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Recoding Therapies for Genetic Diseases

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Recoding: Expansion of Decoding Rules Enriches Gene Expression

Part of the book series: Nucleic Acids and Molecular Biology ((NUCLEIC,volume 24))

Abstract

Strategies aimed at recoding premature stop mutations or frameshift mutations have the potential to treat a genotypic subset of patients afflicted with many different genetic diseases. In this chapter we provide an overview of approaches to promote readthrough of premature stop mutations, including pharmacological agents and suppressor tRNAs. We also describe the use of oligonucleotides to induce differential splicing to exclude disease-causing mutations or to induce site-specific frameshifting as a method of recoding mutations that alter the ribosomal reading frame. Finally, we discuss issues that could complicate the success of these approaches, such as toxicity or nonsense-mediated mRNA decay. Ultimately, these therapies have the potential to be uniquely tailored to a particular patient to optimize the therapeutic effect. Based upon recent progress in this field, one or more of these therapeutic recoding strategies could be used to treat individuals with one or more genetic diseases within the next few years.

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Authors and Affiliations

  1. Department of Microbiology and Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, 35294-2170, Birmingham, AL, USA

    Kim M. Keeling

  2. Department of Microbiology, The University of Alabama at Birmingham, BBRB 432/Box 8, 1530 Third Avenue, South, 35294-2170, Birmingham, AL, USA

    David M. Bedwell

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Correspondence to David M. Bedwell .

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Editors and Affiliations

  1. Molecular Biology Program, University of Utah, N. 2030 E. 15, Salt Late City, 84112-5330, U.S.A.

    John F. Atkins

  2. Dept. Bioengineering, University of Utah, Salt Lake City, 84112, U.S.A.

    Raymond F. Gesteland

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Keeling, K.M., Bedwell, D.M. (2010). Recoding Therapies for Genetic Diseases. In: Atkins, J., Gesteland, R. (eds) Recoding: Expansion of Decoding Rules Enriches Gene Expression. Nucleic Acids and Molecular Biology, vol 24. Springer, New York, NY. https://doi.org/10.1007/978-0-387-89382-2_6

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