NOVEL NONVIRAL VECTORS FOR GENE DELIVERY - SYNTHESIS OF A 2ND-GENERATION LIBRARY OF MONO-FUNCTIONALIZED POLY-(GUANIDINIUM)AMINES AND THEIR INTRODUCTION INTO CATIONIC LIPIDS

The development of new gene delivery technologies is a prerequisite to wards gene therapy clinical trials. Because gene delivery mediated by viral vectors remains of limited scope due to immunological and propag ation risks, the development of new non-viral gene delivery systems is of crucial importance. We have synthesized a secondary library of mon o-functionalized poly(guanidinium)amines generated from a library of m ono-functionalized polyamines applying the concept of ''libraries from libraries.'' The method allows a quick and easy access to mono-functi onalized geometrically varied poly-(guanidinium)amines. The new buildi ng blocks were introduced into cationic lipids to obtain novel poly-(g uanidinium)amine lipids, which are potential DNA vectors for gene deli very. (C) 1998 John Wiley & Sons, Inc.