The promise of genetic therapies has turned into reality in recent years, with new first-line treatments for fatal diseases now available to patients. The development and testing of genetic therapies for respiratory diseases such as cystic fibrosis (CF) has also progressed. The addition of gene editing to the genetic agent toolbox, and its early success in other organ systems, suggests we will see rapid expansion of gene correction options for CF in the future. Although substantial progress has been made in creating techniques and genetic agents that can be highly effective for CF correction in vitro, physiologically relevant functional in vivo changes have been largely prevented by poor delivery efficiency within the lungs. Somewhat hidden from view, however, is the absence of reliable, accurate, detailed, and noninvasive outcome measures that can detect subtle disease and treatment effects in the lungs of humans or animal models. The ability to measure the fundamental function of the lung—ventilation, the effective transport of air throughout the lung—has been constrained by the available measurement technologies. Without sensitive measurement methods, it is difficult to quantify the effectiveness of genetic therapies for CF. The mainstays of lung health assessment are spirometry, which cannot provide adequate disease localization and is not sensitive enough to detect small early changes in disease; and computed tomography, which provides structural rather than functional information. Magnetic resonance imaging using hyperpolarized gases is increasingly useful for lung ventilation assessment, and it removes the radiation risk that accompanies X-ray methods. A new lung imaging technique, X-ray velocimetry, can now offer highly detailed regional lung ventilation information well suited to the diagnosis, treatment, and monitoring needs of CF lung disease, particularly after the application of genetic therapies. In this review, we discuss the options now available for imaging-based lung function measurement in the generation and use of genetic and other therapies for treating CF lung disease.

中文翻译：

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囊性纤维化的气道基因治疗会改善肺功能吗？新的成像技术可以帮助我们找到答案。

近年来，基因疗法的前景已成为现实，患者现在可以使用针对致命疾病的新一线治疗方法。囊性纤维化 (CF) 等呼吸系统疾病的基因疗法的开发和测试也取得了进展。将基因编辑添加到遗传代理工具箱中，以及它在其他器官系统中的早期成功，表明我们将在未来看到 CF 基因校正选项的快速扩展。尽管在创造可在体外CF 校正方面非常有效的技术和遗传因子方面取得了实质性进展，但在体内具有生理相关的功能。肺内传递效率低下，在很大程度上阻止了变化。然而，在某种程度上隐藏起来的是，缺乏可靠、准确、详细和无创的结果测量方法，可以检测人类或动物模型肺部的细微疾病和治疗效果。测量肺的基本功能——通气、空气在整个肺部的有效输送——的能力受到现有测量技术的限制。如果没有灵敏的测量方法，就很难量化 CF 基因疗法的有效性。肺健康评估的主要内容是肺活量测定法，它不能提供足够的疾病定位，也不够灵敏，无法检测到疾病的早期微小变化；和计算机断层扫描，它提供结构而不是功能信息。使用超极化气体的磁共振成像对肺通气评估越来越有用，它消除了伴随 X 射线方法的辐射风险。一种新的肺部成像技术 X 射线测速仪现在可以提供非常详细的区域肺通气信息，非常适合 CF 肺病的诊断、治疗和监测需求，特别是在应用基因疗法之后。在这篇综述中，我们讨论了现在可用于基于成像的肺功能测量的选项，用于治疗 CF 肺病的遗传和其他疗法的产生和使用。现在可以提供非常详细的区域肺通气信息，非常适合 CF 肺病的诊断、治疗和监测需求，特别是在应用基因疗法之后。在这篇综述中，我们讨论了现在可用于基于成像的肺功能测量的选项，用于治疗 CF 肺病的遗传和其他疗法的产生和使用。现在可以提供非常详细的区域肺通气信息，非常适合 CF 肺病的诊断、治疗和监测需求，特别是在应用基因疗法之后。在这篇综述中，我们讨论了现在可用于基于成像的肺功能测量的选项，用于治疗 CF 肺病的遗传和其他疗法的产生和使用。