Introduction

The present invention deals with calixarene based derivatives functionalized with units of arginine or guanidinylated amino acids. These compounds work as non-viral vector at high efficiency and low toxicity for cell transfection. Their activity is related to their ability in non-covalently interacting with nucleic acids and in delivering them into the cells and into their nucleus.

Technical features

Gene therapy represents a medical technique extremely potent for the treatment of severe diseases such as tumors, neuropathies, cardiovascular and inherited pathologies. At the basis of this technique stays the fundamental step of the transfer into the cells of genes or, more in general, of nucleic acids and mimics. This delivery is a complex process involving several different steps, some of them not entirely understood yet, for which proper vectors are needed. The calixarene based compounds object of this patent are non-viral vectors that work with an extremely high efficiency and negligible toxicity, delivering nucleic acids of variable lengths into the cells and into their nucleus. Their performance and properties are superior than those of currently used and commercially available vectors and formulations. Their synthesis and purification are based on well established chemical procedures. For their use is sufficient a simple mixing with the cargo (nucleic acid), followed by a short incubation time and admnistration to cells. A contact time of 24 hours between formulation and cells provides high percentage of transfection.

Possible Applications

• Transport into cells and into their nucleus of DNA of variable lengths;
• Transport into the cells of different types of nucleic acids;
• Transport into the cells of nucleic acid mimics.

Advantages

• High transfection efficiency associated with negligible toxicity;
• Ease of preparation and formulation;
• Reproducibility and unambigous identification of vector structure;
• High efficacy, even in absence of «helper lipids»;
• Chemical modification of the cargo not needed.