• Citer

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

par Aiuti, Alessandro;Cattaneo, Federica;Galimberti, Stefania;Benninghoff, Ulrike;Cassani, Barbara;Callegaro, Luciano;Scaramuzza, Samantha;Andolfi, Grazia;Mirolo, Massimiliano;Brigida, Immacolata;Tabucchi, Antonella;Carlucci, Filippo;Eibl, Martha;Aker, Memet;Slavin, Shimon;Al-Mousa, Hamoud;Al Ghonaium, Abdulaziz;Ferster, Alina ;Duppenthaler, Andrea;Notarangelo, Luigi;Wintergerst, Uwe;Buckley, Rebecca H;Bregni, Marco;Marktel, Sarah;Valsecchi, Maria Grazia;Rossi, Pier Luca;Ciceri, Fabio;Miniero, Roberto;Bordignon, Claudio;Roncarolo, Maria-Grazia
Référence The New England journal of medicine, 360, 5, page (447-458)
Publication Publié, 2009-01

Article révisé par les pairs

• Détails
• Contenu

Résumé :

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.