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AMINOGLYCOSIDE TO CORRECT CELLULAR DEFECT IN CF AIRWAYS EPITHELIA MUTATIONS

AMINOGLYCOSIDE TO CORRECT CELLULAR DEFECT IN CF AIRWAYS EPITHELIA MUTATIONS
氨基糖苷可纠正 CF 气道上皮突变的细胞缺陷
批准号：
6115535
负责人：
JOSEPH Mark PILEWSKI
金额：
$2.06万
依托单位：
CHILDREN'S HOSP PITTSBURGH/UPMC HLTH SYS
依托单位国家：
美国
项目类别：
财政年份：
1998
资助国家：
美国
项目状态：
已结题
起止时间：
1998-12-01 至 1999-11-30
来源：
https://reporter.nih.gov/project-details/6115535
关键词：
child (0-11) chloride channels clinical research clinical trial phase I cystic fibrosis gentamicins human subject human therapy evaluation metabolism disorder chemotherapy respiratory epithelium

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中文摘要
翻译
该研究的目的是进行一项双盲研究，以测试氨基糖苷诱导停止突变的穿透性和恢复鼻上皮CFTR蛋白的功能。将招募具有停止突变G542X、R553X、W1282X或R1162X的囊性纤维化患者。庆大霉素将应用于一个鼻孔的鼻上皮，然后对侧鼻孔单独接受稀释剂。一项检测将用于评估功能性CFTR的恢复。本试验为鼻电位差测定。
英文摘要
The objective of the study is to perform a double-blinded study to test the avility of aminoglycoside to induce read-through of stop mutations and restore functional CFTR protein in the nasal epithelium. Cystic fibrosis patients with the stop mutation G542X, R553X, W1282X or R1162X will be recruited. Gentamicin will be applied to the nasal epithelium of one nostril then the contralateral nostril will receive dilulent alone. One assay will be used to assess restoration of functional CFTR. This assay is nasal potential difference measurement.
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Clinical/Biostatistics Core
Clinical/Biostatistics Core
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