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US 10,329,319 B2Granted
Antisense nucleic acids
AssigneesNippon Shinyaku Co., Ltd., National Center of Neurology and Psychiatry
InventorsNaoki Watanabe, Youhei Satou, Shin'ichi Takeda, Tetsuya Nagata
Life of this patent
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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.
Claims7
An antisense oligomer consisting of any one nucleotide sequence selected from the group consisting of SEQ ID NOS:
- 8, 15, 32, 34, and 36, or pharmaceutically acceptable salt or hydrate thereof,
- wherein the antisense oligomer is a morpholino oligomer, a peptide nucleic acid (PNA), or an oligonucleotide comprising at least one nucleotide having:
- (i) a modified sugar moiety, wherein the 2′—OH group of a ribose is replaced by any one selected from the group consisting of R, R′OR, SH, SR, NH2, NHR, NR2, N3, CN, F, Cl, Br and I (wherein R is an alkyl or an aryl and R′ is an alkylene), or
- (ii) a modified phosphate-binding region selected from the group consisting of a phosphorothioate bond, a phosphorodithioate bond, an alkylphosphonate bond, a phosphoramidate bond, and a boranophosphate bond.
1 more element
Dependencies are inferred from the claim text.