Single-stranded RNA-editing oligonucleotides
AssigneeProQR Therapeutics II B.V.
InventorsJanne Juha Turunen, Petra Geziena De Bruijn, Bart Klein, Roxana Simona Redis, Lenka Van Sint Fiet
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Abstract
The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA in a eukaryotic cell, wherein said oligonucleotide does not, in itself, form an intramolecular hair-pin or stem-loop structure, and wherein said oligonucleotide comprises a cytidine (a non-complementary nucleotide) or a uridine in a position opposite to the target adenosine to be edited in the target RNA region.
Claims12
An antisense oligonucleotide (AON) capable of forming a double stranded complex with an alpha-L-iduronidase (IDUA)-encoding target RNA in a cell for the deamination of a target adenosine present in the target RNA by an ADAR enzyme naturally present in the cell, wherein:
- a) the AON is complementary to a target RNA region comprising the target adenosine;
- b) the nucleotide in the AON that is opposite the target adenosine comprises a deoxyribose with a 2′-H group;
- and
- c) the AON does not comprise a portion that is both:
- (i) non-complementary to the target RNA region, and (ii) capable of forming an intramolecular stem-loop structure.
Dependencies are inferred from the claim text.