Toshifumi Yokota

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Toshifumi Yokota is a medical scientist and Professor of medical genetics at the University of Alberta, holding the title of the Friends of Garrett Cumming Research & Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair^[1]. He is best known for pioneering studies of antisense oligonucleotide-based therapeutics for muscular dystrophy^[2][3]. His research interests include precision health and personalized genetic medicine for muscular dystrophy and genetic diseases^[4]. He edited books “Gapmers” and “Exon skipping and Inclusion Therapies”, both published from Springer Nature^{[5] [6]}. He serves as a member of editorial board for the International Journal of Molecular Sciences, Genes, and Frontiers in Genome Editing^{[7][8][9][10]}.

Biography[edit]

Yokota was born in Morioka, a city in Iwate Prefecture|Iwate Prefecture, and raised in several cities including Tsu, Mie and Nerima, Tokyo, in Japan^[11][12]. He was initially interested in astrophysics; however, his interests changed after taking a genetics class and hearing about gene therapy research on muscular dystrophy at the University of Tokyo^[13]. After receiving research training at the University of Tokyo and the National Center of Neurology and Psychiatry, he received his Ph.D. in Biological science|Biological Science, where he studied the mechanisms underlying muscle regeneration^[14]. He completed his post-doctoral training at Imperial College London, Hammersmith Hospital Campus^[15]. He was a Research Associate at Children's National Medical Center before joining the University of Alberta^[16]. Currently, he is a tenured Professor at the University of Alberta Faculty of Medicine and Dentistry, serving as the Friends of Garrett Cumming Research & Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair since 2011^[17].

Major contributions[edit]

His study demonstrated therapeutic effects of synthetic antisense oligonucleotides, which can restore the function of a mutated gene by modulating pre-mRNA RNA splicing|splicing, called exon skipping, and improvement of muscle function for the first time in a severe animal model of Duchenne muscular dystrophy (DMD)^[18][19][20]. Based on his study, viltolarsen, a phosphorodiamidate morpholino oligomer antisense oligonucleotide, was developed for the treatment of DMD in collaboration with a Japanese pharmaceutical company Nippon Shinyaku (also known as NS Pharma), which was later approved in Japan and by the Food and Drug Administration|FDA in the United States in 2020 after clinical trials in Japan, Canada, and the United States^[21][22]. In 2020, he identified antisense oligonucleotides that knockdown the expression of a toxic gene called DUX4 for the treatment of facioscapulohumeral muscular dystrophy^[23][24].

Honors[edit]

• The Friends of Garrett Cumming Research & Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair (2011-)^[25]
• National Institutes of Health Ruth L. Kirschstein National Research Service Award (2010) ^[26]

References[edit]

External links[edit]

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