Gene Delivery Vectors and Physical Methods: Present and Future Trends

Dear Colleagues,

The past few decades have witnessed the evolution of gene medicine from an experimental technology into a viable strategy for developing therapeutics for a wide range of disorders.

One of the significant advantages of nucleic acid drugs over currently available low-molecular-weight pharmaceuticals is their selective recognition of molecular targets and pathways, which imparts tremendous specificity of action. Nucleic acid therapeutics have been extensively used to generate therapeutic proteins (e.g., pDNA, mRNA) and to eliminate disease-causing proteins (e.g., siRNA, miRNA, ASOs). More recently, the CRISPR/Cas system and its predecessors, TALENs and ZFNs, have generated excitement for new and improved gene therapies.

Despite such potential, therapeutic success still largely depends on the development of safe and effective ways to deliver nucleic acids within targeted cells. Gene delivery technologies are therefore in the limelight. They are broadly classified into two groups: vector-based and physical methods. No one technology can be applied to all cells and all experimental set-ups. The ideal approach must be selected depending on the cell type and experimental needs, must be as effective as possible, display low (cyto)toxicity and minimal effects on physiology, and be easy to use and reproducible.

Moreover, high-throughput technologies—that is, a reliable tool for pre-clinical testing and further improvement of gene delivery vectors and/or nucleic acid drugs—continue to be developed.

Below is an inclusive, but not exhaustive, list of various technologies that this Special Issue will cover:

• Non-viral gene delivery vectors;
• Viral gene delivery vectors;
• Electroporation technology;
• Sonoporation technology;
• Photoporation technology;
• Magnetofection technology;
• Hydroporation technology;
• Biolistic or gene gun technology;
• High-throughput gene delivery technologies.

Prof. Dr. Gabriele Candiani
Guest Editor

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