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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Bibliografski detalji
Izdano u:Blood
Glavni autori: 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
Jezik:Inglês
Izdano: American Society of Hematology 2016
Teme:
Gene Therapy
Online pristup: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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