Conese Massimo
Department of Biomedical Sciences, University of Foggia, Foggia, Italy.
Endocr Metab Immune Disord Drug Targets. 2011 Mar;11(1):8-22. doi: 10.2174/187153011794982022.
Cystic Fibrosis (CF), the most common autosomal lethal disorder in Caucasians, is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Although CF is multi-organ disease, the lung pathology is the chief cause of morbidity and mortality of CF patients. The hallmarks of CF lung disease are respiratory infection by opportunistic pathogens and a deranged inflammatory response. However, clinical and experimental data suggest that CF is a hyperinflammatory disorder which can arise in the absence of infection. Laboratory and animal studies suggest that CFTR is involved in regulating some neutrophil and macrophage functions and indicate that altered properties of immune cells may contribute to the dysregulated inflammation in the CF lung. Moreover, recent investigations point out to the involvement of lymphocyte subpopulations in the onset of an altered immune response to pathogens. The development of novel therapies aimed to reduce the inflammatory and regulate immune responses, including stem cell-based treatment, will be presented.
囊性纤维化（CF）是白种人中最常见的常染色体隐性致死性疾病，由囊性纤维化跨膜传导调节因子（CFTR）基因突变引起。尽管CF是一种多器官疾病，但肺部病变是CF患者发病和死亡的主要原因。CF肺部疾病的特征是机会性病原体引起的呼吸道感染和紊乱的炎症反应。然而，临床和实验数据表明，CF是一种在无感染情况下也可能出现的过度炎症性疾病。实验室和动物研究表明，CFTR参与调节某些中性粒细胞和巨噬细胞的功能，并表明免疫细胞特性的改变可能导致CF肺部炎症失调。此外，最近的研究指出淋巴细胞亚群参与了对病原体免疫反应改变的发病过程。本文将介绍旨在减轻炎症和调节免疫反应的新型疗法的进展，包括基于干细胞的治疗。
