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Ribozymes and siRNAs: From Structure to Preclinical Applications

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Book cover RNA Towards Medicine

Part of the book series: Handbook of Experimental Pharmacology ((HEP,volume 173))

Abstract

The discovery that nucleic acids mediated the inhibition of gene expression in a sequence-specific manner has provided the scientific community with a potentially important tool to analyse gene function and validate drug targets. Selective inhibition of gene expression by ribozymes and small interfering RNAs (siRNAs) is being explored for potential therapeutics against viral infections, inflammatory disorders, haematological diseases and cancer. In order to be used as pharmaceutical drugs, chemical modifications are necessary to increase their stability in vivo. However, such modifications should not affect either the ribozyme cleavage activity or the incorporation of the siRNAs into the RNA interference (RNAi) targeting complex and subsequent mRNA cleavage. To attain stability, ribozymes and siRNAs must also overcome several other problems, including accessibility to target messenger RNAs (mRNAs), efficient delivery to target cells and unwanted non-specific effects.

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Author information

Authors and Affiliations

  1. Institute for Cancer Research, Department of Immunology, Molecular Medicine Group, The Norwegian Radium Hospital, Montebello, 0310, Oslo, Norway

    M. Sioud

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  1. M. Sioud
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Editors and Affiliations

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

    Volker Erdmann

  2. Institute of Bioorganic Chemistry, Polish Academy of Scienes, Noskowskiego 12/14, 61-704, Poznan, Poland

    Jan Barciszewski

  3. Institute of Experimental Pathology, Molecular Neurobiology (ZMBE), University of Münster, Von-Esmarch-Str. 56, 48149, Münster, Germany

    Jürgen Brosius

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Sioud, M. (2006). Ribozymes and siRNAs: From Structure to Preclinical Applications. In: Erdmann, V., Barciszewski, J., Brosius, J. (eds) RNA Towards Medicine. Handbook of Experimental Pharmacology, vol 173. Springer, Berlin, Heidelberg. https://doi.org/10.1007/3-540-27262-3_11

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