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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, pages 497–506 (2012)Cite this article

An Erratum to this article was published on 31 August 2012

Abstract

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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Authors and Affiliations

  1. Department of Biochemistry, Jeju National University, Jeju, 690-756, Republic of Korea

    Young Mee Kim & Moonjae Cho

  2. Department of Pediatrics, Jeju National University, Jeju, 690-756, Republic of Korea

    Kyung Sue Shin

  3. Department of Urology, Jeju National University School of Medicine, Jeju National University, Jeju, 690-756, Republic of Korea

    Huh Jung Sik

  4. Institute of Medical Science, Jeju National University, Jeju, 690-756, Republic of Korea

    Moonjae Cho & Kyung Sue Shin

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  1. Young Mee Kim
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  2. Huh Jung Sik
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  3. Moonjae Cho
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Correspondence to Moonjae Cho or Kyung Sue Shin.

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Kim, Y.M., Sik, H.J., Cho, M. et al. Development of a lentiviral vector and an efficient infection method for gene therapy for p22phox-defective chronic granulomatous disease. J Korean Soc Appl Biol Chem 55, 497–506 (2012). https://doi.org/10.1007/s13765-012-2098-1

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