Toshifumi Yokota Explained

Toshifumi Yokota
Native Name:横田俊文
Birth Place:Morioka, Japan
Education:University of Tokyo

Toshifumi (Toshi) Yokota (ja|横田俊文|Yokota Toshifumi) is a biomedical scientist and professor of medical genetics at the University of Alberta, holding the titles of the Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair and the Henri M. Toupin Chair in Neurological Science.[1] Yokota is widely recognized for pioneering work in antisense therapy for muscular dystrophy and other genetic diseases, which led to the development of viltolarsen, an FDA-approved treatment for Duchenne muscular dystrophy (DMD).[2] With over 100 peer-reviewed publications and several patents, Yokota has made significant contributions to the field of precision medicine. Yokota also co-edited three volumes in the Methods in Molecular Biology series by Humana Press, Springer-Nature and serves on editorial boards of multiple scientific journals. [3] [4] [5] [6] [7] [8] [9] [10] [11] [12]

In 2023, Yokota was elected as a fellow of the Canadian Academy of Health Sciences, an honor that recognizes outstanding achievements in health sciences. In addition, Yokota serves as Chief Scientific Officer at OligomicsTx, is a co-founder of the Canadian Neuromuscular Network (CAN-NMD), and contributes as a member of the Medical and Scientific Advisory Committee of Muscular Dystrophy Canada. [13] [14]

Biography

Yokota was born in Morioka, a city in Iwate Prefecture, and raised in multiple cities including Tsu, Mie and Nerima, Tokyo, in Japan.[15] [16] After completing a Ph.D. in Biological Science in 2003, further training was undertaken at Imperial College London, as a Research Fellow of the Japan Society for the Promotion of Science,[17] and as a Research Associate at the Children's National Medical Center before joining the University of Alberta in 2011.[18] Currently, Yokota 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 Endowed Research Chair and the Henri M. Toupin Chair in Neurological Science since 2011.[19] In 2022, Yokota received the Scientific Achievement and Innovation Award from BioAlberta. In 2023, Yokota was elected as a Fellow of the Canadian Academy of Health Sciences.[20]

Major contributions

Yokota's research focuses on precision health and personalized genetic medicine using single strands of artificial DNA/RNA-like molecules called antisense oligonucleotides for neuromuscular diseases.[21] Yokota demonstrated the therapeutic potential of antisense oligonucleotides for exon skipping, which can be designed to frame-disrupting exons and restore the reading frame and function of a mutated gene by modulating pre-mRNA splicing, leading to the improvement of skeletal muscle function accompanied by dystrophin restoration for the first time in a severe animal model of Duchenne muscular dystrophy (DMD).[22] [23] [24] Based on the study, viltolarsen, a phosphorodiamidate morpholino oligomer antisense oligonucleotide, was developed for the treatment of DMD in collaboration with a Japanese pharmaceutical company.[25] Viltolarsen was later approved by the Pharmaceuticals and Medical Devices Agency in Japan and by the FDA in the United States in March and August 2020, respectively, after clinical trials conducted in Japan, Canada, and the United States.[26] Yokota's team further developed a potential treatment for nearly half of DMD patients using multiple antisense oligonucleotides and demonstrated therapeutic effects in a dystrophic mouse model.[27] [28] Supported by the Heart and Stroke Foundation of Canada, the research team developed a cocktail of peptide-conjugated morpholinos (PPMOs) and restored expression of dystrophin in the myocardium and Purkinje fibers in the heart muscle of dystrophic animal models.[29] [30] [16] In 2021, the team developed eSkip-Finder, a machine learning-mediated free online application with a database of antisense oligonucleotides to facilitate the design of antisense oligonucleotides that can be used for exon skipping targeted for various genes and exons.[31] [32] [33]

Supported by the Canadian Institutes of Health Research and Muscular Dystrophy Canada, Yokota's team is also developing antisense oligonucleotide-mediated therapy for facioscapulohumeral muscular dystrophy using lipid nanoparticles in collaboration with Pieter Cullis.[34] [35] In 2020, they identified antisense oligonucleotides called gapmers that knock down the expression of a toxic gene called DUX4 in cell and mouse models for the treatment of facioscapulohumeral muscular dystrophy.[36] [37]

Yokota’s research identified ASO targets for skipping multiple dysferlin (DYSF) exons (26-27 and 28-29), which were shown to enhance membrane resealing function in patient cells—a therapeutic approach applicable to 5-8% of dysferlinopathy patients globally.[38]

In 2022, Yokota's team identified gapmers that selectively knocked down most of the mutated mRNA for the treatment of fibrodysplasia ossificans progressiva.[39] With support from CIHR and muscular dystrophy Canada, the team also identified novel peptide-conjugated morpholinos called DG9-PMOs that effectively improved mouse models of DMD and spinal muscular atrophy.[40]

Patient advocacy

In addition to research achievements, Yokota has significantly advanced patient advocacy in the realm of genetic disorders, particularly muscular dystrophy.[41] Advocacy efforts are strongly reflected in the integration of individuals affected by muscular dystrophy into the research team, which enhances the research approach with valuable firsthand insights. [42] This initiative aligns with Yokota’s broader dedication to equity, diversity, and inclusion (EDI), actively seeking to bring diverse perspectives into the scientific process.

