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TREATING PROTEIN FOLDING DISORDERS WITH SMALL MOLECULE CFTR CORRECTORS
TREATING PROTEIN FOLDING DISORDERS WITH SMALL MOLECULE CFTR CORRECTORS
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机译:用小分子CFTR校正剂治疗蛋白质折叠疾病
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摘要
Novel CFTR corrector compounds that are effective in rescuing halide efflux in a cell are provided. Also provided are methods for treating protein folding disorders (e.g., cystic fibrosis). The methods include administering a CFTR corrector compound or pharmaceutically acceptable salt or prodrug thereof. Methods of screening for CFTR corrector compounds are also described herein. The methods of screening include contacting a cell that endogenously expresses a CFTR mutation with the compound to be screened and detecting a rescue of halide efflux from the cell.
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