Fragile sites are preferential targets for integrations of MLV vectors in gene therapy

A. C. Bester, M. Schwartz, M. Schmidt, A. Garrigue, S. Hacein-Bey-Abina, M. Cavazzana-Calvo, N. Ben-Porat, C. Von Kalle, A. Fischer, B. Kerem*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

45 Scopus citations

Abstract

Following gene therapy of SCID-X1 using murine leukemia virus (MLV) derived vector, two patients developed leukemia owing to an activating vector integration near the LMO2 gene. We found that these integrations reside within FRA11E, a common fragile site known to correlate with chromosomal breakpoints in tumors. Further analysis showed that fragile sites attract a nonrandom number of MLV integrations, shedding light on its integration mechanism and risk-to-benefit ratio in gene therapy.

Original languageAmerican English
Pages (from-to)1057-1059
Number of pages3
JournalGene Therapy
Volume13
Issue number13
DOIs
StatePublished - Jul 2006

Keywords

  • Fragile sites
  • LMO2
  • Leukemia
  • Viral integration

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