{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T22:39:19Z","timestamp":1792103959465,"version":"4.4.0"},"reference-count":26,"publisher":"American Association for the Advancement of Science (AAAS)","issue":"5954","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Science"],"published-print":{"date-parts":[[2009,11,6]]},"abstract":"<jats:title>Slowing Brain Disease with Gene Therapy<\/jats:title>\n                  <jats:p>\n                    X-linked adrenoleukodystrophy (ALD), the hereditary brain demyelinating disorder that was featured in the movie \u201c\n                    <jats:italic>Lorenzo's Oil<\/jats:italic>\n                    ,\u201d is typically treated by transplantation of bone marrow from matched donors. This treatment slows progression of the disease by introducing cells that differentiate into myelin-producing cells.\n                    <jats:bold>\n                      Cartier\n                      <jats:italic>et al.<\/jats:italic>\n                    <\/jats:bold>\n                    (p.\n                    <jats:related-article xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"doi\" page=\"818\" related-article-type=\"in-this-issue\" vol=\"326\" xlink:href=\"10.1126\/science.1171242\">818<\/jats:related-article>\n                    ; see Perspective by\n                    <jats:bold>\n                      <jats:related-article xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"doi\" issue=\"5954\" page=\"805\" related-article-type=\"in-this-issue\" vol=\"326\" xlink:href=\"10.1126\/science.1181937\">Naldini<\/jats:related-article>\n                    <\/jats:bold>\n                    ) tested an alternative gene therapy\u2013based approach in two young patients without matched donors. A lentiviral vector was used to introduce a wild-type copy of the ALD gene into the patients' hematopoietic stem cells ex vivo. The modified cells were then infused back into the patients. Expression of the transferred gene was still detectable in the patients' blood cells 2 years later, and both patients showed neurological improvement and a delay in disease progression comparable to that seen with bone marrow transplants.\n                  <\/jats:p>","DOI":"10.1126\/science.1171242","type":"journal-article","created":{"date-parts":[[2009,11,5]],"date-time":"2009-11-05T19:42:59Z","timestamp":1257450179000},"page":"818-823","source":"Crossref","is-referenced-by-count":1377,"title":["Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy"],"prefix":"10.1126","volume":"326","author":[{"given":"Nathalie","family":"Cartier","sequence":"first","affiliation":[{"name":"INSERM UMR745, University Paris-Descartes, 75279 Paris, France."},{"name":"Department of Pediatric Endocrinology and Neurology, H\u00f4pital Saint-Vincent de Paul, Assistance Publique\u2013H\u00f4pitaux de Paris, 82 Avenue Denfert-Rochereau, 75674 Paris, France."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salima","family":"Hacein-Bey-Abina","sequence":"additional","affiliation":[{"name":"Department of Biotherapy, H\u00f4pital Necker-Enfants Malades, 75743 Paris, France."},{"name":"INSERM UMR768, University Paris-Descartes, 75743 Paris, France."},{"name":"Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Assistance Publique\u2013H\u00f4pitaux de Paris (Inserm), 75743 Paris, France."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cynthia C.","family":"Bartholomae","sequence":"additional","affiliation":[{"name":"National Center for Tumor Diseases and German Cancer Research Center (Deutsches Krebsforschungszentrum), 69120 Heidelberg, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabor","family":"Veres","sequence":"additional","affiliation":[{"name":"117 Southwest 72nd Place, Gainesville, FL 32608, 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