Department of Pediatrics, Emory University School of Medicine, Atlanta, GA 30322, USA.
Center for CF & Airways Disease Research, Children's Healthcare of Atlanta, Atlanta, GA 30322, USA.
Int J Mol Sci. 2020 May 8;21(9):3331. doi: 10.3390/ijms21093331.
Cystic fibrosis (CF) lung disease is characterized by unconventional mechanisms of inflammation, implicating a chronic immune response dominated by innate immune cells. Historically, therapeutic development has focused on the mutated cystic fibrosis transmembrane conductance regulator (CFTR), leading to the discovery of small molecules aiming at modulating and potentiating the presence and activity of CFTR at the plasma membrane. However, treatment burden sustained by CF patients, side effects of current medications, and recent advances in other therapeutic areas have highlighted the need to develop novel disease targeting of the inflammatory component driving CF lung damage. Furthermore, current issues with standard treatment emphasize the need for directed lung therapies that could minimize systemic side effects. Here, we summarize current treatment used to target immune cells in the lungs, and highlight potential benefits and caveats of novel therapeutic strategies.
囊性纤维化（CF）肺病的特征是炎症的非传统机制，涉及先天免疫细胞主导的慢性免疫反应。从历史上看，治疗方法的开发一直集中在突变的囊性纤维化跨膜电导调节因子（CFTR）上，从而发现了旨在调节和增强质膜上 CFTR 的存在和活性的小分子。然而，CF 患者的治疗负担、现有药物的副作用以及其他治疗领域的最新进展突出表明，需要针对驱动 CF 肺损伤的炎症成分开发新的疾病靶向治疗方法。此外，目前标准治疗存在的问题强调需要进行靶向肺部的治疗，以最大程度地减少全身副作用。在这里，我们总结了目前用于靶向肺部免疫细胞的治疗方法，并强调了新型治疗策略的潜在益处和注意事项。
