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Congenital Pulmonary Alveolar Proteinosis iPS-derived hematopoietic progenitor cells (HPCs) reveal functional defects upon GM-CSF administration

A Mucci, N Lachmann, C Happle, M Ackermann, S Glage, G Hansen, T Moritz · Pneumologie · Ausgabe 06/2014

DOI 10.1055/s-0034-1376847

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Autor:innenA Mucci, N Lachmann, C Happle, M Ackermann, S Glage, G Hansen, T Moritz
ZeitschriftPneumologie
Ausgabe06/2014
Jahrgang / SeitenNicht angegeben

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Abstract-Vorschau

Hereditary Pulmonary Alveolar Proteinosis (herPAP), caused by a mutation in the GM-CSF receptor β-chain (Csf2rb), is an extremely rare lung disease resulting from the inability of alveolar macrophages to clear the alveolar spaces from surfactant phospholipids. Since current treatment options are extremely limited, we here investigate the suitability of a gene therapy approach based on hematopoietic cells derived fro…

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A Mucci, N Lachmann, C Happle, M Ackermann, S Glage, G Hansen, T Moritz (2014) Congenital Pulmonary Alveolar Proteinosis iPS-derived hematopoietic progenitor cells (HPCs) reveal functional defects upon GM-CSF administration. Pneumologie (06). https://doi.org/10.1055/s-0034-1376847

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