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Gapmers pp 3–19Cite as

Invention and Early History of Gapmers

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Part of the book series: Methods in Molecular Biology ((MIMB,volume 2176))

Abstract

Gapmers are antisense oligonucleotides composed of a central DNA segment flanked by nucleotides of modified chemistry. Hybridizing with transcripts by sequence complementarity, gapmers recruit ribonuclease H and induce target RNA degradation. Since its concept first emerged in the 1980s, much work has gone into developing gapmers for use in basic research and therapy. These include improvements in gapmer chemistry, delivery, and therapeutic safety. Gapmers have also successfully entered clinical trials for various genetic disorders, with two already approved by the U.S. Food and Drug Administration for the treatment of familial hypercholesterolemia and transthyretin amyloidosis-associated polyneuropathy. Here, we review the events surrounding the early development of gapmers, from conception to their maturity, and briefly conclude with perspectives on their use in therapy.

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Authors and Affiliations

  1. Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada

    Kenji Rowel Q. Lim & Toshifumi Yokota

  2. The Friends of Garrett Cumming Research and Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, Edmonton, AB, Canada

    Toshifumi Yokota

Authors
  1. Kenji Rowel Q. Lim
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  2. Toshifumi Yokota
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Correspondence to Toshifumi Yokota .

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Editors and Affiliations

  1. Department of Medical Genetics, University of Alberta, Edmonton, AB, Canada

    Toshifumi Yokota

  2. Department of Medical Genetics, University of Alberta, Edmonton, AB, Canada

    Rika Maruyama

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Lim, K.R.Q., Yokota, T. (2020). Invention and Early History of Gapmers. In: Yokota, T., Maruyama, R. (eds) Gapmers. Methods in Molecular Biology, vol 2176. Humana, New York, NY. https://doi.org/10.1007/978-1-0716-0771-8_1

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  • DOI: https://doi.org/10.1007/978-1-0716-0771-8_1

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  • Publisher Name: Humana, New York, NY

  • Print ISBN: 978-1-0716-0770-1

  • Online ISBN: 978-1-0716-0771-8

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