Abstract

Introduction: Cystic fibrosis (CF), is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and affects thousands of people throughout the world. Lung disease is the leading cause of death in CF patients. Despite the advances in treatments, the management of CF mainly targets symptoms. Recent CFTR modulators however target common mutations in patients, alleviating symptoms of CF. Unfortunately, there is still no approved treatments for patients with rare mutations to date.

Areas covered: This paper reviews current treatments of CF that mitigate symptoms and target genetic defects. The use of gene and drug delivery systems such as viral or non-viral vectors and nano-compounds to enhance CFTR expression and the activity of antimicrobials against chronic pulmonary infections respectively, will also be discussed.

Expert opinion: Nano-compounds tackle biological barriers to drug delivery and revitalize antimicrobials, anti-inflammatory drugs and even genes delivery to CF patients. Gene therapy and gene editing are of particular interest because they have the potential to directly target genetic defects. Nanoparticles should be formulated to more specifically target epithelial cells, and biofilms. Finally, the development of more potent gene vectors to increase the duration of gene expression and reduce inflammation is a promising strategy to eventually cure CF.

摘要

简介：囊性纤维化（CF）是由囊性纤维化跨膜电导调节剂（CFTR）基因的突变引起的，影响了全世界成千上万的人。肺部疾病是CF患者的主要死亡原因。尽管治疗有所进步，但CF的治疗仍主要针对症状。然而，最近的CFTR调节剂靶向患者中的常见突变，从而减轻了CF的症状。不幸的是，迄今为止，尚无批准的罕见突变患者治疗方法。

涵盖的领域：本文回顾了减轻症状和靶向遗传缺陷的CF的当前治疗方法。也将讨论使用基因和药物递送系统（例如病毒或非病毒载体和纳米化合物）分别增强CFTR表达和抗菌剂对慢性肺部感染的活性。

专家意见：纳米化合物解决了药物传递的生物学障碍，并重现了抗微生物剂，抗炎药，甚至可以将基因传递给CF患者。基因治疗和基因编辑特别令人感兴趣，因为它们具有直接靶向遗传缺陷的潜力。应当配制纳米颗粒以更具体地靶向上皮细胞和生物膜。最后，开发更有效的基因载体以增加基因表达的持续时间并减少炎症是最终治愈CF的有希望的策略。