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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Vydáno v:Blood
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Hematology 2016
Témata:
Gene Therapy
On-line přístup: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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