Synthesis and characterization of degradable multivalent cationic lipids with disulfide-bond spacers for gene delivery.

ABSTRACT: Gene therapy provides powerful new approaches to curing a large variety of diseases, which are being explored in ongoing worldwide clinical trials. To overcome the limitations of viral gene delivery systems, synthetic nonviral vectors such as cationic liposomes (CLs) are desirable. However, improvements of their efficiency at reduced toxicity and a better understanding of their mechanism of action are required. We present the efficient synthesis of a series of degradable multivalent cationic lipids (CMVLn, n=2 to 5) containing a disulfide bond spacer between headgroup and lipophilic tails. This spacer is designed to be cleaved in the reducing milieu of the cytoplasm and thus decrease lipid toxicity. Small angle X-ray scattering demonstrates that the initially formed lamellar phase of CMVLn

SUBMITTER: Shirazi RS 

PROVIDER: S-EPMC3129426 | biostudies-literature | 2011 Sep

REPOSITORIES: biostudies-literature