Small Interfering RNAs, Antisense Drugs and Therapeutic Oligonucleotide Analogues

Dear Colleagues,

One of the major biomedical and pharmaceutical challenges is a highly selective therapy against abnormal gene expression in disease states. Most conventional small-molecule drugs confront downstream cellular components, when disease is already established in cell components at the level of expressed pathogenic proteins. Although successful at treating or alleviating symptoms for some disease states, these approaches often suffer from off-target effects and severe toxicity, typical for chemotherapy and anti-viral drugs. Small interfering RNAs, antisense oligonucleotides and therapeutic nucleic acid analogues offer a powerful platform for the development of novel therapeutic strategies for the selective inhibition of disease states via the controlled translational arrest of disease-relevant proteins by targeting upstream cellular components (e.g. DNA, messenger RNA, microRNA or viral genomic RNA). Therapeutic nucleic acids and their functional analogues use the most sophisticated and elegant 3D molecular design and offer high efficacy, remarkable selectivity, and fewer side effects due to their unique shape, precision in molecular recognition and action. These therapeutic strategies need to be developed further, optimized, tested in pre-clinical models, and translated into future clinical applications. For this Special Issue, we invite manuscripts addressing one or several of the following aspects:

1. New synthetic strategies for the fabrication of innovative nucleic acid analogues to improve their potency, precision in sequence-specific recognition, biological stability, resistance to cellular nucleases and/or intracellular delivery.
2. Mechanistic studies on such nucleic acid therapeutics, including but not limited to the investigation of their mechanisms of action responsible for the observed effects and/or therapeutic responses at the molecular, cellular, or physiological levels.
3. Development of therapeutic siRNAs or antisense oligonucleotides and/or their functional derivatives that provide means for cell-specific or tissue-selective targeting.
4. Experimental evidence of the substantial transformative potential of nucleic acid therapeutics through the manipulation of gene expression, demonstrated at the tissue or cellular level, and/or in pre-clinical animal models.
5. Study of toxicological and immune responses to treatments with siRNAs, antisense drugs, or therapeutic nucleic acids, with evaluation of potential toxicity and off-target effects.

Dr. Elena V. Bichenkova
Guest Editor

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• nucleic acid therapeutics
• RNA
• antisense DNA
• cellular delivery
• hybridisation
• sequence specificity
• knockdown
• substrate turnover
• gene silencing
• translational arrest