原发性免疫缺陷的基因治疗。
Gene therapy for primary immunodeficiencies.
机构信息
UCL Institute of Child Health, Centre for Immunodeficiency, London WCIN 1EH, United Kingdom.
出版信息
Hum Gene Ther. 2012 Jul;23(7):668-75. doi: 10.1089/hum.2012.116.
Abstract
For over 40 years, primary immunodeficiencies (PIDs) have featured prominently in the development and refinement of human allogeneic hematopoietic stem cell transplantation. More recently, ex vivo somatic gene therapy using autologous cells has provided remarkable evidence of clinical efficacy in patients without HLA-matched stem cell donors and in whom toxicity of allogeneic procedures is likely to be high. Together with improved preclinical models, a wealth of information has accumulated that has allowed development of safer, more sophisticated technologies and protocols that are applicable to a much broader range of diseases. In this review we summarize the status of these gene therapy trials and discuss the emerging application of similar strategies to other PIDs.
摘要
四十多年来，原发性免疫缺陷病（PID）在同种异体造血干细胞移植的发展和完善中起着重要作用。最近，利用自体细胞的体外体细胞基因治疗为没有 HLA 匹配的干细胞供体的患者和异体程序毒性可能很高的患者提供了显著的临床疗效证据。结合改进的临床前模型，积累了大量信息，从而开发出更安全、更复杂的技术和方案，适用于更广泛的疾病。在这篇综述中，我们总结了这些基因治疗试验的现状，并讨论了类似策略在其他 PID 中的新应用。
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