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Chemical Development of Therapeutic Oligonucleotides

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Oligonucleotide-Based Therapies

Part of the book series: Methods in Molecular Biology ((MIMB,volume 2036))

Abstract

The development of several different chemical modifications of nucleic acids, with improved base-pairing affinity and specificity as well as increased resistance against nucleases, has been described. These new chemistries have allowed the synthesis of different types of therapeutic oligonucleotides. Here we discuss selected chemistries used in antisense oligonucleotide (ASO) applications (e.g., small interfering RNA (siRNA), RNase H activation, translational block, splice-switching, and also as aptamers). Recently approved oligonucleotide-based drugs are also presented briefly.

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Acknowledgments

This work was supported by the Swedish Research Council, the Stockholm County Council, Hjärnfonden, and Vinnova/SweLife.

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Authors and Affiliations

  1. Department of Laboratory Medicine, Center for Advanced Therapies, Karolinska Institutet, Stockholm, Sweden

    Karin E. Lundin, Olof Gissberg & Rula Zain

  2. Department of Laboratory Medicine, Center for Advanced Therapies, Karolinska University Hospital Huddinge, Karolinska Institutet, Stockholm, Sweden

    C. I. Edvard Smith

  3. Department of Clinical Genetics, Center for Rare Diseases, Karolinska University Hospital, Stockholm, Sweden

    Rula Zain

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  1. Karin E. Lundin
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  2. Olof Gissberg
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  3. C. I. Edvard Smith
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  4. Rula Zain
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Correspondence to Karin E. Lundin .

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Editors and Affiliations

  1. Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

    Olof Gissberg

  2. Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

    Rula Zain

  3. Center for Advanced Therapies, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden

    Karin E. Lundin

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Lundin, K.E., Gissberg, O., Smith, C.I.E., Zain, R. (2019). Chemical Development of Therapeutic Oligonucleotides. In: Gissberg, O., Zain, R., Lundin, K. (eds) Oligonucleotide-Based Therapies. Methods in Molecular Biology, vol 2036. Humana, New York, NY. https://doi.org/10.1007/978-1-4939-9670-4_1

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  • DOI: https://doi.org/10.1007/978-1-4939-9670-4_1

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