Functional restoration of gp91phox-oxidase activity by BAC transgenesis and gene targeting in X-linked chronic granulomatous disease iPSCs

Molecular Therapy

Volumn 24, Issue 4, 2016, Pages 812-822

  Laugsch, Magdalena ^a   Rostovskaya, Maria ^b   Velychko, Sergiy ^b   Richter, Cornelia ^a   Zimmer, Ariane ^a   Klink, Barbara ^c   Schröck, Evelin ^c   Haase, Michael ^a   Neumann, Katrin ^b   Thieme, Sebastian ^a   Roesler, Joachim ^a   Brenner, Sebastian ^a   Anastassiadis, Konstantinos ^b  

^a UNIVERSITY HOSPITAL CARL GUSTAV CARUS   (Germany)

^b DRESDEN UNIVERSITY OF TECHNOLOGY   (Germany)

^c DRESDEN UNIVERSITY OF TECHNOLOGY   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

LENTIVIRUS VECTOR; RECOMBINASE; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE 2; CYBB PROTEIN, HUMAN; MEMBRANE PROTEIN; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE;

ARTICLE; AUTOTRANSPLANTATION; BACTERIAL ARTIFICIAL CHROMOSOME; BONE MARROW CELL; CELL DIFFERENTIATION; CONTROLLED STUDY; ENZYME ACTIVITY; GENE TARGETING; GRANULOMATOSIS; HOMOLOGOUS RECOMBINATION; HUMAN; HUMAN CELL; KERATINOCYTE; MONOGENIC DISORDER; PLURIPOTENT STEM CELL; TRANSGENICS; TRANSPOSON; X CHROMOSOME LINKED DISORDER; CELL CULTURE; CYTOLOGY; ENZYMOLOGY; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; GRANULOMATOUS DISEASE, CHRONIC; INDUCED PLURIPOTENT STEM CELL; METABOLISM; MUTATION; PATHOLOGY;

CELL DIFFERENTIATION; CELLS, CULTURED; CHROMOSOMES, ARTIFICIAL, BACTERIAL; GENE TARGETING; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; GRANULOMATOUS DISEASE, CHRONIC; HUMANS; INDUCED PLURIPOTENT STEM CELLS; KERATINOCYTES; MEMBRANE GLYCOPROTEINS; MUTATION; NADPH OXIDASE;

EID: 84943176154     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.154     Document Type: Article

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