Gene therapy of chronic granulomatous disease

Bone Marrow Transplantation

Volumn 25, Issue , 2000, Pages S99-S104

  Grez, M ^a   Becker, S ^a   Saulnier, S ^b   Knobeta H ^a   Ott, M G ^c   Maurer, A ^a   Dinauer, M C ^d   Hoelzer, D ^c   Seger, R ^b   Hossle, J P ^b  

^a Laboratory for Molecular Virology   (Germany)

^b UNIVERSITY CHILDREN S HOSPITAL   (Switzerland)

^c GOETHE UNIVERSITY MEDICAL SCHOOL   (Germany)

^d INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Cd34+ cells; Gene therapy; Retroviral vectors; X CGD

Indexed keywords

CD34 ANTIGEN; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE;

ANIMAL CELL; CHRONIC GRANULOMATOUS DISEASE; CONFERENCE PAPER; GENE THERAPY; HUMAN; HUMAN CELL; LEUKEMIA CELL LINE; MOUSE; NEUTROPHIL; NONHUMAN; PRIORITY JOURNAL; RESPIRATORY BURST; X CHROMOSOME LINKAGE;

ANIMALS; ANTIGENS, CD34; BONE MARROW CELLS; BONE MARROW TRANSPLANTATION; CELL LINE; DNA, COMPLEMENTARY; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; GRANULOMATOUS DISEASE, CHRONIC; HUMANS; MALE; MEMBRANE GLYCOPROTEINS; MICE; NADPH OXIDASE; RESPIRATORY BURST; RETROVIRIDAE; SUPEROXIDES; TRANSDUCTION, GENETIC; X CHROMOSOME;

EID: 0033919654     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/sj.bmt.1702365     Document Type: Article

References (21)

1. Fischer A, Segal AW, Seger R, Weening RS. The management of chronic granulomatous disease. Eur J Pediatr 1993; 152: 896-899.
2. Thrasher AJ, Keep NH, Wientjes F, Segal AW. Chronic granulomatous disease. Biochim Biophys Acta 1994; 1227: 1-24.
3. Segal AW. The NADPH oxidase and chronic granulomatous disease. Mol Med Today, 1996; 2: 129-135.
4. Curnutte JT. Chronic granulomatous disease: the solving of a clinical riddle at the molecular level. Clin Immunol Immunopathol 1993; 67: 2-15.
5. Royer-Pokora B, Kunkel LM, Monaco AP et al. Cloning the gene for an inherited human disorder-chronic granulomatous disease-on the basis of its chromosomal location. Nature 1986; 322: 32-38.
6. Roos D, de Boer M, Kuribayashi F et al. Mutations in the Xlinked and autosomal recessive forms of chronic granulomatous disease. Blood 1995; 87: 1663-1681.
7. Roos D, Weening RS, De Boer M, Meerhof LJ. Heterogeneity in chronic granulomatous disease. In: Vossen J, Griscelli C (eds). Progress in Immunodeficeincy Research and Therapy II. Elsevier: Amsterdam. 1986, pp 139-145.
8. Björgvinsdóttir H, Ding C, Pech N et al. Retroviral-mediadted gene transfer of gp91-phox into bone marrow cells rescues defect in host defense against Aspergillus fumigatous in murine X-linked chronic granulomatous disease. Blood 1997; 89: 41-48.
9. Markowitz D, Goff S, Bank A. A safe packaging line for gene transfer: separating viral gene on two different plasmids. J Virol 1988; 62: 1120-1124.
10. Zhen L, King AA, Xiao Y et al. Gene targeting of X-chromosome-linked chronic granulomatous disease locus in a human myeloid leukemia cell line and rescue by expression of recombinant gp91(phox). Proc Natl Acad Sci USA 1993; 90: 9832-9836.
11. Kume A, Dinauer MC. Retrovirus-mediated reconstitution of respiratory burst activity in X-linked chronic granulomatous disease cells. Blood 1994; 84: 3311-3316.
12. Schilz AJ, Brouns G, Knöb H et al. High efficiency gene transfer to human hematopoietic SCID-repopulating cells under serum free conditions. Blood 1998; 92: 1-10.
13. Hawley R, Lieu F, Fong A, Hawley T. Versatile retroviral vectors for potential use in gene therapy. Gene Therapy 1994; 1: 136-138.
14. Dirks W, Wirth M, Hauser H. Dicistronic transcription units for gene expression in mammalian cells. Gene 1993; 128: 247-249.
15. Mayo L, Curnutte J. Kinetic microplate assay for superoxide production by neutrophils and other phagocytic cells. Meth Enzymol 1990; 186: 567-575.
16. Becker S, Wasser S, Hauses M et al. Correction of respiratory burst activity in X-linked chronic granulomatous cells to therapeutically relevant levels after gene transfer into bone marrow CD34 cells. Hum Gene Ther 1998; 9: 1561-1570.
17. Grez M, Akgün E, Hilberg F, Ostertag W. Embryonic stem cell virus, a recombinant murine retrovirus with expression in embryonic stem cells. Proc Natl Acad Sci USA 1990; 87: 9202-9206.
18. Hawley RG, Fong AZC, Burns BF, Hawley TS. Transplantable myeloproliferative disease induced in mice by an interleukin 6 retrovirus. J Exp Med 1992; 176: 1149-1163.
19. Ding C, Kume A, Björgvinsdóttir H et al. High-level reconstitution of respiratory burst activity in a human X-linked chronic granulomatous disease (X-CGD) cell line and correction of murine X-CGD bone marrow cells by retroviral mediated gene transfer of human gp91(phox). Blood 1996; 88: 1834-1840.
20. von Kalle C, Kiem HP, Goehle S et al. Increased gene transfer into human hematopoietic progenitor cells by extended in vitro exposure to a pseudotyped retroviral vector. Blood 1994; 84: 2890-2897.
21. Hanenberg H, Xiao XL, Dillo D et al. Co-localization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction mammalian cells. Nature Med 1996; 8: 876-882.