Keegan Duncan E, Brewington John J
Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center (CCHMC), 3333 Burnet Avenue, Cincinnati, OH 45229, USA.
Department of Pediatrics, University of Cincinnati College of Medicine (UC-COM), 3230 Eden Avenue, Cincinnati, OH 45267, USA.
Int J Mol Sci. 2021 Apr 24;22(9):4448. doi: 10.3390/ijms22094448.
The emergence of highly effective CFTR modulator therapy has led to significant improvements in health care for most patients with cystic fibrosis (CF). For some, however, these therapies remain inaccessible due to the rarity of their individual variants, or due to a lack of biologic activity of the available therapies for certain variants. One proposed method of addressing this gap is the use of primary human cell-based models, which allow preclinical therapeutic testing and physiologic assessment of relevant tissue at the individual level. Nasal cells represent one such tissue source and have emerged as a powerful model for individual disease study. The ex vivo culture of nasal cells has evolved over time, and modern nasal cell models are beginning to be utilized to predict patient outcomes. This review will discuss both historical and current state-of-the art use of nasal cells for study in CF, with a particular focus on the use of such models to inform personalized patient care.
高效CFTR调节剂疗法的出现已使大多数囊性纤维化（CF）患者的医疗保健状况得到显著改善。然而，对一些患者来说，由于其个体变体罕见，或者由于某些变体的现有疗法缺乏生物活性，这些疗法仍然无法获得。一种解决这一差距的提议方法是使用原代人细胞模型，该模型允许在个体水平上对相关组织进行临床前治疗测试和生理评估。鼻细胞就是这样一种组织来源，并已成为个体疾病研究的有力模型。鼻细胞的体外培养随着时间的推移不断发展，现代鼻细胞模型开始被用于预测患者的预后。本综述将讨论鼻细胞在CF研究中的历史和当前的先进应用，特别关注此类模型在为个性化患者护理提供信息方面的应用。
