Development of a lentiviral vector and an efficient infection method for gene therapy for p22phox-defective chronic granulomatous disease

Original Article

DOI: 10.1007/s13765-012-2098-1

Cite this article as:
Kim, Y.M., Sik, H.J., Cho, M. et al. J Korean Soc Appl Biol Chem (2012) 55: 497. doi:10.1007/s13765-012-2098-1


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.


chronic granulomatous diseaselentiviral vectorp22phox

Copyright information

© The Korean Society for Applied Biological Chemistry 2012

Authors and Affiliations

  1. 1.Department of BiochemistryJeju National UniversityJejuRepublic of Korea
  2. 2.Department of PediatricsJeju National UniversityJejuRepublic of Korea
  3. 3.Department of Urology, Jeju National University School of MedicineJeju National UniversityJejuRepublic of Korea
  4. 4.Institute of Medical ScienceJeju National UniversityJejuRepublic of Korea