Introduction

The invention is relative to functionalized calixarene derivatives able to non-covalently interact with and bind to microRNA and antagomiRNA for their efficient and safe delivery into the cells. Such calixarene derivatives can be used as non-viral vectors for transfecting cells with microRNAs and antagomiRNAs.​

Technical features

MicroRNAs (miRNAs) are short non-coding RNAs regulating the gene expression in cells that can be involved in many severe diseases. For this reason, tehy can be considered relevant targets for the development of new therapies. When the pathology is associated to miRNA deficiency, the introduction into cells of either these molecules or their precursors (premiRNAs) is extremely useful. On the contrary, when the pathology is due to miRNA overproduction or alteration, it is necessary to vehiculate into cells their antagonists (antimiRNAs). The proposed calixarene based vectors are able to efficiently transport miRNAs, premiRNAs and antagomiRNAs into cells. They show performances and ability superior to the commonly used, commercially available transfecting agents. The formulation of the vector-cargo construct is simple and rapid and does not entail chemical modifications of the substrate to transfect.

Possible Applications

• Efficient transfection of mature miRNAs;​
• Efficient transfection of premiRNAs;​
• Efficient transfection of antagomiRNAs.

Advantages

• High efficiency associated to low/negligible citotoxicity and better performances with respect to the current gold standards;​
• Easy preparation and formulation;​
• High efficiency also in absence of helper lipids/adjuvants;​
• Reproducibility and unequivocal identification of the vector structure;​
• No need of chemical modification of the PNA cargo​