Gene Therapy in a Patient with Sickle Cell Disease: Brief Report

@article{Ribeil2017GeneTI,
  title={Gene Therapy in a Patient with Sickle Cell Disease: Brief Report},
  author={J. Ribeil and S. Hacein-Bey-Abina and E. Payen and A. Magnani and M. Semeraro and Elisa Magrin and L. Caccavelli and B. Neven and P. Bourget and W. El Nemer and P. Bartolucci and Leslie Weber and H. Puy and J. Meritet and D. Gr{\'e}vent and Y. Beuzard and S. Chrétien and T. Lefebvre and R. W. Ross and O. Negre and G. Veres and L. Sandler and Sandeep Soni and M. de Montalembert and S. Blanche and P. Leboulch and M. Cavazzana},
  journal={The New England Journal of Medicine},
  year={2017},
  volume={376},
  pages={848–855}
}
Abstract Sickle cell disease results from a homozygous missense mutation in the β‐globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achieving effective, persistent inhibition of polymerization of hemoglobin S. We describe our first patient treated with lentiviral vector–mediated addition of an antisickling β‐globin gene into autologous hematopoietic stem cells. Adverse… Expand
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Gene therapy using autologous HSC transduced with LentiGlobin BB305 lentiviral vector is a promising approach for the treatment of patients with hemoglobinopathies. Expand
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