T
Toshifumi Yokota
Researcher at University of Alberta
Publications - 156
Citations - 5315
Toshifumi Yokota is an academic researcher from University of Alberta. The author has contributed to research in topics: Duchenne muscular dystrophy & Exon skipping. The author has an hindex of 36, co-authored 131 publications receiving 4332 citations. Previous affiliations of Toshifumi Yokota include Children's National Medical Center & University of Toronto.
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Eteplirsen in the treatment of Duchenne muscular dystrophy
TL;DR: Eteplirsen is a drug that acts to promote dystrophin production by restoring the translational reading frame of DMD through specific skipping of exon 51 in defective gene variants, which is applicable for approximately 14% of patients with DMD mutations.
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Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
Qi Long Lu,Adam Rabinowitz,Yun Chao Chen,Toshifumi Yokota,HaiFang Yin,Julia Alter,Atif Jadoon,George Bou-Gharios,Terence A. Partridge +8 more
TL;DR: It is shown that systemic delivery of specific 2OMeAOs, together with the triblock copolymer F127, induced dystrophin expression in all skeletal muscles but not in cardiac muscle of the mdx dystrophic mice, suggesting that a significant therapeutic effect may be achieved by further optimization in dose and regime of administration of antisense oligonucleotide.
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Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs.
Toshifumi Yokota,Qi-Long Lu,Terence A. Partridge,Masanori Kobayashi,Akinori Nakamura,Shin'ichi Takeda,Eric P. Hoffman,Eric P. Hoffman +7 more
TL;DR: This work sought to test efficacy and toxicity of intravenous oligonucleotide (morpholino)‐induced exon skipping in the DMD dog model and found it safe and effective.
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Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy
Mariko Taniguchi-Ikeda,Kazuhiro Kobayashi,Motoi Kanagawa,Chih Chieh Yu,Kouhei Mori,Tetsuya Oda,Atsushi Kuga,Hiroki Kurahashi,Hasan O. Akman,Salvatore DiMauro,Ryuji Kaji,Toshifumi Yokota,Shin'ichi Takeda,Tatsushi Toda +13 more
TL;DR: It is shown that aberrant messenger RNA (mRNA) splicing, induced by SVA exon-trapping, underlies the molecular pathogenesis of FC MD and the promise of splicing modulation therapy as the first radical clinical treatment for FCMD and other SVA-mediated diseases is demonstrated.
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Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery
Yoshitsugu Aoki,Toshifumi Yokota,Tetsuya Nagata,Akinori Nakamura,Jun Tanihata,Takashi Saito,Stephanie Duguez,Stephanie Duguez,Kanneboyina Nagaraju,Kanneboyina Nagaraju,Eric P. Hoffman,Eric P. Hoffman,Terence A. Partridge,Terence A. Partridge,Shin'ichi Takeda +14 more
TL;DR: A unique successful demonstration of effective rescue by exon 45–55 skipping in a dystrophin-deficient animal model is demonstrated.