Citation:
Bester AC., M., Schwartz , M., Schmidt , A., Garrigue , S., Hacein-Bey-Abina , M., Cavazzana-Calvo , N., Ben-Porat , CV., Kalle , A., Fischer , and B., Kerem . 2006.
“Fragile Sites Are Preferential Targets For Integrations Of Mlv Vectors In Gene Therapy”. Gene Therapy, 13, 13, Pp. 1057-1059.
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.
