Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID
Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID
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DOI：
10.1007/s12026-009-8107-8
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发表时间：
2009-07-01
影响因子：
4.4
通讯作者：
Roncarolo, Maria-Grazia
中科院分区：
文献类型：
--
作者：
Aiuti, Alessandro;Brigida, Immacolata;Roncarolo, Maria-Grazia
基因治疗对于许多类型的免疫系统遗传性疾病是一种非常有吸引力的策略。腺苷脱氨酶（ADA）缺乏-严重联合免疫缺陷（SCID）已经成为基于使用逆转录病毒载体工程化的造血干/祖细胞的几个临床试验的目标。低强度预处理方案的引入是实现造血干细胞稳定植入和ADA表达细胞达到治疗水平的关键因素。近年来的研究表明，ADA-SCID的基因治疗具有良好的安全性，并能有效地恢复正常的嘌呤代谢和免疫功能。干细胞基因治疗结合适当的预处理方案可能会扩展到造血系统的其他遗传性疾病。
Gene therapy is a highly attractive strategy for many types of inherited disorders of the immune system. Adenosine deaminase (ADA) deficient-severe combined immunodeficiency (SCID) has been the target of several clinical trials based on the use of hematopoietic stem/progenitor cells engineered with retroviral vectors. The introduction of a low intensity conditioning regimen has been a crucial factor in achieving stable engrafment of hematopoietic stem cells and therapeutic levels of ADA-expressing cells. Recent studies have demonstrated that gene therapy for ADA-SCID has favorable safety profile and is effective in restoring normal purine metabolism and immune functions. Stem cell gene therapy combined with appropriate conditioning regimens might be extended to other genetic disorders of the hematopoietic system.