Successful combination of sequential gene therapy and rescue allo-HSCT in two children with X-CGD - importance of timing

Current Gene Therapy

Volumn 15, Issue 4, 2015, Pages 416-427

  Siler, Ulrich ^a   Paruzynski, Anna ^b   Holtgreve Grez, Heidi ^c   Kuzmenko, Elena ^a   Koehl, Ulrike ^d   Renner, Eleonore D ^e   Alhan, Canan ^f   van de Loosdrecht, Arjan A ^f   Schwäble, Joachim ^g,j   Pfluger, Thomas ^h   Tchinda, Joelle ^a   Schmugge, Markus ^a   Jauch, Anna ^c   Naundorf, Sonja ⁱ   Kühlcke, Klaus ⁱ   Notheis, Gundula ^e   Güngör, Tayfun ^a   Kalle, Christof V ^b   Schmidt, Manfred ^b   Grez, Manuel ^j   Seger, Reinhard ^a   Reichenbach, Janine ^a,k   more..

^a UNIVERSITY CHILDREN S HOSPITAL   (Switzerland)

^b GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

^c UNIVERSITY HOSPITAL HEIDELBERG   (Germany)

^d HANNOVER MEDICAL SCHOOL   (Germany)

^e LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

^f VU UNIVERSITY MEDICAL CENTER   (Netherlands)

^g GOETHE UNIVERSITY   (Germany)

^h UNIVERSITY OF MUNICH   (Germany)

ⁱ BIONTECH AG   (Germany)

^j INSTITUTE FOR TUMOR BIOLOGY AND EXPERIMENTAL THERAPY   (Germany)

^k Zurich Centre for Integrative Human Physiology (ZIHP)   (Germany)

more..

Author keywords

Allo HSCT; CGD; EVI1; Gene therapy; Primary immunodeficiency; Retroviral vector; STAT3; Transactivation

Indexed keywords

BUSULFAN; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE; RETROVIRUS VECTOR; RITUXIMAB; STAT3 PROTEIN; CYBB PROTEIN, HUMAN; DNA BINDING PROTEIN; MECOM PROTEIN, HUMAN; MEMBRANE PROTEIN; STAT3 PROTEIN, HUMAN; TRANSCRIPTION FACTOR;

ARTICLE; ASPERGILLUS NIDULANS; CASE REPORT; CHILD; CHRONIC GRANULOMATOUS DISEASE; CYTOGENETICS; FLOW CYTOMETRY; GENE; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNE DEFICIENCY; MALE; MDS1 GENE; MYELODYSPLASTIC SYNDROME; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; POLYMERASE CHAIN REACTION; PRESCHOOL CHILD; TRANSACTIVATION; X CHROMOSOME LINKED DISORDER; ADVERSE EFFECTS; ASPERGILLOSIS; CHROMOSOME 7; CHROMOSOME DELETION; CLINICAL TRIAL; GAMMARETROVIRUS; GENETICS; GRANULOMATOUS DISEASE, CHRONIC; MYELODYSPLASTIC SYNDROMES; PATHOGENICITY; PROCEDURES; PROTO ONCOGENE;

EMERICELLA NIDULANS;

ASPERGILLOSIS; ASPERGILLUS NIDULANS; CHILD; CHROMOSOME DELETION; CHROMOSOMES, HUMAN, PAIR 7; DNA-BINDING PROTEINS; GAMMARETROVIRUS; GENETIC THERAPY; GRANULOMATOUS DISEASE, CHRONIC; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MALE; MEMBRANE GLYCOPROTEINS; MYELODYSPLASTIC SYNDROMES; NADPH OXIDASE; PROTO-ONCOGENES; STAT3 TRANSCRIPTION FACTOR; TRANSCRIPTION FACTORS;

EID: 84938680047     PISSN: 15665232     EISSN: 18755631     Source Type: Journal    
DOI: 10.2174/1566523215666150515145255     Document Type: Article

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