Antisense oligonucleotides for targeting progranulin
AssigneeHoffmann-La Roche Inc.
InventorsJesper WORM, Lars JOENSON, Mads MANSØ, Dorthe VANG
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Abstract
Antisense oligonucleotides for altering the splicing pattern of progranulin, and their use in the treatment of neurological disorders. The antisense oligonucleotides are modified to better increase up-regulation or expression restoration of the Exon1-Exon2 progranulin splice variant in cells.
Claims23
An antisense oligonucleotide, wherein the antisense oligonucleotide is 8-40 nucleotides in length and comprises a contiguous nucleotide sequence of 8-40 nucleotides in length which is complementary to a splice regulation site of the human progranulin pre-mRNA transcript, wherein the contiguous nucleotide sequence comprises one or more methanesulfonyl phosphoramidate internucleotide linkages.
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