Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial.

@article{Wang2010DynamicsOG,
  title={Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial.},
  author={Gary P Wang and Charles C. Berry and Nirav Malani and Philippe Leboulch and Alain Fischer and Salima Hacein-Bey-Abina and Marina Cavazzana-Calvo and Frederic D. Bushman},
  journal={Blood},
  year={2010},
  volume={115 22},
  pages={
          4356-66
        }
}
X-linked severe-combined immunodeficiency (SCID-X1) has been treated by therapeutic gene transfer using gammaretroviral vectors, but insertional activation of proto-oncogenes contributed to leukemia in some patients. Here we report a longitudinal study of gene-corrected progenitor cell populations from 8 patients using 454 pyrosequencing to map vector integration sites, and extensive resampling to allow quantification of clonal abundance. The number of transduced cells infused into patients… CONTINUE READING
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