Claims for Patent: 9,079,934

Summary for Patent: 9,079,934

Title:	Antisense nucleic acids
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.
Inventor(s):	Watanabe; Naoki (Tsukuba, JP), Satou; Youhei (Tsukuba, JP), Takeda; Shin'ichi (Tokyo, JP), Nagata; Tetsuya (Tokyo, JP)
Assignee:	NIPPON SHINYAKU CO., LTD. (Kyoto-shi, Kyoto, JP) NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY (Kodaira-shi, Tokyo, JP)
Application Number:	13/819,520
Patent Claims:	1. An antisense oligomer which causes skipping of the 53rd exon in the human dystrophin gene, consisting of the nucleotide sequence of SEQ ID NO: 35, wherein the antisense oligomer is an oligonucleotide having the sugar moiety and/or the phosphate-binding region of at least one nucleotide constituting the oligonucleotide modified, or a morpholino oligomer. 2. The antisense oligomer according to claim 1, wherein the antisense oligomer is a morpholino oligomer. 3. The antisense oligomer according to claim 1, wherein the sugar moiety of at least one nucleotide constituting the oligonucleotide is a ribose in which the 2'-OH group is replaced by any one selected from the group consisting of OR, R, R'OR, SH, SR, NH.sub.2, NHR, NR.sub.2, N.sub.3, CN, F, Cl, Br and I (wherein R is an alkyl or an aryl and R' is an alkylene). 4. The antisense oligomer according to claim 1, wherein the phosphate-binding region of at least one nucleotide constituting the oligonucleotide is any one selected from the group consisting of a phosphorothioate bond, a phosphorodithioate bond, an alkylphosphonate bond, a phosphoramidate bond and a boranophosphate bond. 5. The antisense oligomer according to claim 2, wherein the morpholino oligomer is a phosphorodiamidate morpholino oligomer. 6. The antisense oligomer according to claim 2, wherein the 5 end of the morpholino oligomer is one of the groups of chemical formulae (1) to (3) below: ##STR00024## 7. A pharmaceutical composition for the treatment of muscular dystrophy, comprising as an active, ingredient the antisense oligomer according to claim 1, or a pharmaceutically acceptable salt or hydrate thereof.