Journal
GENE THERAPY
Volume 13, Issue 13, Pages 1057-1059Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.gt.3302752
Keywords
fragile sites; viral integration; LMO2; leukemia
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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.
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