TY -的T1 Theratyping囊胞性纤维症体外< em > < / em > ALI-culture和瀑样模型产生patient-derived鼻上皮有条件地重组干细胞JF -欧洲呼吸杂志》乔和J - 10.1183/13993003.00908 -2021欧元SP - 2100908 AU Sette乔凡尼非盟- Lo西塞罗,普盟——Blacona乔凡娜AU - Pierandrei,西尔维亚AU -布鲁诺,萨比娜玛丽亚盟——Salvati瓦伦蒂娜AU -卡斯泰利,Germana盟——Falchi马里奥盟——Fabrizzi贝内黛塔非盟-西米诺朱盟- De Maria,另盟——Biffoni毛罗。非盟- Eramo,阿德里亚娜AU -卢卡雷利,马可Y1 - 2021/01/01 UR - //www.qdcxjkg.com/content/early/2021/07/29/13993003.00908 - 2021. -抽象N2 -问题囊性纤维化(CF)是由于致病变种在囊性纤维化跨膜电导调节(雌性生殖道)基因。最近的改进使药物疗法针对恢复CFTR突变检测表达和功能。雌性生殖道“调节器”已经彻底改变了CF治疗景观,尤其是最后Trikafta批准。这种药物组合表示,FDA最近,EMA的CFTR基因型携带至少一份检测F508del致病变种。然而,一些基因型,还没有资格获得Trikafta治疗,。材料/病人和方法,我们利用了一个创新的细胞方法允许高效的vitro-expansion气道上皮干细胞(AESC)通过有条件的改变(CRC)鼻刷牙的CF患者。AESC-derived个性化疾病的这种方法,耦合发展模式,如瀑样液体和空气界面(ALI)文化,揭示了高度适合雌性生殖道pharmacological-testing。结果和对这个问题的回答我们完全验证了实验模型和实现的雌性生殖道CFTR功能分析和生化检测蛋白质描述,允许评估可用的临床疗效调节器在恢复每个patient-derived雌性生殖道的成熟和功能“阿凡达”(theratyping)。F508del纯合基因型,用于控制,证实了更高的临床活动Trikafta相比,年长的调节器。Trikafta显示其疗效也在三个罕见基因型以前没有资格获得modulators-treatment,打开方式临床翻译。 Finally, encouraging results for innovative drug combinations were also obtained.FootnotesThis manuscript has recently been accepted for publication in the European Respiratory Journal. It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJ online. Please open or download the PDF to view this article.Conflict of interest: Giovanni Sette has nothing to disclose.Conflict of interest: Stefania Lo Cicero has nothing to disclose.Conflict of interest: Giovanna Blaconà has nothing to disclose.CConflict of interest: Silvia Pierandrei has nothing to disclose.Conflict of interest: Sabina Maria Bruno has nothing to disclose.Conflict of interest: Valentina Salvati has nothing to disclose.Conflict of interest: Germana Castelli has nothing to disclose.Conflict of interest: Mario Falchi has nothing to disclose.Conflict of interest: Benedetta Fabrizzi has nothing to disclose.Conflict of interest: Giuseppe Cimino has nothing to disclose.Conflict of interest: Ruggero De Maria has nothing to disclose.Conflict of interest: Mauro Biffoni has nothing to disclose.Conflict of interest: Adriana Eramo has nothing to disclose.Conflict of interest: Marco Lucarelli has nothing to disclose. ER -