4 research outputs found
Non-adhesive and highly stable biodegradable nanoparticles that provide widespread and safe transgene expression in orthotopic brain tumors
A StructureâActivity Investigation on Modified Analogues of an Argininocalixarene Based Nonâviral Gene Vector
AbstractThe tetraâLâargininoâtetrahexyloxycalix[4]arene 1 has shown extraordinary abilities to compact and internalize different types of Nucleid Acid cargos (DNA, microRNA, PNA) into cells even known to be transfected with great difficulties by commercial nonâviral gene delivery systems. This activity, accompanied by negligible toxicity, makes this calixarene a rather promising prototype of vector for Gene Therapy. In this study we report how small structural changes like i) the lower rim alkyl substituents, ii) the type of the terminal cationic headgroups (guanidinium or primary ammonium), iii) the length of the linker between the macrocycle and the terminal cationic headgroup, iv) the presence/absence of the basic αâamino group of Arg, and v) the stereochemistry (L or D) of Arg, might affect the ability of the novel calixarene vectors to compact DNA and to deliver its cargo into the cells