Efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene

Abstract The application of Peptide Nucleic Acids (PNAs), mimics of DNA lacking the sugar-phosphate backbone, for antisense/anti-gene therapy and gene editing is limited by their low uptake by cells. Currently, no simple and efficient delivery systems and methods are available to solve this open iss...

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Autores principales:	Jessica Gasparello, Alex Manicardi, Alessandro Casnati, Roberto Corradini, Roberto Gambari, Alessia Finotti, Francesco Sansone
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2019
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/2b8966ee83064b558c0ea0e41dbfe452
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spelling	oai:doaj.org-article:2b8966ee83064b558c0ea0e41dbfe4522021-12-02T15:09:24ZEfficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene10.1038/s41598-019-39211-42045-2322https://doaj.org/article/2b8966ee83064b558c0ea0e41dbfe4522019-02-01T00:00:00Zhttps://doi.org/10.1038/s41598-019-39211-4https://doaj.org/toc/2045-2322Abstract The application of Peptide Nucleic Acids (PNAs), mimics of DNA lacking the sugar-phosphate backbone, for antisense/anti-gene therapy and gene editing is limited by their low uptake by cells. Currently, no simple and efficient delivery systems and methods are available to solve this open issue. One of the most promising approach is the modification of the PNA structure through the covalent linkage of poliarginine tails, but this means that every PNA intended to be internalized must be modified. Herein we report the results relative to the delivery ability of a macrocyclic multivalent tetraargininocalix[4]arene (1) used as non-covalent vector for anti-miR-221-3p PNAs. High delivery efficiency, low cytotoxicity, maintenance of the PNA biological activity and ease preparation of the transfection formulation, simply attained by mixing PNA and calixarene, candidate this vector as universal delivery system for this class of nucleic acid analogues.Jessica GasparelloAlex ManicardiAlessandro CasnatiRoberto CorradiniRoberto GambariAlessia FinottiFrancesco SansoneNature PortfolioarticleMedicineRScienceQENScientific Reports, Vol 9, Iss 1, Pp 1-10 (2019)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Jessica Gasparello Alex Manicardi Alessandro Casnati Roberto Corradini Roberto Gambari Alessia Finotti Francesco Sansone Efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene
description	Abstract The application of Peptide Nucleic Acids (PNAs), mimics of DNA lacking the sugar-phosphate backbone, for antisense/anti-gene therapy and gene editing is limited by their low uptake by cells. Currently, no simple and efficient delivery systems and methods are available to solve this open issue. One of the most promising approach is the modification of the PNA structure through the covalent linkage of poliarginine tails, but this means that every PNA intended to be internalized must be modified. Herein we report the results relative to the delivery ability of a macrocyclic multivalent tetraargininocalix[4]arene (1) used as non-covalent vector for anti-miR-221-3p PNAs. High delivery efficiency, low cytotoxicity, maintenance of the PNA biological activity and ease preparation of the transfection formulation, simply attained by mixing PNA and calixarene, candidate this vector as universal delivery system for this class of nucleic acid analogues.
format	article
author	Jessica Gasparello Alex Manicardi Alessandro Casnati Roberto Corradini Roberto Gambari Alessia Finotti Francesco Sansone
author_facet	Jessica Gasparello Alex Manicardi Alessandro Casnati Roberto Corradini Roberto Gambari Alessia Finotti Francesco Sansone
author_sort	Jessica Gasparello
title	Efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene
title_short	Efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene
title_full	Efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene
title_fullStr	Efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene
title_full_unstemmed	Efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene
title_sort	efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene
publisher	Nature Portfolio
publishDate	2019
url	https://doaj.org/article/2b8966ee83064b558c0ea0e41dbfe452
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Efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene

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