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Recoding Therapies for Genetic Diseases

  • Kim M. Keeling
  • David M. Bedwell
Chapter
Part of the Nucleic Acids and Molecular Biology book series (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.

Keywords

Stop Codon Duchenne Muscular Dystrophy Antisense Oligonucleotide Premature Stop Codon Duchenne Muscular Dystrophy 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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© Springer Science+Business Media, LLC 2010

Authors and Affiliations

  1. 1.Department of Microbiology and Gregory Fleming James Cystic Fibrosis Research CenterUniversity of Alabama at BirminghamBirminghamUSA
  2. 2.Department of MicrobiologyThe University of Alabama at BirminghamBirminghamUSA

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