Gene correction of human induced pluripotent stem cells repairs the cellular phenotype in pulmonary alveolar proteinosis

Hereditary pulmonary alveolar proteinosis (hPAP) caused by granulocyte-macrophage colony-stimulating factor (GM-CSF) receptor α-chain (CSF2RA) deficiency is a rare, life-threatening lung disease characterized by accumulation of proteins and phospholipids in the alveolar spaces. The disease is caused...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Veröffentlicht in:	American journal of respiratory and critical care medicine 2014-01, Vol.189 (2), p.167-182
Hauptverfasser:	Lachmann, Nico, Happle, Christine, Ackermann, Mania, Lüttge, Doreen, Wetzke, Martin, Merkert, Sylvia, Hetzel, Miriam, Kensah, George, Jara-Avaca, Monica, Mucci, Adele, Skuljec, Jelena, Dittrich, Anna-Maria, Pfaff, Nils, Brennig, Sebastian, Schambach, Axel, Steinemann, Doris, Göhring, Gudrun, Cantz, Tobias, Martin, Ulrich, Schwerk, Nicolaus, Hansen, Gesine, Moritz, Thomas
Format:	Artikel
Sprache:	eng
Schlagworte:	Bone marrow Cell Culture Techniques Cell Differentiation - drug effects Cell Differentiation - genetics Child, Preschool Female Fibroblasts Gene therapy Genetic Diseases, X-Linked - genetics Genetic Diseases, X-Linked - metabolism Genetic Diseases, X-Linked - therapy Genetic Therapy Granulocytes Humans Induced Pluripotent Stem Cells Infections Lung diseases Macrophages, Alveolar - metabolism Models, Biological Monocytes - metabolism Patients Phosphorylation Proteins Pulmonary Alveolar Proteinosis - genetics Pulmonary Alveolar Proteinosis - metabolism Pulmonary Alveolar Proteinosis - therapy Receptors, Granulocyte-Macrophage Colony-Stimulating Factor - deficiency Receptors, Granulocyte-Macrophage Colony-Stimulating Factor - genetics Receptors, Granulocyte-Macrophage Colony-Stimulating Factor - therapeutic use Signal Transduction - drug effects Signal Transduction - genetics Stem cells
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Schreiben Sie den ersten Kommentar!