Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency

Molecular Therapy

Volumn 23, Issue 4, 2015, Pages 737-745

  Carmo, Marlene ^a   Risma, Kimberly A ^b   Arumugam, Paritha ^b   Tiwari, Swati ^b   Hontz, Adrianne E ^b   Montiel Equihua, Claudia A ^a   Alonso Ferrero, Maria E ^a   Blundell, Michael P ^a   Schambach, Axel ^c   Baum, Christopher ^c   Malik, Punam ^b   Thrasher, Adrian J ^a   Jordan, Michael B ^b   Gaspar, H Bobby ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY OF CINCINNATI   (United States)

^c HANNOVER MEDICAL SCHOOL   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

GAMMA INTERFERON; LENTIVIRUS VECTOR; PERFORIN; PHOSPHOGLYCERATE KINASE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIGEN SPECIFICITY; ARTICLE; CD8+ T LYMPHOCYTE; CELL MEDIATED CYTOTOXICITY; CONTROLLED STUDY; CYTOKINE RELEASE; GENE EXPRESSION; GENE TRANSFER; HEMATOPOIETIC STEM CELL; HEMOPHAGOCYTIC SYNDROME; IMMUNOREGULATION; LYMPHOBLAST; MOUSE; NATURAL KILLER CELL; NONHUMAN; PROMOTER REGION; TRANSCRIPTION REGULATION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; ANIMAL; DISEASE MODEL; GENETICS; IMMUNOLOGY; LYMPHOHISTIOCYTOSIS, HEMOPHAGOCYTIC; METABOLISM; PHENOTYPE; TRANSGENIC MOUSE;

MURINAE; MUS;

ANIMALS; CD8-POSITIVE T-LYMPHOCYTES; DISEASE MODELS, ANIMAL; GENE TRANSFER TECHNIQUES; HEMATOPOIETIC STEM CELLS; KILLER CELLS, NATURAL; LYMPHOHISTIOCYTOSIS, HEMOPHAGOCYTIC; MICE; MICE, TRANSGENIC; PERFORIN; PHENOTYPE;

EID: 84926979642     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.242     Document Type: Article

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