Gene correction of human induced pluripotent stem cells repairs the cellular phenotype in pulmonary alveolar proteinosis
Summary
These data establish PAP-iPSC-derived monocytes and macrophages as a valid in vitro disease model of CSF2RA-deficient PAP, and introduce gene-corrected iPSC-derived monocytes and macrophages as a potential autologous cell source for innovative therapeutic strategies. Transplantation of such cells to patients with hPAP could serve as a paradigmatic proof for the potential of iPSC-derived cells in clinical gene therapy.
| Authors | Lachmann N, Happle C, Ackermann M, Lüttge D, Wetzke M, Merkert S, Hetzel M, Kensah G, Jara-Avaca M, Mucci A, Skuljec J, Dittrich AM, Pfaff N, Brennig S, Schambach A, Steinemann D, Göhring G, Cantz T, Martin U, Schwerk N, Hansen G, Moritz T |
|---|---|
| Journal | American journal of respiratory and critical care medicine |
| Publication Date | 2014 Jan 15;189(2):167-82 |
| PubMed | 24279725 |
| DOI | 10.1164/rccm.201306-1012oc |