AMINOGLYCOSIDE ANTIBIOTICS FOR CYSTIC FIBROSIS

大约百分之五到百分之十的囊性纤维化患者 人们相信患有这种疾病，因为呼吸道内壁的细胞没有 能够合成一种称为囊性纤维化跨膜的蛋白质 过早停止突变导致的电导调节因子（CFTR）。 我们有 发现在某些情况下可能会刺激细胞 通过用相同的抗生素处理它们来制造正常的蛋白质 囊性纤维化患者通常接受治疗作为治疗的一部分。 氨基糖苷类抗生素（妥布霉素、庆大霉素、阿米卡星） 常规给予囊性纤维化患者治疗肺部疾病 感染。 本研究的目的是确定这些是否相同 抗生素也可能通过以下方式帮助囊性纤维化患者的亚组： 允许他们的肺细胞产生正常的 CFTR 蛋白。 迄今为止，7 患者已完成研究。 我们打算研究另外 20 个 未来的患者。

Approximately five to ten percent of patients with cystic fibrosis are believed to have the disease because cells lining their airways are not capable of synthesizing a protein called the cystic fibrosis transmembrane conductance regulator (CFTR) due to premature stop mutations. We have discovered that in some cases it may be possible to stimulate cells to make the normal protein by treating them with the same antibiotics which cystic fibrosis patients routinely receive as part of their therapy. Aminoglycoside antibiotics (Tobramycin, Gentamicin, Amikacin) are routinely given to cystic fibrosis patients for the treatment of lung infections. The purpose of this study is to determine whether these same antibiotics might also help a subgroup of cystic fibrosis patients by allowing their lung cells to produce a normal CFTR protein. To date, 7 patients have completed the study. We intend to study 20 additional patients in the future.