Antisense oligonucleotides for the treatment of Stargardt disease
AssigneeProQR Therapeutics II B.V.
InventorsAliye Seda Yilmaz-Elis, Peter Adamson, Kalyana Chakravarthi Dulla, Iris Antoinette Ernestine Schulkens
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Abstract
The invention relates to the fields of medicine and biotechnology. In particular, it relates to novel antisense oligonucleotides (AONs) that may be used in the treatment, prevention and/or delay of Stargardt disease and/or ABCA4-associated eye disease. More in particular, the invention relates to AONs that are used in inhibiting or blocking exon 39 skipping in the human ABCA4 pre-mRNA.
Claims5
An antisense oligonucleotide (AON) that is able to inhibit skipping of at least one exon in human ABCA4 pre-mRNA, wherein the exon skipping is due to a c.5461-10T>C mutation in intron 38 of the human ABCA4 gene, wherein said AON comprises or consists of the sequence of any of SEQ ID NO:
- 12, 31 and 32, wherein said AON has at least one phosphorothioate linkage.
A viral vector expressing antisense oligonucleotide (AON) that is able to inhibit skipping of at least one exon in human ABCA4 pre-mRNA, wherein the exon skipping is due to a c.5461-10T>C mutation in intron 38 of the human ABCA4 gene, wherein said AON comprises or consists of the sequence of any of SEQ ID NO:
- 12, 31 and 32.
Dependencies are inferred from the claim text.