AMINOGLYCOSIDE ANTIBIOTICS FOR CYSTIC FIBROSIS

大约5%到10%的囊性纤维化患者 被认为患有这种疾病是因为他们呼吸道内的细胞没有 能够合成一种称为囊性纤维化跨膜的蛋白质 由于过早停止突变而导致的电导调节器(CFTR)。我们有 发现在某些情况下有可能刺激细胞 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在治疗过程中接受治疗。 氨基糖苷类抗生素(妥布霉素、庆大霉素、阿米卡星) 常规给囊性纤维化患者治疗肺部疾病 感染。这项研究的目的是确定这些相同的 抗生素也可能通过以下方式帮助囊性纤维化患者亚组 让他们的肺细胞产生正常的CFTR蛋白。到目前为止，7 患者已经完成了这项研究。我们打算再研究20个 未来的病人。

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.