating lentiviral vector comprising a human CSF2RA cDNA, the human CSF2RA cDNA comprising the sequence identified by nucleotides 2084 to 3299 of SEQ ID NO:1, wherein the macrophage cells express the human CSF2RA.
A composition comprising virions containing a self-inactivating lentiviral vector comprising a human CSF2RA cDNA, the human CSF2RA cDNA comprising the sequence identified by nucleotides 2084 to 3299 of SEQ ID NO:1.
Dependencies are inferred from the claim text.