Nasal Epithelial Cell-Based Models for Individualized Study in Cystic Fibrosis.

Nasal Epithelial Cell-Based Models for Individualized Study in Cystic Fibrosis.
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DOI:
10.3390/ijms22094448
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发表时间:
2021-04-24
影响因子:
5.6
通讯作者:
Brewington JJ
Brewington JJ
中科院分区:
生物学2区
文献类型:
--
作者:
Keegan DE;Brewington JJ

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高效CFTR调节剂疗法的出现使大多数囊性纤维化(CF)患者的医疗保健得到显著改善。然而,对于一些人来说,由于其个体CFTR变体的罕见性,或者由于某些变体的可用疗法缺乏生物活性,这些疗法仍然无法获得。解决这一差距的一种建议方法是使用基于原代人类细胞的模型,其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。鼻细胞代表了这样一种组织来源,并且已经成为个体疾病研究的有力模型。鼻细胞的离体培养随着时间的推移而发展,现代鼻细胞模型开始用于预测患者的结果。本次审查将讨论历史和当前最先进的鼻细胞用于CF研究,特别关注使用此类模型来告知个性化的患者护理。
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 CFTR 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.
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