Antisense nucleic acids
AssigneesNippon Shinyaku Co., Ltd., National Center of Neurology and Psychiatry
InventorsNaoki WATANABE, Youhei SATOU, Shin'ichi TAKEDA, Tetsuya NAGATA
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Abstract
The present invention provides a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency. The present invention provides an oligomer which efficiently enables to cause skipping of the 53rd exon in the human dystrophin gene.
Claims12
An antisense oligomer which causes skipping of the 53rd exon in the human dystrophin gene, consisting of a nucleotide sequence complementary to any one of the sequences consisting of the 31st to the 55th, the 32nd to the 53rd, the 32nd to the 56th, the 33rd to the 54th, the 34th to the 58th, the 36th to the 53rd, the 36th to the 55th, the 36th to the 57th, or the 33rd to the 57th nucleotides from the 5′ end of the 53rd exon in the human dystrophin gene.
2-14. (canceled)
Dependencies are inferred from the claim text.