Structural Nucleic Acids Therapeutics: Design, Synthesis and Applications

Dear Colleagues,

Among biomolecules, nucleic acids are considered potent tools useful for the development of new, responsive therapeutics. This is due to their biocompatibility, programmability, and potential for a variety of chemical modifications. For many years, DNA and RNA oligomers have been designed, synthetized, and modified using chemical and enzymatic methods. Many of them were designed to be used as specific therapeutics, and from the very beginning cancer was their primary target. However, in 2019 a new enemy emerged, causing the global COVID-19 pandemic and affecting every level of our society. Whether or not the DNA and/or RNA will be used to target the causes of such pandemics, the research dedicated to those two players will be advanced. The concept of virus targeting with DNA fragments is not new, since Zamecnik proposed a synthetic oligonucleotide to target the Rous sarcoma virus (RSV) 35S RNA in 1978. However, the advancement in our understanding of important structural and functional properties of nucleic acids, especially RNA, is more recent.

Nucleic acid therapeutics, including antisense oligonucleotides (ASOs), aptamers, DNA-zymes, ribozymes, and small interfering RNA (siRNA) are considered to be some of the new responsive materials to be applied in medical research. The advantage of DNA/RNA also lies in their unique potential to be used for combined therapeutic and diagnostic purposes—leading to their being dubbed as “theranostics”. On one hand, a specific nucleotide fragment of the therapeutic particle can act as a guide, increasing the efficiency of delivery of the effector part to the right location in the human body to inhibit the function of a particular disease-related gene or a set of genes in the cell. On the other hand, the guide can carry a fluorescent, IR, or PET tag necessary for visualization of specific cells or organelles.

Both branches, DNA/RNA therapeutics and diagnostic tools, are widely studied, and year by year new constructs are developed and validated with justified hopes for better effectiveness, increased sensitivity, and broadened applicability. However, new chemical and biochemical ideas are still necessary to make such tools cheaper and more affordable. My hope is that many of you will consider this Special Issue to be a good platform for presenting in-depth reviews and research articles on the latest achievements, present challenges, and future directions of research on nucleic acid therapeutics.

Prof. Dr. Arkadiusz Chworos
Guest Editor

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• structural RNA nanotechnology
• therapeutic nucleic acids
• nucleic acid nanoparticles
• drug delivery
• oligonucleotide therapeutics
• gene expression regulation