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

American Journal of Respiratory and Critical Care Medicine

Volumn 189, Issue 2, 2014, Pages 167-182

  Lachmann, Nico ^a,b   Happle, Christine ^a,c   Ackermann, Mania ^a,b   Lüttge, Doreen ^a,b   Wetzke, Martin ^a   Merkert, Sylvia ^a   Hetzel, Miriam ^a,b   Kensah, George ^a   Jara Avaca, Monica ^a   Mucci, Adele ^a,b   Skuljec, Jelena ^a   Dittrich, Anna Maria ^a,c   Pfaff, Nils ^a,b   Brennig, Sebastian ^a,b   Schambach, Axel ^a,b   Steinemann, Doris ^d   Göhring, Gudrun ^d   Cantz, Tobias ^e   Martin, Ulrich ^a,c   Schwerk, Nicolaus ^a,c   Hansen, Gesine ^a,c   Moritz, Thomas ^a,b   more..

^a HANNOVER MEDICAL SCHOOL   (Germany)

^b Institute of Experimental Hematology   (Germany)

^c Leibniz Research Laboratories for Biotechnology and Artificial Organs   (Germany)

^d German Center for Lung Research   (Germany)

^e BOSTON CHILDREN S HOSPITAL   (United States)

Author keywords

Gene therapy; Hematopoiesis; Induced pluripotent stem cells; Macrophages; Pulmonary alveolar proteinosis

Indexed keywords

CD11B ANTIGEN; CD34 ANTIGEN; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR RECEPTOR; LENTIVIRUS VECTOR; OCTAMER TRANSCRIPTION FACTOR 4; STAT5 PROTEIN; TRANSCRIPTION FACTOR NANOG; TRANSCRIPTION FACTOR SOX2;

ARTERIAL OXYGEN SATURATION; ARTICLE; BONE MARROW CELL; CASE REPORT; CELL DIFFERENTIATION; CELL POPULATION; CHILD; COMPUTER ASSISTED TOMOGRAPHY; CONTROLLED STUDY; COPY NUMBER VARIATION; COUGHING; DYSPNEA; FAILURE TO THRIVE; FEMALE; FLOW CYTOMETRY; GENE MUTATION; GENE TRANSFER; GENETIC ANALYSIS; GERM LAYER; HETEROZYGOTE; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; LENTIVIRINAE; LUNG ALVEOLUS MACROPHAGE; LUNG ALVEOLUS PROTEINOSIS; MACROPHAGE; MONOCYTE; NUCLEOTIDE SEQUENCE; PHAGOCYTOSIS; PHENOTYPE; PLURIPOTENT STEM CELL; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN PHOSPHORYLATION; REAL TIME POLYMERASE CHAIN REACTION; SPECTRAL KARYOTYPING; STABLE EXPRESSION; STEM CELL GENE THERAPY; TARGETED GENE REPAIR; THORAX RADIOGRAPHY; UPREGULATION; VIRUS PNEUMONIA;

CELL CULTURE TECHNIQUES; CELL DIFFERENTIATION; CHILD, PRESCHOOL; FEMALE; GENETIC DISEASES, X-LINKED; GENETIC THERAPY; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MACROPHAGES, ALVEOLAR; MODELS, BIOLOGICAL; MONOCYTES; PULMONARY ALVEOLAR PROTEINOSIS; RECEPTORS, GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR; SIGNAL TRANSDUCTION;

EID: 84892666915     PISSN: 1073449X     EISSN: 15354970     Source Type: Journal    
DOI: 10.1164/rccm.201306-1012OC     Document Type: Article

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