{"id":13400,"date":"2019-03-13T17:37:38","date_gmt":"2019-03-13T16:37:38","guid":{"rendered":"http:\/\/cyber.pixilie.fr\/?post_type=publications&#038;p=13400"},"modified":"2025-09-11T16:48:04","modified_gmt":"2025-09-11T14:48:04","slug":"gene-therapy-in-a-patient-with-sickle-cell-disease","status":"publish","type":"publication","link":"https:\/\/cri1149.fr\/en\/publication\/gene-therapy-in-a-patient-with-sickle-cell-disease\/","title":{"rendered":"Gene Therapy in a Patient with Sickle Cell Disease."},"content":{"rendered":"","protected":false},"featured_media":0,"template":"","meta":{"_acf_changed":false,"_EventAllDay":false,"_EventTimezone":"","_EventStartDate":"","_EventEndDate":"","_EventStartDateUTC":"","_EventEndDateUTC":"","_EventShowMap":false,"_EventShowMapLink":false,"_EventURL":"","_EventCost":"","_EventCostDescription":"","_EventCurrencySymbol":"","_EventCurrencyCode":"","_EventCurrencyPosition":"","_EventDateTimeSeparator":"","_EventTimeRangeSeparator":"","_EventOrganizerID":[],"_EventVenueID":[],"_OrganizerEmail":"","_OrganizerPhone":"","_OrganizerWebsite":"","_VenueAddress":"","_VenueCity":"","_VenueCountry":"","_VenueProvince":"","_VenueState":"","_VenueZip":"","_VenuePhone":"","_VenueURL":"","_VenueStateProvince":"","_VenueLat":"","_VenueLng":"","_VenueShowMap":false,"_VenueShowMapLink":false},"category_publication":[],"class_list":["post-13400","publication","type-publication","status-publish","hentry"],"acf":{"numero_de_publication":"N Engl J Med. ","date_de_publication":"20170302","numero_doi":"","equipe":[124],"auteurs-liste":[{"texte_libre":true,"auteur-lien":null,"auteur-text":"Ribeil JA"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Hacein-Bey-Abina S"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Payen E"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Magnani A"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Semeraro M"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Magrin E"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Caccavelli L"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Neven B"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Bourget P"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"El Nemer W"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Bartolucci P"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Weber L"},{"texte_libre":false,"auteur-lien":13142,"auteur-text":""},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Meritet JF"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Grevent D"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Beuzard Y"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Chr\u00e9tien S"},{"texte_libre":false,"auteur-lien":13104,"auteur-text":""},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Ross RW"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Negre O"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Veres G"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Sandler L"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Soni S"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"De Montalembert M"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Blanche S"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Leboulch P"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"Cavazzana M"}],"auteurs-manuel":"","liens_externes":null,"liens":[{"lien":"https:\/\/www.ncbi.nlm.nih.gov\/pubmed\/28249145"}],"paragraphe":"<h3 style=\"text-align: justify;\">Abstract<\/h3>\r\n<div class=\"\">\r\n<p style=\"text-align: justify;\">Sickle cell disease results from a homozygous missense mutation in the \u03b2-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achieving effective, persistent inhibition of polymerization of hemoglobin S. We describe our first patient treated with lentiviral vector-mediated addition of an antisickling \u03b2-globin gene into autologous hematopoietic stem cells. Adverse events were consistent with busulfan conditioning. Fifteen months after treatment, the level of therapeutic antisickling \u03b2-globin remained high (approximately 50% of \u03b2-like-globin chains) without recurrence of sickle crises and with correction of the biologic hallmarks of the disease. (Funded by Bluebird Bio and others; HGB-205 ClinicalTrials.gov number,\u00a0<a title=\"See in ClinicalTrials.gov\" href=\"http:\/\/clinicaltrials.gov\/show\/NCT02151526\">NCT02151526<\/a>\u00a0.).<\/p>\r\n<\/div>","paragraphe_en":"","documents":null},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Gene Therapy in a Patient with Sickle Cell Disease. - CRI<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/cri1149.fr\/publication\/gene-therapy-in-a-patient-with-sickle-cell-disease\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Gene Therapy in a Patient with Sickle Cell Disease. - CRI\" \/>\n<meta property=\"og:url\" content=\"https:\/\/cri1149.fr\/publication\/gene-therapy-in-a-patient-with-sickle-cell-disease\/\" \/>\n<meta property=\"og:site_name\" content=\"CRI\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/CRI-Centre-de-recherche-sur-linflammation-100116118884153\" \/>\n<meta property=\"article:modified_time\" content=\"2025-09-11T14:48:04+00:00\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/cri1149.fr\\\/publication\\\/gene-therapy-in-a-patient-with-sickle-cell-disease\\\/\",\"url\":\"https:\\\/\\\/cri1149.fr\\\/publication\\\/gene-therapy-in-a-patient-with-sickle-cell-disease\\\/\",\"name\":\"Gene Therapy in a Patient with Sickle Cell Disease. - 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