Few human genetic diseases can rely on the availability of as many and as diverse animal models as cystic fibrosis (CF), a multiorgan syndrome caused by functional absence of cystic fibrosis transmembrane regulator (CFTR). The recent development of highly effective CFTR modulator drug therapies simultaneously highlighted the remarkable clinical improvement achievable with these treatments, the lack of therapeutic alternatives for non-responders, and the need to understand the kinetics of disease upon early life/chronic treatment. These advances have rekindled efforts to leverage animal models to address critical knowledge gaps in CF. This article provides a concise overview of the areas of interests for therapeutic intervention in the current CF landscape, focusing on the contributions of in vivo models to understand CF pathogenesis, identify therapeutic windows, and develop novel therapies for all CFTR mutations.

很少有人类遗传病可以依赖像囊性纤维化 (CF) 这样多且多样的动物模型的可用性，囊性纤维化是一种由囊性纤维化跨膜调节因子 (CFTR) 功能缺失引起的多器官综合征。高效 CFTR 调节剂药物疗法的最新发展同时强调了这些治疗可实现的显着临床改善、无反应者缺乏治疗替代方案以及了解早期生命/慢性治疗的疾病动力学的必要性。这些进步重新点燃了利用动物模型来解决 CF 中关键知识缺口的努力。本文简要概述了当前 CF 景观中治疗干预的兴趣领域，重点关注体内的贡献了解 CF 发病机制、确定治疗窗口并为所有 CFTR 突变开发新疗法的模型。