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Design and Self-Assembly of Therapeutic Nucleic Acid Nanoparticles (NANPs) with Controlled Immunological Properties

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Handbook of Chemical Biology of Nucleic Acids

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Abstract

Nucleic acids carry out a diverse number of functions as carriers and regulators of gene expression. By combining naturally occurring structural motifs with known base pairing rules, nucleic acids have also been demonstrated to rationally assemble into nucleic acids nanoparticles (NANPs) of varying shapes, sizes, and compositions. These constructs can be utilized as scaffolds for the delivery of therapeutic moieties and, owing to their programmable nature, can be designed to interact and perform dynamic functions within the cellular environment. However, in order to defend against pathogenic nucleic acids, cells have long since evolved signaling pathways capable of recognizing patterns of nucleic acids which are distinctly nonself in order to elicit a respective immune response. While unknown immunostimulation was previously a hurdle for therapeutic nucleic acids, these established pathways now offer an additional facet of design for therapeutic NANPs, which can be constructed to fit a favorable immunostimulatory profile depending on the therapeutic application.

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Acknowledgments

Research reported in this publication was supported by the National Institute of General Medical Sciences of the National Institutes of Health under Award Numbers R01GM120487 and R35GM139587. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

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Authors and Affiliations

  1. Nanoscale Science Program, Department of Chemistry, University of North Carolina at Charlotte, Charlotte, NC, USA

    Morgan Chandler, Leyla Danai & Kirill A. Afonin

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  1. Morgan Chandler
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  2. Leyla Danai
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  3. Kirill A. Afonin
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Correspondence to Kirill A. Afonin .

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  1. Frontier Institute for Biomolecular Engineering Research (FIBER), Konan University, Kobe, Japan

    Naoki Sugimoto

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  1. Nankai University, Tianjin, China

    Zhen Xi

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Chandler, M., Danai, L., Afonin, K.A. (2023). Design and Self-Assembly of Therapeutic Nucleic Acid Nanoparticles (NANPs) with Controlled Immunological Properties. In: Sugimoto, N. (eds) Handbook of Chemical Biology of Nucleic Acids. Springer, Singapore. https://doi.org/10.1007/978-981-19-9776-1_99

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