期刊
EUROPEAN JOURNAL OF ORGANIC CHEMISTRY
卷 2021, 期 29, 页码 4076-4087出版社
WILEY-V C H VERLAG GMBH
DOI: 10.1002/ejoc.202100338
关键词
Calixarene; Gene delivery systems; Gene therapy; Multivalency; Non-viral vector
资金
- Italian Ministry of Instruction, University and Research project (COMP-HUB initiative)
- Italian Ministry of Instruction, University and Research project (Departments of Excellence Program)
- Italian Ministry of Instruction, University and Research project (Bac-Hound PRIN) [2017E44A9P]
The tetra-L-arginino-tetrahexyloxycalix[4]arene 1 has shown remarkable abilities to compact and deliver various types of nucleic acid cargos into cells. Small structural changes in the calixarene vectors may affect their ability to compact DNA and deliver cargo into cells.
The tetra-L-arginino-tetrahexyloxycalix[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 non-viral 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 alpha-amino 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.
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