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US 2019/0194660 A1Published application
Inhibitors of srsf1 to treat neurodegenerative disorders
AssigneeThe University of Sheffield
InventorsGuillaume Hautbergue, Mimoun Azzouz, Alexander Whitworth, Pamela Shaw
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Abstract
The present disclosure relates to antagonists that target Serine/Arginine-Rich Splicing Factor 1 (SRSF1); expression vectors comprising SRSF1 antagonists; and the use of such antagonists in therapy for the treatment of neurodegenerative disorders and cancer and screening methods that identify agents that inhibit the expression or activity of SRSF1.
Claims34
An expression vector comprising a transcription cassette comprising:
- a nucleic acid molecule encoding an antagonistic agent wherein said agent inhibits the expression of a nucleic acid encoding a Serine/Arginine-Rich Splice Factor [SRSF1] or inhibits the activity of a SRSF1 protein wherein said nucleic acid molecule is operably linked to a promoter adapted to express said antagonistic agent.
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The vector according to claim 34 wherein said inhibitory RNA is a siRNA or shRNA molecule.
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Dependencies are inferred from the claim text.