Antisense nucleic acid

Antisense oligomers targeting exon 53 in the dystrophin gene induce high-efficiency exon skipping, addressing the lack of effective treatments for DMD by restoring dystrophin protein production and stabilizing muscle cells, thus alleviating DMD symptoms.

JP2024170458AInactive Publication Date: 2024-12-10NIPPON SHINYAKU CO LTD +1
4 Cites -1 Cited by

Patent Information

Application Number
JP2024145055
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2010-09-01
Filing Date
2024-08-27
Publication Date
2024-12-10
Estimated Expiration
Not applicable · inactive patent
Patent Text Reader

Abstract

To provide an agent which causes skipping of the 53rd exon in the human dystrophin gene with high efficiency.SOLUTION: The present invention provides an antisense oligomer which enables skipping of the 53rd exon in the human dystrophin gene. The antisense oligomer consists of a base sequence complementary to any one of specific sequences of the 53rd exon in the human dystrophin gene.SELECTED DRAWING: None
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Citation Information

Patent Citations

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  • ENA NUCLEIC ACID DRUGS MODIFYING SPLICING IN mRNA PRECURSOR

    WO2004048570A1

  • Antisense oligonucleotides for inducing exon skipping and methods of use thereof

    WO2006000057A1

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