Quantification of the physiological changes in people with cystic fibrosis (pwCF) has long been important for clinical diagnosis. Since the original discovery of abnormal sweat electrolytes in pwCF, the standardised pilocarpine iontophoresis sweat test was developed1 to clearly distinguish those with CF. While the sweat chloride test remains an important early step in the diagnostic algorithm, complementary testing methods are required to quantify the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein for both diagnosis and the development of new therapies.2 Building on this foundation, standardised protocols for the in vivo measurement of the nasal potential difference (NPD)3 were developed to evaluate sodium and chloride transport defects in the respiratory epithelium. This test played a pivotal role in the London gene therapy trial.4 5 However, the complexity of measuring CFTR function in respiratory tissues in vivo, particularly with interactions among multiple ion channels, underscored the need for more direct measurement techniques in vitro. The intestinal current measurement technique was developed, using rectal biopsies mounted in Ussing chambers to measure electrophysiological properties. However, Ussing chamber techniques requiring viable, functionally intact tissues presented significant challenges and limited widespread use. To address this gap, the Beckman team cultured the rectal …

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直肠类器官形态分析 (ROMA) — 回复：自动优化测量


囊性纤维化 (pwCF) 患者生理变化的量化长期以来对于临床诊断很重要。自从最初发现 pwCF 汗液电解质异常以来，开发了标准化毛果芸香碱离子电渗汗液测试1，以明确区分 CF 患者。虽然汗液氯化物测试仍然是诊断算法中重要的早期步骤，但需要补充测试方法来量化囊性纤维化跨膜电导调节蛋白 (CFTR) 蛋白的功能，以用于诊断和新疗法的开发。2 在此基础上，开发了鼻电位差 (NPD)3 体内测量的标准化方案，以评估呼吸道上皮中的钠和氯转运缺陷。该测试在伦敦基因治疗试验中发挥了关键作用。4 5 然而，体内测量呼吸组织 CFTR 功能的复杂性，特别是多个离子通道之间的相互作用，强调需要更直接的体外测量技术。开发了肠道电流测量技术，使用安装在尤斯室中的直肠活检来测量电生理特性。然而，使用需要可行的、功能完整的组织的腔室技术提出了重大挑战并限制了广泛使用。为了解决这一差距，贝克曼团队培养了直肠……