Abstract: The invention relates to the fields of medicine and to antisense oligonucleotides (AONs) that are capable of skipping exon 50 from human USH2A pre-mRNA and that may be used in the treatment, prevention and/or delay of Usher syndrome type II and/or USH2A-associated non syndromic retina degeneration.

Abstract: The invention relates to RNA editing oligonucleotides (EONs) that can bring 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 Stargardt disease, and more preferably for the deamination of target adenosines present in the ABCA4 pre-mRNA or ABCA4 mRNA.

Abstract: The invention relates to the fields of medicine. In particular, it relates to novel antisense oligonucleotides (AONs) that are capable of skipping exon 62 from human USH2A premRNA and that may be used in the treatment, prevention and/or delay of Usher syndrome type II and/or USH2A-associated non syndromic retina degeneration.

Abstract: The invention relates to antisense oligonucleotides (AONs) and the use thereof in the treatment of human disease. In particular the present invention is concerned with AONs suitable for the treatment of Dystrophic Epidermolysis Bullosa (DEB). More specifically, the invention relates to AONs that are capable of inducing exon 105 skipping from human COL7A1 pre-mRNA and that outperform—in exon 105 skipping efficiency—the AONs known from the prior art. The invention further relates to the use of these new AONs in the treatment of DEB in a patient carrying a mutation in exon 105 of the COL7A1 gene.

Abstract: 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.

Abstract: The invention relates to antisense oligonucleotides (AON) capable of inducing the skip of exon 36 from human CEP290 pre-mRNA. The c.4723A>T mutation in the human CEP290 gene is the cause of Leber's Congenital Amaurosis type 10 (LCA10) in patients carrying this mutation. The AONs of the present invention can be used in the treatment of LCA10 caused by mutations in exon 36, such as the c.4723A>T mutation. The invention relates to AONs, pharmaceutical formulations comprising such AONs, and viral vectors expressing such AONs, that may be used in the treatment of LCA10.

Abstract: A method for making a change in an endogenous chromosomal DNA sequence of a mammalian cell, comprising steps of: (i) introducing into said cell an oligonucleotide having a sequence that is complementary to the chromosomal DNA sequence and that includes the change; (ii) allowing sufficient time for the cell to incorporate the change into the endogenous chromosomal DNA sequence through endogenous nucleic acid modifying pathways; and (iii) identifying the presence of the change in the chromosomal DNA sequence. The invention is particularly useful for correcting mutations in the CFTR gene.