Genzyme Corporation

Quiming Chu

Seng H Cheng

Ronald K Scheule

Simon J Eastman

Jennifer D Tousignant

The well-differentiated airway epithelium is the principal target tissue for gene therapy for the treatment of CF. However, recent studies have shown that gene delivery vehicles, such as cationic lipid:DNA complexes, can be inefficient at binding to and internalizing into polarized epithelial cells. The present invention provides a method to improve gene therapy by using a compound capable of disrupting tight junctions. In the practice of the invention, the transfection of a biologically active molecule by a cationic amphiphile:biologically active molecule complex or other lipid or viral or nonviral vectors is improved by treating the cells with a class of compounds known in the art as absorption enhancers or tight junction disrupting compounds.

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Melrose, MA, United States of America

Quiming Chu