针对囊性纤维化黏液清除缺陷的黏液靶向治疗：简短综述。
Mucus-targeting therapies of defective mucus clearance for cystic fibrosis: A short review.
机构信息
Marsico Lung Institute/Cystic Fibrosis Center, University of North Carolina, Chapel Hill, NC, 27599-7248, USA.
Marsico Lung Institute/Cystic Fibrosis Center, University of North Carolina, Chapel Hill, NC, 27599-7248, USA; Department of Medicine, University of North Carolina, Chapel Hill, NC, 27599-7248, USA; Department of Cell Biology and Physiology, University of North Carolina, Chapel Hill, NC, 27599-7248, USA.
出版信息
Curr Opin Pharmacol. 2022 Aug;65:102248. doi: 10.1016/j.coph.2022.102248. Epub 2022 Jun 8.
In the lungs, defective CFTR associated with cystic fibrosis (CF) represents the nidus for abnormal mucus clearance in the airways and consequently a progressive lung disease. Defective CFTR-mediated Cl secretion results in altered mucus properties, including concentration, viscoelasticity, and the ratio of the two mucins, MUC5B and MUC5AC. In the past decades, therapies targeting the CF mucus defect, directly or indirectly, have been developed; nevertheless, better treatments to prevent the disease progression are still needed. This review summarizes the existing knowledge on the defective mucus in CF disease and highlights it as a barrier to the development of future inhaled genetic therapies. The use of new mucus-targeting treatments is also discussed, focusing on their potential role to halt the progress of CF lung disease.
在肺部，与囊性纤维化（CF）相关的缺陷 CFTR 代表了气道中异常黏液清除的核心，从而导致进行性肺病。缺陷 CFTR 介导的 Cl 分泌导致黏液特性改变，包括浓度、粘弹性以及两种粘蛋白 MUC5B 和 MUC5AC 的比例。在过去的几十年中，已经开发出了针对 CF 黏液缺陷的直接或间接靶向治疗方法；然而，仍需要更好的治疗方法来预防疾病进展。本文综述了 CF 疾病中缺陷黏液的现有知识，并强调其是未来吸入性基因治疗发展的障碍。还讨论了新型黏液靶向治疗的应用，重点讨论了它们在阻止 CF 肺部疾病进展方面的潜在作用。
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