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基因疗法治疗先天性免疫缺陷:过去的进展,目前的状况和未来的方向

Robert Torrance1, Kate Orf1,2, Thomas A Fox1,2

  • 1UCL Institute of Immunity and Transplantation, UCL, London NW3 2PP, United Kingdom.

Rare disease and orphan drugs journal
|February 16, 2026
PubMed
概括

基因疗法 (GT) 提供了一种独立于捐赠者的安全和有效的治疗方法,用于治疗先天性免疫错误 (IEI),纠正遗传缺陷. 本综述强调了四十年的进展,当前的基因编辑技术,以及IEI治疗的未来方向.

关键词:
基因治疗是一种基因疗法.免疫的先天错误 免疫的先天错误原发性免疫缺陷是一个主要的疾病.

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