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Development and Clinical Applications of Nucleic Acid Therapeutics

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Nucleic Acid Drugs

Part of the book series: Advances in Polymer Science ((POLYMER,volume 249))

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Abstract

In the past decade, our new understanding of post-transcriptional gene silencing (PTGS) has opened up new vistas that can help us to decipher the function of genes and thus to unravel the basic mechanistic principles and genetic networks in biology. This enormous potential of PTGS to selectively turn off genes either by antisense or RNA interference pathways has made a huge impact on research in basic science. Further, we can apply this knowledge and new set of rules in biomedical research for the development of more potent therapeutics. With a plethora of advantages of PTGS, there are still certain obstacles that need to be addressed to enable translation of this technology to the clinic. This article will focus on a brief history of the discovery and evolution of PTGS and its potential as a technology. We will also discuss the importance of target availability and some strategies for identification of the target sequence. Further, we will give an overview of some past experiences in the development of this technology in the clinic for cancer therapy, with a recent update on clinical development and trials on nucleic acid therapeutics.

Dedicated to the memory of our mentor and a visionary – Dr. Alan M. Gewirtz.

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Authors and Affiliations

  1. Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA

    Veenu Aishwarya, Anna Kalota & Alan M. Gewirtz

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  1. Veenu Aishwarya
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  2. Anna Kalota
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  3. Alan M. Gewirtz
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Correspondence to Veenu Aishwarya .

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  1. Dept. Biomolecular Engineering, Kyoto Institute of Technology, Matsugasaki, Kyoto, 606-8585, Japan

    Akira Murakami

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Aishwarya, V., Kalota, A., Gewirtz, A.M. (2011). Development and Clinical Applications of Nucleic Acid Therapeutics. In: Murakami, A. (eds) Nucleic Acid Drugs. Advances in Polymer Science, vol 249. Springer, Berlin, Heidelberg. https://doi.org/10.1007/12_2011_146

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