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
Life of this patent
Estimating the term…
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.
Claims21
A method for modulating splicing of ABCA4 pre-mRNA in a cell, said method comprising contacting said cell with an antisense oligonucleotide (AON) comprising or consisting of the sequence of any one of SEQ ID NO:
- 12, 31 and 32, wherein the AON has at least one phosphorothioate linkage.
A method for the treatment of Stargardt disease comprising contacting a cell of an individual having Stargardt disease with an antisense oligonucleotide (AON) comprising or consisting of the sequence of any one of SEQ ID NO:
- 12, 31 and 32, wherein the AON has at least one phosphorothioate linkage.
A method for modulating splicing of ABCA4 pre-mRNA in a cell, said method comprising contacting said cell with a viral vector encoding an antisense oligonucleotide (AON) comprising or consisting of the sequence of any one of SEQ ID NO:
- 12, 31 and 32.
A method for the treatment of Stargardt disease comprising contacting a cell of an individual having Stargardt disease with a viral vector encoding an antisense oligonucleotide (AON) comprising or consisting of the sequence of any one of SEQ ID NO:
- 12, 31 and 32.
A method for modulating splicing of ABCA4 pre-mRNA of an individual in need thereof, said method comprising contacting a cell of said individual with an antisense oligonucleotide (AON) comprising or consisting of the sequence of any one of SEQ ID NO:
- 12, 31 and 32, wherein the AON has at least one phosphorothioate linkage.
A method for modulating splicing of ABCA4 pre-mRNA of an individual in need thereof, said method comprising contacting a cell of said individual with a viral vector encoding an antisense oligonucleotide (AON) comprising or consisting of the sequence of any one of SEQ ID NO:
- 12, 31 and 32.
Dependencies are inferred from the claim text.