abstract |
The present invention relates to RNA-edited oligonucleotides capable of resulting in specific editing of a target nucleotide (adenosine) in a target RNA molecule of eukaryotic cells, wherein said oligonucleotide is for use in the treatment of Asher syndrome. , And more preferably for deamination of the target adenosine, which is part of the immature arrest codon present in the USH2A pre-mRNA or USH2A mRNA. |