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Here, we present comprehensive, long-term follow-up results (median follow-up, 7.6\u2009years) (phase I\/II trial no.\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02333760\" ext-link-type=\"uri\">NCT02333760<\/jats:ext-link>\n                    ) for eight patients with WAS having undergone phase I\/II lentiviral vector-based gene therapy trials (nos.\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT01347346\" ext-link-type=\"uri\">NCT01347346<\/jats:ext-link>\n                    and\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT01347242\" ext-link-type=\"uri\">NCT01347242<\/jats:ext-link>\n                    ), with a focus on thrombocytopenia and autoimmunity. Primary outcomes of the long-term study were to establish clinical and biological safety, efficacy and tolerability by evaluating the incidence and type of serious adverse events and clinical status and biological parameters including lentiviral genomic integration sites in different cell subpopulations from 3\u2009years to 15\u2009years after gene therapy. Secondary outcomes included monitoring the need for additional treatment and T\u2009cell repertoire diversity. An interim analysis shows that the study meets the primary outcome criteria tested given that the gene-corrected cells engrafted stably, and no serious treatment-associated adverse events occurred. Overall, severe infections and eczema resolved. Autoimmune disorders and bleeding episodes were significantly less frequent, despite only partial correction of the platelet compartment. The results suggest that lentiviral gene therapy provides sustained clinical benefits for patients with WAS.\n                  <\/jats:p>","DOI":"10.1038\/s41591-021-01641-x","type":"journal-article","created":{"date-parts":[[2022,1,24]],"date-time":"2022-01-24T12:02:39Z","timestamp":1643025759000},"page":"71-80","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":127,"title":["Long-term safety and efficacy of lentiviral hematopoietic stem\/progenitor cell gene therapy for Wiskott\u2013Aldrich syndrome"],"prefix":"10.1038","volume":"28","author":[{"given":"A.","family":"Magnani","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M.","family":"Semeraro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6603-2687","authenticated-orcid":false,"given":"F.","family":"Adam","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C.","family":"Booth","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"L.","family":"Dupr\u00e9","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4834-1130","authenticated-orcid":false,"given":"E. 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