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US 11,236,333 B2Granted
Antisense oligonucleotides for the treatment of Stargardt disease
AssigneeStichting Katholieke Universiteit
InventorsRobert Wilhelmus Johanna Collin, Alejandro Garanto Iglesias, Franciscus Peter Maria Cremers, Silvia Albert
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Abstract
The present invention relates to the field of medicine. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Stargardt disease.
Claims12
An antisense oligonucleotide for redirecting splicing that is:
- complementary or substantially complementary to a polynucleotide with a nucleotide sequence consisting of SEQ ID NO:
- 10, 30, or a part thereof;
- and wherein the antisense oligonucleotide comprises a 2′-O alkyl phosphorothioate antisense oligonucleotide, such as 2′-O-methyl modified ribose (RNA), 2′-O-ethyl modified ribose, 2′-O-propyl modified ribose, and/or substituted derivatives of these modifications such as halogenated derivatives.
Dependencies are inferred from the claim text.