Animal Core

囊性纤维化（CF）是一种常见的常染色体隐性遗传病，由基因突变引起 编码囊性纤维化跨膜传导调节因子（CFTR）（1）。CF影响多个 器官，包括肺、胰腺、肠、肝、汗腺、胆囊和男性生殖道。 呼吸道感染和炎症是目前发病率和死亡率最高的原因。虽然 几种疗法已经改善了患者的生活，目前的治疗是不够的，CF 仍然是一种致命的疾病。我们对这种疾病的发病机制，其进展， 新生儿肺部的状况不佳此外，尽管基因转移的潜力， 虽然这是一种有效的治疗方法，但一些最根本的问题仍然没有答案。这些 我们知识的空白阻碍了开发更好的CF治疗和预防方法的尝试 肺病。解决这些问题的一个主要障碍是电流的限制。 动物模型尽管携带CFTR无效和错义突变的小鼠品系已经使 巨大的贡献，CF小鼠不发展气道或胰腺疾病通常发现在 人类

Cystic fibrosis (CF) is a common autosomal recessive disease caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) (1). CF affects multiple organs, including lungs, pancreas, intestine, liver, sweat glands, gall bladder and male genital tract. Airway infection and inflammation cun'ently cause most of the morbidity and mortality. Although several therapies have improved the lives of patients, current treatments are inadequate and CF remains a lethal disease. Our knowledge about the pathogenesis of the disease, its progression, and the state of the neonatal lung is inadequate. Moreover, despite the potential of gene transfer to be an effective treatment, some of the most fundamental questions remain unanswered. These gaps in our knowledge have hindered attempts to develop better treatments and preventions for CF lung disease. A major impediment to addressing these issues has been limitations of current animal models. Although mouse strains carrying null and missense CFTR mutations have made enormous contributions, CF mice do not develop the airway or pancreatic disease typically found in humans.