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Liver-Targeted Gene and Cell Therapies: An Overview

  • Chapter
Gene Therapy and Cell Therapy Through the Liver

Abstract

Until very recently, management of several liver malignancies, viral hepatitis, hepatic cirrhosis, and hereditary metabolic diseases remained unsatisfactory, and thus, efficient therapeutic approaches have always been in need. In parallel with recent advances in molecular biology and recombinant DNA technologies, research in liver diseases and the quest for molecular insights of disease pathology have witnessed remarkable progression, and early and specific detection of genetic, infectious, and malignant liver diseases has become feasible like never before. Several molecular approaches combining genetics, biology, chemistry, and computer sciences have been introduced, and in particular, gene- and cell-based therapies opened up new opportunities that step out beyond classical pharmacology. Gene therapy emerged as promising therapeutic strategy aiming to introduce genetic material into cells to generate curative effects. Gene therapy comprises various methods of gene delivery and innovative overexpression and silencing designs for specific therapeutic needs (Kay MA, Nat Rev Genet 12:316–328, 2011). Cell-based therapies, on the other hand, aim to use biologically active living cells instead of DNA or RNA as treatment modality. Several extracorporeal and implantable cell therapies have been developed, such as bioartificial liver (Baquerizo A, Mhoyan A, Kearns-Jonker M, Arnaout WS, Shackleton C, Busuttil RW et al, Transplantation 67:5–18, 1999) for short-term treatment and liver cell transplantation for permanent liver replacement (Fox IJ, Chowdhury JR, Kaufman SS, Goertzen TC, Chowdhury NR, Warkentin PI et al, N Engl J Med 338:1422–1426, 1998). In this chapter, we will briefly discuss the current theories and potential applications of gene- and cell-based therapies for the treatment and/or prevention of various liver diseases.

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Acknowledgment

We would like to thank Ms. Ryan Fugett for editing and proofreading the manuscript. The work was supported in part by the National Institute of Health (EB002946 and HL075542).

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Authors and Affiliations

  1. Department of Pharmaceutical and Biomedical Sciences, University of Georgia College of Pharmacy, 250 W. Green Street, Athens, GA, 30602, USA

    Mohammad Alsaggar & Dexi Liu

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  1. Mohammad Alsaggar
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  2. Dexi Liu
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Correspondence to Dexi Liu .

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Editors and Affiliations

  1. Division of Gastroenterology and Hepatology, Graduate School of Medical and Dental Sciences, Niigata University , Niigata, Niigata, Japan

    Shuji Terai

  2. Department of Gastroenterology and Hepatology, Uonuma Institute of Community Medicine, Niigata University, Niigata, Niigata, Japan

    Takeshi Suda

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Alsaggar, M., Liu, D. (2016). Liver-Targeted Gene and Cell Therapies: An Overview. In: Terai, S., Suda, T. (eds) Gene Therapy and Cell Therapy Through the Liver. Springer, Tokyo. https://doi.org/10.1007/978-4-431-55666-4_1

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  • DOI: https://doi.org/10.1007/978-4-431-55666-4_1

  • Publisher Name: Springer, Tokyo

  • Print ISBN: 978-4-431-55665-7

  • Online ISBN: 978-4-431-55666-4

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