2 research outputs found
Glycine-Terminated Dendritic Amphiphiles for Nonviral Gene Delivery
Development of nonviral vectors for the successful application
of gene therapy through siRNA/DNA transfection of cells is still a
great challenge in current research., In the present
study, we have developed multivalent polyglycerol dendron based amphiphiles
with well-defined molecular structures that express controlled glycine
arrays on their surfaces. The structure–activity relationships
with respect to the siRNA complexation, toxicity, and transfection
profiles were studied with synthesized amphiphilic polycations. Our
findings revealed that a second-generation amphiphilic dendrimer (G2-octaamine, <b>4</b>) that has eight amine groups on its surface and a hydrophobic
C-18 alkyl chain at the core of the dendron, acts as an efficient
vector to deliver siRNA and achieve potent gene silencing by investigating
the knockdown of luciferase and GAPDH gene activity in HeLa cells.
Interestingly, the amphiphilic vector is nontoxic even at higher ratio
of N/P 100. To the best of our knowledge this is the first example
of successful in vitro siRNA transfection using dendritic amphiphiles.
We believe that this supramolecular complex may serve as a new promising
alternative for nonviral siRNA delivery systems and will be investigated
for in vivo siRNA delivery in the future