Efficacy of gene therapy for X-linked severe combined immunodeficiency.

  title={Efficacy of gene therapy for X-linked severe combined immunodeficiency.},
  author={Salima Hacein-Bey-Abina and Julia Hauer and Annick Lim and Capucine Picard and Gary P. Wang and Charles C. Berry and Chantal Martinache and Fr{\'e}d{\'e}ric Rieux-Laucat and Sylvain Latour and Bernd H. Belohradsky and Lily Leiva and Ricardo U. Sorensen and Marianne Debr{\'e} and Jean Casanova and St{\'e}phane Blanche and Anne H Durandy and Frederic D. Bushman and Alain Fischer and Marina Cavazzana-Calvo},
  journal={The New England journal of medicine},
  volume={363 4},
BACKGROUND The outcomes of gene therapy to correct congenital immunodeficiencies are unknown. We reviewed long-term outcomes after gene therapy in nine patients with X-linked severe combined immunodeficiency (SCID-X1), which is characterized by the absence of the cytokine receptor common gamma chain. METHODS The nine patients, who lacked an HLA-identical donor, underwent ex vivo retrovirus-mediated transfer of gamma chain to autologous CD34+ bone marrow cells between 1999 and 2002. We… CONTINUE READING