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Antisense oligonucleotides (AONs) are small molecules able to bind to the pre-mRNA and modulate splicing. The increasing amount of intronic mutations leading to pseudoexon insertion in genes underlying inherited retinal… Click to show full abstract
Antisense oligonucleotides (AONs) are small molecules able to bind to the pre-mRNA and modulate splicing. The increasing amount of intronic mutations leading to pseudoexon insertion in genes underlying inherited retinal dystrophies (IRDs) has highlighted the potential of AONs as a therapeutic tool for these disorders. Here we describe how to design and test AON molecules in vitro in order to correct pre-mRNA splicing defects involved in IRDs.
Keywords:
correct pre;
retinal dystrophies;
mrna splicing;
pre mrna;
inherited retinal;
antisense oligonucleotides
Journal Title: Methods in molecular biology
Year Published: 2018
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Journal Title: Methods in molecular biology
Year Published: 2018
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!