Recoding Therapies for Genetic Diseases
作者： Kim M. Keeling , David M. Bedwell
DOI: 10.1007/978-0-387-89382-2_6
关键词:
摘要: Strategies aimed at recoding premature stop mutations or frameshift have the potential to treat a genotypic subset of patients afflicted with many different genetic diseases. In this chapter we provide an overview approaches promote readthrough mutations, including pharmacological agents and suppressor tRNAs. We also describe use oligonucleotides induce differential splicing exclude disease-causing site-specific frameshifting as method that alter ribosomal reading frame. Finally, discuss issues could complicate success these approaches, such toxicity nonsense-mediated mRNA decay. Ultimately, therapies be uniquely tailored particular patient optimize therapeutic effect. Based upon recent progress in field, one more strategies used individuals diseases within next few years.
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