Proqr Therapeutics Ii B.v.

Antti Aalto

Janne Juha Turunen

Bart Klein

Lenka Van Sint Fiet

Julien Auguste Germain Boudet

The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA sequence in a eukaryotic cell, wherein said oligonucleotide does not, in itself, form an intramolecular hairpin or stem-loop structure, and wherein said oligonucleotide comprises a non-complementary nucleotide in a position opposite to the nucleotide to be edited in the target RNA sequence.

Chemically modified single-stranded rna-editing oligonucleotides

Chemically modified single-stranded RNA-editing oligonucleotides

The invention relates to double stranded oligonucleotide complexes comprising an antisense oligonucleotide (AON) and a complementary sense oligonucleotide (SON), for use in the deamination of a target adenosine in a sense target RNA sequence in a cell by an ADAR enzyme, wherein at least the nucleotide in the AON that is directly opposite the target adenosine in the target RNA sequence does not have a 2'-O-alkyl modification and the SON comprises nucleotides that are at least complementary to all nucleotides in the AON that do not have a 2&#x2032;-O-alkyl modification. The invention further relates to methods of RNA editing using the AON/SON complexes of the invention.

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Leiden, Netherlands

Antti Aalto