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 α-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.

A Structure-Activity Investigation on Modified Analogues of an Argininocalixarene Based Non-viral Gene Vector / Lomazzi, M.; Franceschi, V.; Bagnacani, V.; Vezzoni, C. A.; Donofrio, G.; Casnati, A.; Sansone, F.. - In: EUROPEAN JOURNAL OF ORGANIC CHEMISTRY. - ISSN 1434-193X. - (2021), pp. 4076-4087. [10.1002/ejoc.202100338]

A Structure-Activity Investigation on Modified Analogues of an Argininocalixarene Based Non-viral Gene Vector

Lomazzi M.;Franceschi V.;Bagnacani V.;Donofrio G.;Casnati A.;Sansone F.
2021

Abstract

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 α-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.
A Structure-Activity Investigation on Modified Analogues of an Argininocalixarene Based Non-viral Gene Vector / Lomazzi, M.; Franceschi, V.; Bagnacani, V.; Vezzoni, C. A.; Donofrio, G.; Casnati, A.; Sansone, F.. - In: EUROPEAN JOURNAL OF ORGANIC CHEMISTRY. - ISSN 1434-193X. - (2021), pp. 4076-4087. [10.1002/ejoc.202100338]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11381/2899034
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