Antisense Oligonucleotide for Targeting Progranulin
AssigneeHoffmann-La Roche Inc.
InventorsRavi Jagasia, Lars Jonson, Soren Rasmussen, Jacob Ravn, Disa Tehler, Dorthe Vang, Jesper Worm
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Abstract
The present invention relates to oligonucleotides which alter the splicing pattern of progranulin in cells, and their use in the treatment of neurological disorders.
Claims18
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.
An antisense oligonucleotide having the structure:
An antisense oligonucleotide wherein the oligonucleotide is the oligonucleotide compound GaGctGggTcAagAAT (SEQ ID NO:
- 71), GgtCaaGaAtgGtgTG (SEQ ID NO:
- 73), CaGaAtGgtGtGgTC (SEQ ID NO:74), GaAtGgtGtGgTccC (SEQ ID NO:75) or CtcAagCtcAcAtgGC (SEQ ID NO:134) wherein capital letters represent beta-D-oxy LNA nucleosides, lowercase letters represent DNA nucleosides, all LNA C are 5-methyl cytosine, and all internucleoside linkages are phosphorothioate internucleoside linkages.
Dependencies are inferred from the claim text.