Toshifumi Yokota

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Toshifumi Yokota

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Born|

Japan

Nationality| Japanese
Title| Professor
Academic background
Education| University of Tokyo
Academic work
Institutions| Imperial College London

National Center of Neurology and Psychiatry Children's National Medical Center

University of Alberta

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].

## Contents

* 1 Biography
* 2 Major contributions
* 3 Honors
* 4 References
* 5 External links

## 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]

1. ↑ "Seeking a cure for muscular dystrophy". WCHRI. 2020-05-16. Retrieved 2021-08-17.
2. ↑ O'Byrne, Ryan. "FDA approves new drug to treat common form of muscular dystrophy based on research from U of A scientist". www.ualberta.ca. Retrieved 2021-08-17.
3. ↑ "Researchers find DNA therapy could treat patients with DMD". Drug Target Review. Retrieved 2021-08-18.
4. ↑ "Profile | Faculty of Medicine & Dentistry". www.ualberta.ca. Retrieved 2021-08-17.
5. ↑ Yokota, T.; et al. (2019). Gapmers. Springer Nature. doi:10.1007/978-1-0716-0771-8. ISBN 978-1-0716-0770-1.
6. ↑ Yokota, T.; et al. (2017). Exon skipping and Inclusion Therapies. Springer Nature. doi:10.1007/978-1-4939-8651-4. ISBN 978-1-4939-8650-7.
7. ↑ "International Journal of Molecular Sciences". www.mdpi.com. Retrieved 2021-08-18.
8. ↑ "Genes". www.mdpi.com. Retrieved 2021-08-18.
9. ↑ "Frontiers in Genome Editing". www.frontiersin.org. Retrieved 2021-08-18.
10. ↑ "Loop | Toshifumi Yokota". loop.frontiersin.org. Retrieved 2021-08-18.
11. ↑ "Toshifumi Yokota | Medical Genetics". www.ualberta.ca. Retrieved 2021-08-17.
12. ↑ 4581857\. "HEROES - Spring 2016". Issuu. Retrieved 2021-08-17.`{{cite web}}`: CS1 maint: numeric names: authors list (link)
13. ↑ 4581857\. "HEROES - Spring 2016". Issuu. Retrieved 2021-08-17.`{{cite web}}`: CS1 maint: numeric names: authors list (link)
14. ↑ "Profile | Faculty of Medicine & Dentistry". www.ualberta.ca. Retrieved 2021-08-17.
15. ↑ "Profile | Faculty of Medicine & Dentistry". www.ualberta.ca. Retrieved 2021-08-17.
16. ↑ "Edmonton researchers at the University of Alberta have found a way to regrow a protien missing in Duchenne muscular dystrophy in mice". edmontonsun. Retrieved 2021-08-18.
17. ↑ "University of Alberta". Department of Medical Genetics. Retrieved July 24, 2021.
18. ↑ Yokota, T.; et al. (2009-06-01). "Efficacy of systemic morpholino exon‐skipping in Duchenne dystrophy dogs". Annals of Neurology. 65 (6): 667–676. doi:10.1002/ana.21627. PMC 5951302. PMID 19288467.
19. ↑ "First treatment for muscular dystrophy in sight: Scientists successfully harness exon-skipping". EurekAlert!. Retrieved 2021-08-17.
20. ↑ "Researchers Develop DNA "Patch" For Canine Form of Muscular Dystrophy". National Institutes of Health (NIH). 2015-08-28. Retrieved 2021-08-17.
21. ↑ Roshmi, R.; et al. (2019-10-01). "Viltolarsen for the treatment of Duchenne muscular dystrophy". Drugs of Today. 55 (10): 627–639. doi:10.1358/dot.2019.55.10.3045038. PMID 31720560.
22. ↑ O'Byrne, Ryan. "FDA approves new drug to treat common form of muscular dystrophy based on research from U of A scientist". www.ualberta.ca. Retrieved 2021-08-17.
23. ↑ Zahid, Hina (2020-07-15). "New promising treatment for common form of muscular dystrophy". medicaldialogues.in. Retrieved 2021-08-18.
24. ↑ Lim, Kenji Rowel Q.; Maruyama, Rika; Echigoya, Yusuke; Nguyen, Quynh; Zhang, Aiping; Khawaja, Hunain; Sen Chandra, Sreetama; Jones, Takako; Jones, Peter; Chen, Yi-Wen; Yokota, Toshifumi (2020-07-14). "Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy". Proceedings of the National Academy of Sciences. 117 (28): 16509–16515.
25. ↑ "Seeking a cure for muscular dystrophy". WCHRI. 2020-05-16. Retrieved 2021-08-17.
26. ↑ "Federal RePORTER - Project Details". federalreporter.nih.gov. Retrieved 2021-08-18.

## External links[edit]

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