Novel non-viral vectors for gene delivery: synthesis of a second-generation library of mono-functionalized poly-(guanidinium)amines and their introduction into cationic lipids.
The development of new gene delivery technologies is a prerequisite towards gene therapy clinical trials. Because gene delivery mediated by viral vectors remains of limited scope due to immunological and propagation risks, the development of new non-viral gene delivery systems is of crucial importance. We have synthesized a secondary library of mono-functionalized poly-(guanidinium)amines generated from a library of mono-functionalized polyamines applying the concept of "libraries from libraries." The method allows a quick and easy access to mono-functionalized geometrically varied poly-(guanidinium)amines. The new building blocks were introduced into cationic lipids to obtain novel poly-(guanidinium)amine lipids, which are potential DNA vectors for gene delivery.
Byk
G
G
UMR-133 Rhône-Poulenc Rorer Gencell/CNRS 13, Quai Jules Guesde B.P. 14, 94403-Vitry sur Seine, France. gerardo.byk@rp-rorer.fr
Soto
J
J
Mattler
C
C
Frederic
M
M
Scherman
D
D
D002712:Chlorides / Q000737:chemistry; D002851:Chromatography, High Pressure Liquid; D005029:Ethylenediamines / Q000737:chemistry; D005822:Genetic Vectors / Q000138:chemical synthesis*; D019791:Guanidine / Q000737:chemistry*; D008055:Lipids / Q000737:chemistry*; D009682:Magnetic Resonance Spectroscopy; D013058:Mass Spectrometry; D011073:Polyamines / Q000138:chemical synthesis*; D012116:Resins, Plant / Q000737:chemistry; D013096:Spermine / Q000737:chemistry; D014269:Trifluoroacetic Acid / Q000737:chemistry
D002712:Chlorides; D005029:Ethylenediamines; D008055:Lipids; D011073:Polyamines; D012116:Resins, Plant; D013096:Spermine; C099539:tris(2-aminoethyl)amine; D014269:Trifluoroacetic Acid; D019791:Guanidine