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Updated: Jul 1, 2025

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
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一种独特有效的CFTR校正器,具有互补的作用模式

Valentina Marchesin1, Lucile Monnier1, Peter Blattmann1

  • 1Idorsia Pharmaceuticals Ltd., 4123 Allschwil, Switzerland.

Science advances
|March 1, 2024
PubMed
概括

新的宏环化合物作为"IV型"校正剂,在囊性纤维化模型中显著改善F508del-囊性纤维化跨膜导电性调节器 (CFTR) 蛋白质的折叠和功能.

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