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Comparison of Organoid Swelling and Biomarkers of CFTR Function to Determine Effects of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation.

作者信息

Graeber Simon Y, van Mourik Peter, Vonk Annelotte M, Kruisselbrink Evelien, Hirtz Stephanie, van der Ent Cornelis K, Mall Marcus A, Beekman Jeffrey M

机构信息

Charité - Universitätsmedizin Berlin, Berlin, Germany.

Berlin Institute of Health (BIH), Berlin, Germany.

出版信息

Am J Respir Crit Care Med. 2020 Dec 1;202(11):1589-1592. doi: 10.1164/rccm.202004-1200LE.

DOI:10.1164/rccm.202004-1200LE
PMID:32687398
Abstract
摘要

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Comparison of Organoid Swelling and Biomarkers of CFTR Function to Determine Effects of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation.比较类器官肿胀和CFTR功能生物标志物以确定鲁马卡托-依伐卡托对F508del突变纯合子囊性纤维化患者的影响。
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Effects of Lumacaftor-Ivacaftor Therapy on Cystic Fibrosis Transmembrane Conductance Regulator Function in Phe508del Homozygous Patients with Cystic Fibrosis.利那洛肽治疗在囊性纤维化跨膜电导调节因子功能对囊性纤维化纯合子 Phe508del 患者的影响。
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