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

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 <25%...

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Bibliografiske detaljer
Udgivet i:Blood
Main Authors: Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Pajno, Roberta, Barzaghi, Federica, Giannelli, Stefania, Dionisio, Francesca, Brigida, Immacolata, Bonopane, Marco, Casiraghi, Miriam, Tabucchi, Antonella, Carlucci, Filippo, Grunebaum, Eyal, Adeli, Mehdi, Bredius, Robbert G., Puck, Jennifer M., Stepensky, Polina, Tezcan, Ilhan, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Ciceri, Fabio, Roncarolo, Maria Grazia, Aiuti, Alessandro
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Hematology 2016
Fag:
Gene Therapy
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5325048/
https://ncbi.nlm.nih.gov/pubmed/27129325
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2016-01-688226
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