Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

Aiuti, Alessandro; Cattaneo, Federica; Galimberti, Stefania; Benninghoff, Ulrike; Cassani, Barbara; Callegaro, Luciano; Scaramuzza, Samantha; Andolfi, Grazia; Mirolo, Massimiliano; Brigida, Immacolata; Tabucchi, Antonella; Carlucci, Filippo; M. Eibl, Martha; Aker, Memet; Slavin, Shimon; Al‐Mousa, Hamoud; Al Ghonaium, Abdulaziz; Ferster, Alina; Duppenthaler, Andrea; D. Notarangelo, Luigi; Wintergerst, Uwe; H. Buckley, Rebecca; Bregni, Marco; Marktel, Sarah; Grazia Valsecchi, Maria; Rossi, Paolo; Ciceri, Fabio; Miniero, R; Bordignon, Claudio; Roncarolo, Maria‐Grazia

doi:10.1056/nejmoa0805817

Public

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

Published in New England Journal of Medicine • Jan 28, 2009

Authors:

Alessandro Aiuti

Federica Cattaneo

Stefania Galimberti

Abstract

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 ...

Subject

Adenosine deaminase deficiency

Medicine

Adenosine deaminase

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