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Tissue-Specific Delivery of Oligonucleotides

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Book cover Oligonucleotide-Based Therapies

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

Abstract

From the initial discovery of short-interfering RNA (siRNA) and antisense oligonucleotides for specific gene knockdown at the posttranscriptional level to the current CRISPR-Cas9 system offering gene editing at the genomic level, oligonucleotides, in addition to their biological functions in storing and conveying genetic information, provide the most prominent solutions to targeted gene therapies. Nonetheless, looking into the future of curing cancer and acute diseases, researchers are only cautiously optimistic as the cellular delivery of these polyanionic biomacromolecules is still the biggest hurdle for their therapeutic realization. To overcome the delivery obstacle, oligonucleotides have been encapsulated within or conjugated with delivery vehicles for enhanced membrane penetration, improved payload, and tissue-specific delivery. Such delivery systems include but not limited to virus-based vehicles, gold-nanoparticle vehicles, formulated liposomes, and synthetic polymers. In this chapter, delivery challenges imposed by biological barriers are briefly discussed; followed by recent advances in tissue-specific oligonucleotide delivery utilizing both viral and nonviral delivery vectors, discussing their advantages, and how judicious design and formulation could improve and expand their potential as delivery vehicles.

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Authors and Affiliations

  1. Department of Molecular and Cellular Biology, Beckman Research Institute, City of Hope, Duarte, CA, USA

    Xin Xia, Nicolette Pollock, Jiehua Zhou & John Rossi

Authors
  1. Xin Xia
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  2. Nicolette Pollock
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  3. Jiehua Zhou
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  4. John Rossi
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Corresponding author

Correspondence to John Rossi .

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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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Xia, X., Pollock, N., Zhou, J., Rossi, J. (2019). Tissue-Specific Delivery of 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_2

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  • DOI: https://doi.org/10.1007/978-1-4939-9670-4_2

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  • Publisher Name: Humana, New York, NY

  • Print ISBN: 978-1-4939-9669-8

  • Online ISBN: 978-1-4939-9670-4

  • eBook Packages: Springer Protocols

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