The lung has long been a target for gene therapy, yet efficient delivery and phenotypic disease correction has remained challenging. Although there have been significant advancements in gene therapies of other organs, including the development of several ex vivo therapies, in vivo therapeutics of the lung have been slower to transition to the clinic. Within the past few years, the field has witnessed an explosion in the development of new gene addition and gene editing strategies for the treatment of monogenic disorders. In this review, we will summarize current developments in gene therapy for cystic fibrosis, alpha-1 antitrypsin deficiency, and surfactant protein deficiencies. We will explore the different gene addition and gene editing strategies under investigation and review the challenges of delivery to the lung.

中文翻译：

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肺基因治疗的新方向。

肺部长期以来一直是基因治疗的目标，但有效的递送和表型疾病的纠正仍然具有挑战性。尽管其他器官的基因疗法取得了重大进展，包括开发了几种体外疗法，但在体内肺部疗法向临床过渡的速度较慢。在过去的几年中，该领域见证了用于治疗单基因疾病的新基因添加和基因编辑策略的发展。在这篇综述中，我们将总结囊性纤维化、α1 抗胰蛋白酶缺乏症和表面活性蛋白缺乏症基因治疗的最新进展。我们将探索不同的基因添加和基因编辑策略，并回顾递送到肺部的挑战。