Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Maria Pia Cicalese, Francesca Ferrua, Laura Castagnaro, Roberta Pajno, Federica Barzaghi, Stefania Giannelli, Francesca Dionisio, Immacolata Brigida, Marco Bonopane, Miriam Casiraghi, Antonella Tabucchi, Filippo Carlucci, Eyal Grunebaum, Mehdi Adeli, Robbert G. Bredius, Jennifer M. Puck, Polina Stepensky, Ilhan Tezcan, Katie Rolfe, Erika De BoeverRickey R. Reinhardt, Jonathan Appleby, Fabio Ciceri, Maria Grazia Roncarolo, Alessandro Aiuti

Research output: Contribution to journalArticle

Abstract

Adenosine deaminase (ADA) deficiency is a rare, autosomal-recessive systemic metabolic disease characterized by severe combined immunodeficiency (SCID). The treatment of choice for ADA-deficient SCID (ADA-SCID) is hematopoietic stem cell transplant from an HLA-matched sibling donor, although

Original languageEnglish
Pages (from-to)45-54
Number of pages10
JournalBlood
Volume128
Issue number1
DOIs
Publication statusPublished - Jul 7 2016

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ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

Cicalese, M. P., Ferrua, F., Castagnaro, L., Pajno, R., Barzaghi, F., Giannelli, S., Dionisio, F., Brigida, I., Bonopane, M., Casiraghi, M., Tabucchi, A., Carlucci, F., Grunebaum, E., Adeli, M., Bredius, R. G., Puck, J. M., Stepensky, P., Tezcan, I., Rolfe, K., ... Aiuti, A. (2016). Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency. Blood, 128(1), 45-54. https://doi.org/10.1182/blood-2016-01-688226