Abstract
Nanoscale systems have emerged in the past two decades as attractive platforms for delivering nucleic acids in vivo while performing other therapeutic or diagnostic functions, though their full potential for improving human health has yet to be realized in the clinic. Bioengineering techniques have been crucial for modifying and optimizing synthetic and viral delivery systems to include drugs, imaging agents and targeting moieties as well as reducing toxicity effects and increasing delivery efficiency and specificity. Directed delivery technologies can complement these nanoscale systems to localize therapy in vivo. The use of nucleic acid analogs can also enhance therapeutic efficacy under ideal circumstances. This chapter will review some of the recent developments in RNA and DNA delivery research with a focus on progress toward human therapies, the challenges that have been encountered, and the engineering approaches that have been employed. In addition to on-going work on the optimization of delivery systems, three challenging areas are identified: (1) the development of heterogeneous, three-dimensional microenvironments for testing delivery systems, (2) imaging approaches to understand the dynamic interactions of systems from administration through delivery in the human population, and (3) development and translation of directed technologies capable of enhancing delivery in a clinical setting and producing a sustained therapeutic effect.
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Acknowledgments
The authors are indebted to Drs. William Heetderks and Antonio Sastre for their critical review and to Ms. Christine Rogers for her assistance with preparing the manuscript. Figure 26.4 Used with permission and modified from Biochimica et Biophysica Acta (BBA)—Proteins and Proteomics, 1697, Yoon S. Cho-Chung, Antisense protein kinase A RIα-induced tumor reversion: portrait of a microarray, 71–79, Copyright 2004, with permission from Elsevier.
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Conroy, R., Seto, B. (2014). Multifunctional Nanoscale Delivery Systems for Nucleic Acids. In: Cai, W. (eds) Engineering in Translational Medicine. Springer, London. https://doi.org/10.1007/978-1-4471-4372-7_18
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