The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases has become a clinical standard but is limited by the availability of suitable matched donors and potential immunologic complications. Gene therapy using autologous HSCs should avoid these limitations and thus may be safer. Progressive improvements in techniques for genetic correction of HSCs, by either vector gene addition or gene editing, are facilitating successful treatments for an increasing number of diseases. We highlight the progress, successes, and remaining challenges toward the development of HSC gene therapies and discuss lessons they provide for the development of future clinical stem cell therapies.

中文翻译：

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造血干细胞基因治疗：进展和经验教训。

使用异基因造血干细胞（HSC）来治疗遗传性血细胞疾病已成为临床标准，但受到合适的匹配供体的可用性和潜在的免疫并发症的限制。使用自体HSC的基因疗法应避免这些局限性，因此可能更安全。通过添加载体基因或编辑基因，对HSC进行遗传校正的技术的逐步改进正在促进成功治疗越来越多的疾病。我们重点介绍了HSC基因疗法发展的进展，成功和尚存的挑战，并讨论了它们为未来临床干细胞疗法的发展提供的经验教训。