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The Challenges and Current Advances in Delivering RNAi as Therapeutics

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DNA and RNA Nanobiotechnologies in Medicine: Diagnosis and Treatment of Diseases

Part of the book series: RNA Technologies ((RNATECHN))

Abstract

The discovery of RNA interference (RNAi) has transformed molecular biology and offers enormous therapeutic potential. Currently, there are a few approaches to deliver RNAi into cells including the use of a small interfering RNA (siRNA) or a plasmid to express short hairpin RNA (shRNA) or microRNA. Each of these approaches has its advantages and drawbacks. To fully realise the potential of RNAi in clinic, hurdles including specificity, stability and interferon activation of the small RNA need to be overcome. In order to develop RNAi as pharmaceutics for a range of diseases, strategies to deliver the small RNA in specific cell types need to be further developed and refined. In this chapter, the different classes of small RNA, current strategies to overcome therapeutic hurdles and different methods to deliver RNAi are discussed.

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  1. Department of Biochemistry, University of Cambridge, Sanger building, 80 Tennis Court Road, Cambridge, CB2 1GA, UK

    Albert Kwok

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  1. Institute of Chemistry/Biochemistry, Free University Berlin, Thielallee 63, Berlin, 14195, Berlin, Germany

    Volker A. Erdmann

  2. Inst. Bioorganic Chemistry, PAN Poznan, ul. Z. Noskowskiego 12/14, Poznan, 61-704, Poland

    Jan Barciszewski

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Kwok, A. (2013). The Challenges and Current Advances in Delivering RNAi as Therapeutics. In: Erdmann, V., Barciszewski, J. (eds) DNA and RNA Nanobiotechnologies in Medicine: Diagnosis and Treatment of Diseases. RNA Technologies. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-36853-0_8

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