Over 3 decades, gene therapy has advanced from a logical idea to becoming a clinical reality for several of the most severe primary immune deficiencies, as well as other inherited disorders. The first gene therapy medicines have been licensed for marketing and several more are advancing toward that goal to make them widely available, beyond clinical trials. Although common platforms of cells, vectors, or editing reagents are used for these disorders, each individual genetic cause of an immune deficiency requires its own vector or editing tools and a package of preclinical data on efficacy and safety to initiate clinical trials. One-by-one, gene therapy for primary immune deficiencies is being brought to the clinic and hopefully will provide safe and effective therapies.

在过去的30多年中，基因治疗已从一种合理的想法发展为针对一些最严重的原发性免疫缺陷以及其他遗传性疾病的临床现实。第一种基因疗法药物已获得许可销售，并且有更多的这种药物正在朝着这个目标迈进，以使其广泛应用于临床试验之外。尽管细胞，载体或编辑试剂的通用平台可用于这些疾病，但免疫缺陷的每个单独的遗传原因都需要其自己的载体或编辑工具以及一整套有关功效和安全性的临床前数据，以启动临床试验。用于原发性免疫缺陷的一对一基因疗法已被带到​​临床，有望提供安全有效的疗法。