Abstract: Methods and compositions for treating CF by mobilizing mutant forms of CFTR, which retain at least some functional activity, to the plasma membrane where they can mediate chloride ion transport are disclosed.

Abstract: Methods and compositions for treating CF by mobilizing mutant forms of CFTR, which retain at least some functional activity, to the plasma membrane where they can mediate chloride ion transport are disclosed.

Abstract: Methods for treating cystic fibrosis are described. The methods involve the administration of a protein enhancing agent, differentiating agent and/or carboxy-compound to a subject afflicted with cystic fibrosis such that mutant cystic fibrosis transmembrane regulator protein present within cystic fibrosis-associated cells becomes functional. Other aspects described include therapeutic compositions and packaged drugs.

Abstract: A DNA comprising at least one inactivated hormone responsive element and a nucleic acid sequence encoding a membrane-associated protein is described. Therapeutic compositions and cells including the DNA are also described. Other aspects of the invention include methods of treating subjects having cystic fibrosis which include administering an effective amount of the DNA to subjects having cystic fibrosis such that functional cystic fibrosis transmembrane conductance regulator is produced by the subject at a level which is not detrimental to the subject. The present invention also pertains to a method of introducing the DNA into a cell such that the membrane-associated protein is produced at a level which is not detrimental to the cell and cells produced by this method.

Abstract: Methods and compositions for treating Cystic Fibrosis by mobilizing mutant forms of CFTR, which retain at least some functional activity, to the plasma membrane where they can mediate chloride ion transport are disclosed.