关键词: CFTR mutations cystic fibrosis drug delivery nanoformulations pulmonary route

来  源:   DOI:10.1016/j.drudis.2023.103729

Abstract:
Cystic fibrosis (CF), a fatal genetic condition, causes thick, sticky mucus. It also causes pancreatic dysfunction, bacterial infection, and increased salt loss. Currently available treatments can improve the patient\'s quality of life. Drug delivery aided by nanotechnology has been explored to alter the pharmacokinetics and toxicity of drugs. In this short review, we aim to summarize various conventional formulations and highlight advanced formulations delivered via the pulmonary route for the treatment of CF. There is considerable interest in advanced drug delivery formulations addressing the various challenges posed by CF. Despite their potential to be translated for clinical use, we anticipate that a significant amount of effort may still be required for translation to the clinic.
摘要:
囊性纤维化(CF),一种致命的遗传病,原因很厚,粘稠的粘液。它还会导致胰腺功能障碍,细菌感染,和增加盐损失。目前可用的治疗方法可以改善患者的生活质量。已经探索了纳米技术辅助的药物递送以改变药物的药代动力学和毒性。在这篇简短的评论中,我们的目的是总结各种常规制剂,并重点介绍通过肺部途径治疗CF的高级制剂。对于解决CF带来的各种挑战的先进药物递送制剂存在相当大的兴趣。尽管它们有可能被翻译为临床用途,我们预计转诊至临床仍需付出相当大的努力.
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