Design and In Vitro Use of Antisense Oligonucleotides to Correct Pre-mRNA Splicing Defects in Inherited Retinal Dystrophies.
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DOI: 10.1007/978-1-4939-7522-8_5
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… read more here.
Keywords: correct pre; retinal dystrophies; mrna splicing; pre mrna ... See more keywords