Oligonucleotides targeting gal3st1
OwnerSøren Vestergaard Rasmussen, Peter Hagedorn +2inventors
InventorsSøren Vestergaard Rasmussen, Peter Hagedorn, Marianne Lerbech Jensen, Tue Fryland
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Abstract
The present invention relates to antisense oligonucleotides (oligomers) complementary to GAL3ST1 pre-mRNA sequences, which are capable of inhibiting the expression of GAL3ST1. Inhibition of GAL3ST1 expression is expected to be beneficial for the treatment of metachromatic leukodystrophy.
Claims29
An antisense oligonucleotide comprising a stretch of at least 10 nucleotides which is at least 90% complementary to a target sequence in the human GAL3ST1 gene (Galactosylceramide sulfotransferase), such as a target sequence selected from the target sequences shown in Table B1, B2 or B3.
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