A phase I/II clinical trial of β-globin gene therapy for β-thalassemia.
A phase I/II clinical trial of β-globin gene therapy for β-thalassemia.
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DOI：
10.1196/annals.1345.007
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发表时间：
2005-01-01
期刊：
影响因子：
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通讯作者：
Leboulch, P
中科院分区：
文献类型：
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作者：
Bank, A;Dorazio, R;Leboulch, P
最近，通过慢病毒载体长期纠正人类β-地中海贫血和镰状细胞性贫血小鼠模型的成功，以及有证据表明在转导的人类造血细胞中进行了高水平的基因转移和表达，导致了这种疾病的基因治疗的第一次临床试验。将使用含有P-珠蛋白基因(β(A-T87Q))的LentiGlobin载体，该基因可产生可与正常血红蛋白区分的血红蛋白(Hbβ(A-T87Q))。LentiGlobin载体是自我失活的，包含大量的β-珠蛋白基因位点控制区元素，以及染色质绝缘体和其他应该防止不良事件发生的功能。这项研究将在巴黎进行，伊莱恩·格鲁克曼担任首席研究员，菲利普·勒布尔奇担任科学总监。
Recent success in the long-term correction of mouse models of human beta-thalassemia and sickle cell anemia by lentiviral vectors and evidence of high gene transfer and expression in transduced human hematopoietic cells have led to a first clinical trial of gene therapy for the disease. A LentiGlobin vector containing a P-globin gene (beta(A-T87Q)) that produces a hemoglobin (Hb beta(A-T87Q)) that can be distinguished from normal hemoglobin will be used. The LentiGlobin vector is self-inactivating and contains large elements of the beta-globin locus control region as well as chromatin insulators and other features that should prevent untoward events. The study will be done in Paris with Eliane Gluckman as the principal investigator and Philippe Leboulch as scientific director.