Proqr Therapeutics Ii B.v.

University of Rochester

Lenka Van Sint Fiet

Janne Juha Turunen

Bart Klein

Julien Auguste Germain Boudet

Antti Aalto

Roxana Simona Redis

Petra Geziena De Bruijn

Yi-Tao Yu

Kalyana Chakravarthi Dulla

Pedro Duarte Morais Fernandes Arantes Da Silva

Meemanage De Zoysa

Hironori Adachi

Jim Swildens

The invention relates to RNA editing oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA molecule in a eukaryotic cell, wherein said oligonucleotide is for use in the treatment of Usher syndrome, and more preferably for the deamination of target adenosines that are part of a premature stop codon present in the USH2A pre-mRNA or USH2A mRNA.

RNA-editing oligonucleotides for the treatment of usher syndrome

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

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 hairpin 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.

Single-stranded RNA-editing oligonucleotides

The invention relates to nucleic acid molecules for pseudouridylation of a target uridine in a target RNA in a mammalian cell, wherein the nucleic acid molecule comprises a guide region capable of forming a partially double stranded nucleic acid complex with the target RNA comprising the target uridine, wherein the partially double stranded nucleic acid complex is capable of engaging a mammalian pseudouridylation enzyme, wherein the guide region assists in positioning the target uridine in the partially double stranded nucleic acid complex for it to be converted to a pseudouridine by the mammalian pseudouridylation enzyme.

Nucleic acid molecules for pseudouridylation

Chemically modified single-stranded RNA-editing oligonucleotides

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 hairpin or stem-loop structure, and wherein said oligonucleotide comprises a cytidine (a non-complementary nucleotide) or a uridine in position opposite to the target adenosine to be edited in the target RNA region.

Growing community of inventors

Leiden, Netherlands

Lenka Van Sint Fiet