TREATING PROTEIN FOLDING DISORDERS WITH SMALL MOLECULE CFTR CORRECTORS
申请人:Schwiebert Erik M.
公开号:US20140073632A1
公开(公告)日:2014-03-13
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.
US9221840B2
申请人:——
公开号:US9221840B2
公开(公告)日:2015-12-29
[EN] TREATING PROTEIN FOLDING DISORDERS WITH SMALL MOLECULE CFTR CORRECTORS<br/>[FR] TRAITEMENT DES TROUBLES LIÉS AU REPLIEMENT DES PROTÉINES AVEC DES CORRECTEURS DE CFTR À PETITE MOLÉCULE
申请人:DISCOVERYBIOMED INC
公开号:WO2012158913A2
公开(公告)日:2012-11-22
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.