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US 2026/0103714 A1Published application
Antisense oligonucleotides for the treatment of canavan disease
OwnerTue Fryland, Marianne Lerbech Jensen +1inventors
InventorsTue Fryland, Marianne Lerbech Jensen, Søren Vestergaard Rasmussen
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Abstract
The present invention relates to antisense oligonucleotides (oligomers) complementary to NAT8L pre-mRNA and mRNA and which are capable of inhibiting the expression of NAT8L. Inhibition of NAT8L expression is beneficial for the treatment of Canavan disease.
Claims33
An antisense oligonucleotide comprising a stretch of at least 10 nucleotides which is at least 90% complementary to a target sequence in the human NAT8L (N-acetyltransferase 8 like) gene.
Dependencies are inferred from the claim text.