University of California

C Randell Brown

William J Welch

J&ouml;rg Tatzelt

A method of improving a phenotypic defect in a cell that contains a conformationally defective target protein wherein the conformational defect causes the phenotype defect, comprising contacting a first cell that expresses said conformationally defective target protein with an amount of a protein stabilizing agent that is effective to improve the conformational defect, thereby improving the phenotypic defect of the first cell in comparison with a second cell having the same conformationally defective target protein and phenotypic defect, wherein the second cell is not contacted with the protein stabilizing agent.

Correction of genetic defects using chemical chaperones

A method of improving a phenotypic defect in a cell that contains a conformationally defective target protein wherein the conformational defect causes the phenotype defect, comprising contacting a first cell that expresses said confonnationally defective target protein with an amount of a protein stabilizing agent that is effective to improve the conformational defect, thereby improving the phenotypic defect of the first cell in comparison with a second cell having the same conformationally defective target protein and phenotypic defect, wherein the second cell is not contacted with the protein stabilizing agent.

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Hershey, PA, United States of America

C Randell Brown