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Autologous bone marrow CD34+ cells were transduced ex vivo with the MND (myeloproliferative sarcoma virus, negative control region deleted, dl587rev primer binding site)\u2013ADA gammaretroviral vector (gRV) and infused following busulfan reduced-intensity conditioning. These patients were monitored in a long-term follow-up protocol over 8 to 11 years. Nine of 10 patients have sufficient immune reconstitution to protect against serious infections and have not needed to resume ERT or proceed to secondary allogeneic HSCT. ERT was restarted 6 months after GT in the oldest patient who had no evidence of benefit from GT. Four of 9 evaluable patients with the highest gene marking and B-cell numbers remain off immunoglobulin replacement therapy and responded to vaccines. There were broad ranges of responses in normalization of ADA enzyme activity and adenine metabolites in blood cells and levels of cellular and humoral immune reconstitution. Outcomes were generally better in younger patients and those receiving higher doses of gene-marked CD34+ cells. No patient experienced a leukoproliferative event after GT, despite persisting prominent clones with vector integrations adjacent to proto-oncogenes. These long-term findings demonstrate enduring efficacy of GT for ADA SCID but also highlight risks of genotoxicity with gRVs. This trial was registered at www.clinicaltrials.gov as #NCT00794508.<\/jats:p>","DOI":"10.1182\/blood.2020010260","type":"journal-article","created":{"date-parts":[[2021,5,11]],"date-time":"2021-05-11T16:41:10Z","timestamp":1620751270000},"page":"1304-1316","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":49,"title":["Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency"],"prefix":"10.1182","volume":"138","author":[{"given":"Bryanna","family":"Reinhardt","sequence":"first","affiliation":[{"name":"Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Omar","family":"Habib","sequence":"additional","affiliation":[{"name":"Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of 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