Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

  title={Gene therapy for immunodeficiency due to adenosine deaminase deficiency.},
  author={Alessandro Aiuti and Federica Cattaneo and Stefania Galimberti and Ulrike Benninghoff and Barbara Cassani and Luciano Callegaro and Samantha Scaramuzza and Grazia Andolfi and Massimiliano Mirolo and Immacolata Brigida and Antonella Tabucchi and Filippo Carlucci and Martha Marianne Eibl and Memet Aker and Shimon Slavin and Hamoud Al‐Mousa and Abdulaziz Al Ghonaium and Alina Ferster and Andrea Duppenthaler and Luigi Daniele Notarangelo and Uwe Wintergerst and Rebecca H Buckley and Marco Bregni and Sarah Marktel and Maria Grazia Valsecchi and Paolo Rossi and Fabio Ciceri and Roberto Miniero and Claudio Bordignon and Maria Grazia Roncarolo},
  journal={The New England journal of medicine},
  volume={360 5},
BACKGROUND We investigated the long-term outcome of gene therapy for severe combined immunodeficiency (SCID) due to the lack of adenosine deaminase (ADA), a fatal disorder of purine metabolism and immunodeficiency. METHODS We infused autologous CD34+ bone marrow cells transduced with a retroviral vector containing the ADA gene into 10 children with SCID due to ADA deficiency who lacked an HLA-identical sibling donor, after nonmyeloablative conditioning with busulfan. Enzyme-replacement… 

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