AMINOGLYCOSIDE ANTIBIOTICS FOR CYSTIC FIBROSIS

大约5%到10%的囊性纤维化患者 他们被认为患有这种疾病，因为他们的气道细胞没有 能够合成一种叫做囊性纤维化跨膜蛋白质 电导调节因子（CFTR）由于过早停止突变。 我们有 发现在某些情况下，刺激细胞 通过使用抗生素来制造正常蛋白质， 囊性纤维化患者常规接受的治疗作为其治疗的一部分。 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用于治疗肺 感染. 这项研究的目的是确定这些相同的 抗生素也可能有助于囊性纤维化患者的亚组， 让他们的肺细胞产生正常的CFTR蛋白。 迄今为止， 患者已完成研究。 我们打算再研究20个 患者在未来

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.