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

  title={Gene Therapy in a Patient with Sickle Cell Disease: Brief Report},
  author={Jean-Antoine Ribeil and Salima Hacein-Bey-Abina and Emmanuel Payen and Alessandra Magnani and Michaela Semeraro and Elisa Magrin and Laure Caccavelli and B{\'e}n{\'e}dicte Neven and Philippe Bourget and Wassim El Nemer and Pablo Bartolucci and Leslie Weber and Herv{\'e} Puy and Jean François Meritet and David Grevent and Yves Beuzard and Stany Chrétien and Thibaud Lef{\`e}bvre and R W Ross and Olivier N{\`e}gre and Gabor Istvan Veres and Laura Sandler and Sandeep Soni and Mariane de Montalembert and St{\'e}phane Blanche and Philippe Leboulch and Marina Cavazzana},
  journal={The New England Journal of Medicine},
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… 

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