Cavazzana-Calvo Marina, Fischer Alain
INSERM U768 and Université Paris Descartes, Paris, France.
J Clin Invest. 2007 Jun;117(6):1456-65. doi: 10.1172/JCI30953.
Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marrow transplantation can circumvent the severe immunological complications that occur when a related HLA-mismatched donor is used and thus represents an attractive alternative. In this review, we summarize the advantages and limitations associated with the use of gene therapy to cure SCID. Insertional mutagenesis and technological improvements aimed at increasing the safety of this strategy are also discussed.
造血系统的遗传性和获得性疾病可通过异基因造血干细胞移植治愈。当有人类白细胞抗原（HLA）匹配的同胞供体时，这种治疗策略非常成功，但如果没有，则几乎没有治疗选择。基因修饰的自体骨髓移植可以规避使用相关HLA不匹配供体时出现的严重免疫并发症，因此是一种有吸引力的替代方法。在本综述中，我们总结了与使用基因疗法治疗重症联合免疫缺陷病（SCID）相关的优缺点。还讨论了插入诱变以及旨在提高该策略安全性的技术改进。
