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• W47643599 abstract "The majority of eukaryotic genes undergo alternative splicing, an evolutionarily conserved phenomenon, to generate functionally diverse protein isoforms from a single transcript. The fact that defective pre-mRNA splicing can generate non-functional and often toxic proteins with catastrophic effects, accurate removal of introns and joining of exons is vital for cell homeostasis. Thus, molecular tools that could either silence a disease-causing gene or regulate its expression in trans will find many therapeutic applications. Here we present two RNA-based approaches, namely RNAi and theophylline-responsive riboswitch that can regulate gene expression by loss-of-function and modulation of splicing, respectively. These strategies are likely to continue to play an integral role in studying gene function and drug discovery." @default.
• W47643599 created "2016-06-24" @default.
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• W47643599 date "2009-01-01" @default.
• W47643599 modified "2023-10-01" @default.
• W47643599 title "Alternative Splicing as a Therapeutic Target for Human Diseases" @default.
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• W47643599 doi "https://doi.org/10.1007/978-1-60327-295-7_10" @default.
• W47643599 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/3076216" @default.
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