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After 2 years of treatment, long-term survival of T and B lymphocytes, marrow cells, and granulocytes expressing the transferred ADA gene was demonstrated and resulted in normalization of the immune repertoire and restoration of cellular and humoral immunity. After discontinuation of treatment, T lymphocytes, derived from transduced peripheral blood lymphocytes, were progressively replaced by marrow-derived T cells in both patients. These results indicate successful gene transfer into long-lasting progenitor cells, producing a functional multilineage progeny.<\/jats:p>","DOI":"10.1126\/science.270.5235.470","type":"journal-article","created":{"date-parts":[[2006,10,27]],"date-time":"2006-10-27T18:19:42Z","timestamp":1161973182000},"page":"470-475","source":"Crossref","is-referenced-by-count":655,"title":["Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA\n            <sup>\u2212<\/sup>\n            Immunodeficient Patients"],"prefix":"10.1126","volume":"270","author":[{"given":"Claudio","family":"Bordignon","sequence":"first","affiliation":[{"name":"C. Bordignon, N. Nobili, G. Ferrari, D. Maggioni, C. Rossi, P. Servida, F. Mavilio, Telethon Gene Therapy Program for Genetic Diseases, DIBIT, Istituto Scientifico H. S. Raffaele, Milan, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luigi D.","family":"Notarangelo","sequence":"additional","affiliation":[{"name":"L. D. 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