Deciphering of polycationic carbohydrate based non-viral gene delivery agents by ESI-LTQ-Orbitrap using CID/HCD pairwise tandem mass spectrometry

摘要: For almost three decades, gene therapy has been gaining interest to efficiently treat some severe diseases. In such context, the discovery of an efficient non-viral carrier deliver genetic material into targeted cell nuclei is prime importance. Numerous synthetic vectors that have designed exhibit high transfection efficiency but also suffer from extensive cytotoxicity, thus justifying efforts synthesize more bio-compatible ones, for example, with carbohydrate scaffolds. this sense, cyclodextrins (CDs) are well known present low very cytotoxicity in humans and potential, after polycationization, serve as suitable compaction/transfection agents RNA/DNA. However, polycationic CDs must be accurately characterized establish a straightforward structure–biological activity relationship which guided by nitrogen/phosphorus ratio (N/P). study herein, we demonstrated electrospray-(tandem) mass spectrometry (ESI-(MS)MS) combining Collision Induced Dissociation (CID) Higher induced (HCD) useful tool agent characterization. The suitability CID/HCD pairwise combination was investigated structural deciphering five representative members cyclodextrin library. Our approach allows easy access content, type localisation amino groups thereby offering correlate delivery effective compaction oligo-/polynucleotides.