Ontology highlight
ABSTRACT:
SUBMITTER: Cicalese MP
PROVIDER: S-EPMC5910668 | biostudies-literature | 2018 Mar
REPOSITORIES: biostudies-literature
Cicalese Maria Pia MP Ferrua Francesca F Castagnaro Laura L Rolfe Katie K De Boever Erika E Reinhardt Rickey R RR Appleby Jonathan J Roncarolo Maria Grazia MG Aiuti Alessandro A
Molecular therapy : the journal of the American Society of Gene Therapy 20180104 3
Loss of adenosine deaminase activity leads to severe combined immunodeficiency (ADA-SCID); production and function of T, B, and natural killer (NK) cells are impaired. Gene therapy (GT) with an autologous CD34<sup>+</sup>-enriched cell fraction that contains CD34<sup>+</sup> cells transduced with a retroviral vector encoding the human ADA cDNA sequence leads to immune reconstitution in most patients. Here, we report short- and medium-term safety analyses from 18 patients enrolled as part of sing ...[more]