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 an oligomer which efficiently enables to cause skipping of the 53rd exon in the human dystrophin gene. Also provided is a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency.
Claims18
A method of treating Duchenne muscular dystrophy (DMD) in a patient in need thereof comprising administering an antisense oligomer, or a pharmaceutically acceptable salt or hydrate thereof, wherein the antisense oligomer consists of a nucleotide sequence complementary to the sequence consisting of the 36th to the 56th nucleotides from the 5′ end of the 53rd exon in a human dystrophin pre-mRNA, and wherein the 53rd exon in the human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO:
- 1.
A method of treating Duchenne muscular dystrophy (DMD) in a patient in need thereof comprising administering a pharmaceutical composition comprising as an active ingredient an antisense oligomer, or a pharmaceutically acceptable salt or hydrate thereof, wherein the antisense oligomer consists of a nucleotide sequence complementary to the sequence consisting of the 36th to the 56th nucleotides from the 5′ end of the 53rd exon in a human dystrophin pre-mRNA, and wherein the 53rd exon in the human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.