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Progress in the Development of Nucleic Acid Therapeutics

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RNA Towards Medicine

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

Abstract

Abnormal gene expression is a hallmark of many diseases. Gene-specific down-regulation of aberrant genes could be useful therapeutically and potentially less toxic than conventional therapies due its specificity. Over the years, many strategies have been proposed for silencing gene expression in a gene-specific manner. Three major approaches are antisense oligonucleotides (AS-ONs), ribozymes/DNAzymes, and RNA interference (RNAi). In this brief review, we will discuss the successes and shortcomings of these three gene-silencing methods, and the approaches being taken to improve the effectiveness of antisense molecules. We will also provide an overview of some of the clinical applications of antisense therapy.

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Authors and Affiliations

  1. Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, 421 Curie Blvd, Philadelphia, PA, 19104, USA

    A. Kalota, V.R. Dondeti & A.M. Gewirtz

Authors
  1. A. Kalota
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  2. V.R. Dondeti
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  3. A.M. Gewirtz
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Editor information

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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© 2006 Springer-Verlag Berlin Heidelberg

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Kalota, A., Dondeti, V., Gewirtz, A. (2006). Progress in the Development of Nucleic Acid Therapeutics. 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_9

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