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- W2768804540 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." @default.
- W2768804540 created "2017-12-04" @default.
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- W2768804540 date "2017-11-30" @default.
- W2768804540 modified "2023-10-18" @default.
- W2768804540 title "Design and In Vitro Use of Antisense Oligonucleotides to Correct Pre-mRNA Splicing Defects in Inherited Retinal Dystrophies" @default.
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- W2768804540 doi "https://doi.org/10.1007/978-1-4939-7522-8_5" @default.
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