CareLit · DOI-Nachweis · Pneumologie
Preclinical assessment of improved lentiviral vectors for gene and cell therapy of pulmonary alveolar proteinosis
DOI 10.1055/s-0034-1376845
Bibliografische Angaben
Nachweis in CareLit
Autor:innenM Hetzel, N Lachmann, C Happle, A Kuhn, M Ackermann, S Brennig, A Schambach, G Hansen, T Moritz
ZeitschriftPneumologie
Ausgabe06/2014
Jahrgang / SeitenNicht angegeben
Kernaussage aus der Literatur
Abstract-Vorschau
Congenital pulmonary alveolar proteinosis (PAP) caused by mutations in GM-CSF receptor α chain (CSF2RA) represents a rare, life-threatening disease characterized by the accumulation of phospholipids and proteins in the lungs due to a functional insufficiency of alveolar macrophages. So far, therapy options are limited, but recent data suggest that a gene therapy approach based on the intratracheal application of gen…
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Zitierempfehlung
M Hetzel, N Lachmann, C Happle, A Kuhn, M Ackermann, S Brennig, A Schambach, G Hansen, T Moritz (2014) Preclinical assessment of improved lentiviral vectors for gene and cell therapy of pulmonary alveolar proteinosis. Pneumologie (06). https://doi.org/10.1055/s-0034-1376845
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