Fellner Robert C, Terryah Shawn T, Tarran Robert
Cystic Fibrosis and Pulmonary Diseases Research and Treatment Center, University of North Carolina, 7102 Marsico Hall, 125 Mason Farm Road, Chapel Hill, NC, 27599-7248, USA.
Spyryx Biosciences, 801-9 Capitola Drive, Durham, NC, 27713, USA.
Mol Cell Pediatr. 2016 Dec;3(1):16. doi: 10.1186/s40348-016-0044-8. Epub 2016 Apr 20.
Asthma, chronic obstructive pulmonary disease (COPD), and cystic fibrosis (CF) are all chronic pulmonary diseases, albeit with different etiologies, that are characterized by airflow limitation, chronic inflammation, and abnormal mucus production/rheology. Small synthetic molecule-based therapies are commonly prescribed for all three diseases. However, there has been increased interest in "biologicals" to treat these diseases. Biologicals typically constitute protein- or peptide-based therapies and are often more potent than small molecule-based drugs. In this review, we shall describe the pros and cons of several different biological-based therapies for respiratory disease, including dornase alfa, a recombinant DNAase that reduces mucus viscosity and short palate lung and nasal epithelial clone 1 (SPLUNC1)-derived peptides that treat Na(+) hyperabsorption and rebalance CF airway surface liquid homeostasis.
哮喘、慢性阻塞性肺疾病（COPD）和囊性纤维化（CF）均为慢性肺部疾病，尽管病因不同，但都具有气流受限、慢性炎症以及异常黏液产生/流变学等特征。基于小合成分子的疗法通常用于这三种疾病的治疗。然而，人们对治疗这些疾病的“生物制剂”的兴趣日益增加。生物制剂通常是基于蛋白质或肽的疗法，且往往比基于小分子的药物更有效。在本综述中，我们将描述几种不同的基于生物制剂的呼吸系统疾病治疗方法的优缺点，包括重组脱氧核糖核酸酶多纳酶α，它可降低黏液黏度；以及源自短腭、肺和鼻上皮克隆1（SPLUNC1）的肽，可治疗钠（Na⁺）过度吸收并恢复CF气道表面液体的稳态平衡。
