| 摘要 |
<p>A method for generating cystic fibrosis transmembrane conductance regulator (CFTR) function in cells containing mutant CFTR and methods for treating cystic fibrosis are described. The method for generating CFTR function in cells containing mutant CFTR involves contacting the cell with an agent which increases the level of mutant CFTR at cellular locations where wild-type CFTR normally functions such that the mutant CFTR generates functional ion channels or mediates ion transport at the cellular locations where wild-type CFTR normally functions. The methods for treating cystic fibrosis involve administering to a subject the above-described agent. Other aspects described include therapeutic compositions and packaged drugs.</p> |
