Development of a lentiviral vector and an efficient infection method for gene therapy for p22phox-defective chronic granulomatous disease
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Chronic granulomatous disease (CGD) is caused by impaired antimicrobial activity in phagocytes due to the absence or malfunction of the respiratory burst NADPH oxidase. In a previous study, we found that 12 patients from 10 unrelated families on Jeju Island had an identical homozygous single-base C-to-T substitution in exon 1 (c.7C > T) of CYBA, which encodes p22phox. Autosomal recessive p22phox-defective CGD carrierderived white blood cells were efficiently transduced by the elongation factor 1-alpha lentivirus constructs, as up to 90% of cells were green fluorescent protein (eGFP)-positive at 3 days post-transduction. pLL3.7-driven eGFP expression was stable for at least 4 weeks after transduction and persisted after CGD carrierderived cells were immortalized by human telomerase reverse transcriptase (hTERT) and B lymphoma Mo-MLV insertion region 1 (Bmi-1). Upon macrophage-like differentiation of the transduced HL-60 cells by dimethyl sulfoxide, up to 28% of the cells had higher mean levels of superoxide production than undifferentiated cells, and lentivirus efficiently transduced cells and induced the expression of genes for extended periods.
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- Development of a lentiviral vector and an efficient infection method for gene therapy for p22phox-defective chronic granulomatous disease
Journal of the Korean Society for Applied Biological Chemistry
Volume 55, Issue 4 , pp 497-506
- Cover Date
- Print ISSN
- Online ISSN
- The Korean Society for Applied Biological Chemistry
- Additional Links
- chronic granulomatous disease
- lentiviral vector
- Author Affiliations
- 1. Department of Biochemistry, Jeju National University, Jeju, 690-756, Republic of Korea
- 3. Department of Urology, Jeju National University School of Medicine, Jeju National University, Jeju, 690-756, Republic of Korea
- 4. Institute of Medical Science, Jeju National University, Jeju, 690-756, Republic of Korea
- 2. Department of Pediatrics, Jeju National University, Jeju, 690-756, Republic of Korea