Other

Daiichi Sankyo Company, Limited

Orphan Disease Treatment Institute Co., Ltd.

Knc Laboratories Co., Ltd.

Nonprofit Organization Translational Research Organization of Duchenne Muscular Dystrophy

Takeshima, Yasuhiro

Matsuo, Masafumi

Yasuhiro Takeshima

Masafumi Matsuo

Makoto Koizumi

Oligonucleotides having a nucleotide sequence complementary to nucleotide numbers such as 2571-2607, 2578-2592, 2571-2592, 2573-2592, 2578-2596, 2578-2601 or 2575-2592 of the dystrophin cDNA (Gene Bank accession No. NM_004006.1) and therapeutic agents for muscular dystrophy comprising such oligonucleotides.

ENA nucleic acid pharmaceuticals capable of modifying splicing of mRNA precursors

ENA nucleic acid drugs modifying splicing in mRNA precursor

Antisense oligonucleotides comprising a nucleotide sequence complementary to the nucleotide sequence of SEQ ID NO:1 or NO:2 are disclosed. The antisense oligonucleotides are used for treatment of specific types of Duchenne muscular dystrophy which is attributed to a change in number of the nucleotides composing one or more exons adjacent to exon 43 or 53, respectively, in human dystrophin mRNA, wherein the change is due to deletion of one or more nucleotides from the normal nucleotide sequence for the exons, wherein the net of the change in number of the nucleotides is expressed as a reduction of (3&times;N&plus;1) nucleotides, wherein N is zero or a natural number.

Medicament for treatment of Duchenne muscular dystrophy

Growing community of inventors

Nishinomiya, Japan

Yasuhiro Takeshima