C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 5/00 (2006.01) A61K 31/19 (2006.01) C07K 14/47 (2006.01)
Patent
CA 2143306
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 mut- ant 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 cel- lular 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.
Cheng Seng H.
Fang Shaona L.
Hoppe Henry
Smith Alan E.
Welsh Michael J.
Genzyme Corporation
Smart & Biggar
University Of Iowa Research Foundation
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