Progress toward effective gene therapy for chronic granulomatous disease

Japanese Journal of Infectious Diseases

Volumn 57, Issue 5, 2004, Pages

  Malech, Harry L ^a   Choi, Uimook ^a   Brenner, Sebastian ^a  

^a NATIONAL INSTITUTES OF HEALTH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; GLYCOPROTEIN GP 91; OXIDOREDUCTASE; PROTEIN P47; RETROVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; CHRONIC GRANULOMATOUS DISEASE; CONFERENCE PAPER; CONTROLLED STUDY; DRUG EFFICACY; EX VIVO STUDY; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUMAN CELL; IMMUNOSUPPRESSIVE TREATMENT; MOUSE; NEUTROPHIL; NONHUMAN; STEM CELL; TREATMENT OUTCOME;

ANIMALS; GENE THERAPY; GENETIC VECTORS; GRANULOMATOUS DISEASE, CHRONIC; HUMANS; MEMBRANE GLYCOPROTEINS; NADPH OXIDASE; PHOSPHOPROTEINS;

EID: 9144271097     PISSN: 13446304     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Conference Paper

References (7)

1. Malech, H. L. and Nauseef, W. M. (1997): Primary inherited defects in neutrophil function: etiology and treatment. Semin. Hematol., 34, 279-290.
2. Malech, H. L., Maples, P. B., Whiting-Theobald, N., Linton, G. F., Sekhsaria, S., Vowells, S. J., Li, F., Miller, J. A., DeCarlo, E., Holland, S, M., Leitman, S. F., Carter, C. S., Butz, R. E., Read, E. J. Fleisher, T. A., Schneiderman, R. D., Van Epps, D. E., Spratt, S. K., Maack, C. A., Rokovich, J. A., Cohen, L. K. and Gallin, J. I. (1997): Prolonged production of NADPH oxidase-corrected granulocytes following gene therapy of chronic granulomatous disease. Proc. Natl. Acad. Sci. USA, 94, 12133-12138.
3. Malech, H. L. Bauer, T. R. Jr. and Hickstein, D. D. (1997): Prospects for gene therapy of neutrophil defects. Semin. Hematol., 34, 355-361.
4. Malech, H. L. (2000): Use of serum-free medium with fibronectin fragment enhance transduction in a system of gas permeable plastic containers to achieve high levels of retrovirus transduction at clinical scale. Stem Cells, 18, 155-156.
5. Malech, H. L. (1999): Progress in gene therapy for chronic granulomatous disease. J. Infect. Dis., 179 (Suppl. 2), S318-S325.
6. + cells. Blood, 102, 2789-2797.
7. Choi, U. and Malech, H. L. (2002): Efficient selective enrichment of hematopoietic stem cells transduced with MFGS retrovirus encoding benzyl guanine resistant methylguanine methyltransferase linked to therapeutic X-linked chronic granulomatous disease gene. Blood, 100, 1696. Part 1. Nov. 16 (abstract).