Honors

Yokota is honored as a ScholarGPS Highly Ranked Scholar.[43] Yokota's publication record, impact of work, and quality of scholarly contributions have earned rankings of 2nd in Muscular dystrophy and 4th in Personalized medicine globally over the last five years, placing in the top 0.01 percent of highly ranked scholars.

Selected publications

External links

Notes and References

  1. Web site: 2020-10-09. FDA approves new drug to treat common form of muscular dystrophy based on research from University of Alberta . 2021-08-20. MirageNews.com. en-AU.
  2. Web site: Heroes - Spring 2016 . 2021-08-17 . Issuu . 5 May 2016 . en.
  3. Book: 2020. Yokota. Toshifumi. Maruyama . Rika. Gapmers. Methods in Molecular Biology. 2176. en-gb. 10.1007/978-1-0716-0771-8. 978-1-0716-0770-1 . 221372639 . 1064-3745 .
  4. Book: 2018. Yokota . Toshifumi. Maruyama . Rika. Exon Skipping and Inclusion Therapies . Methods in Molecular Biology . 1828 . en-gb. 10.1007/978-1-4939-8651-4. 978-1-4939-8650-7 . 52141624 . 1064-3745 .
  5. Book: 2023 . Maruyama . Rika . Yokota . Toshifumi . Muscular Dystrophy Therapeutics . Methods in Molecular Biology . 2587 . en . 10.1007/978-1-0716-2772-3 . 978-1-0716-2771-6 . 1064-3745.
  6. Web site: Toshifumi Yokota . 2021-08-19. scholar.google.com.
  7. Web site: Toshifumi Yokota elected 2023 Fellow of the Canadian Academy of Health Sciences . 2024-01-17 . www.ualberta.ca . en.
  8. Web site: International Journal of Molecular Sciences . 2021-08-18 . www.mdpi.com . en.
  9. Web site: Genes . 2021-08-18 . www.mdpi.com . en.
  10. Web site: Frontiers in Genome Editing . 2021-08-18 . www.frontiersin.org . en.
  11. Web site: Nucleic Acid Therapeutics Mary Ann Liebert, Inc., publishers . 2022-05-30 . home.liebertpub.com.
  12. Web site: Frontiers in Physiology . 2022-06-29 . www.frontiersin.org . en.
  13. Web site: 2019-07-24 . Medical and Scientific Advisory Committee . Muscular Dystrophy Canada. 2021-08-31. en-CA.
  14. Web site: Neuromuscular disease network to enhance Canadian research and patient care . 2021-09-08 . www.ualberta.ca . en.
  15. Web site: Toshifumi Yokota . 2021-08-17 . www.ualberta.ca . en.
  16. Web site: 2022-04-30 . Heart Warriors: 'Genetics is the future of medicine' according to U of A researcher, who uses it to help with muscular dystrophy . 2022-05-09 . The Gateway . en-US.
  17. Web site: 【研究者データ】横田俊文 . 2021-08-19. 日本の研究.com.
  18. Web site: Edmonton researchers at the University of Alberta have found a way to regrow a protein [sic] missing in Duchenne muscular dystrophy in mice. 2021-08-18. edmontonsun. en-CA.
  19. Web site: 2020-05-16. Seeking a cure for muscular dystrophy. 2021-08-20. WCHRI. en-CA.
  20. Web site: Toshifumi Yokota elected 2023 Fellow of the Canadian Academy of Health Sciences . 2024-01-10 . www.ualberta.ca . en.
  21. Web site: U of A Researchers Find Possible Relief for Muscular Dystrophy Patients. 2021-08-19. edmonton.ctvnews.ca.
  22. Web site: 2015-08-28. Researchers Develop DNA "Patch" For Canine Form of Muscular Dystrophy. 2021-08-17. National Institutes of Health (NIH). EN.
  23. Yokota. Toshifumi. Lu. Qi-Long . Partridge . Terence . Kobayashi . Masanori . Nakamura . Akinori . Takeda. Shińichi . Hoffman . Eric . June 2009. Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs . Annals of Neurology. 65. 6. 667–676 . 10.1002/ana.21627 . 1531-8249. 5951302. 19288467.
  24. Web site: First treatment for muscular dystrophy in sight: Scientists successfully harness exon-skipping. 2021-08-20. medicalxpress.com. en.
  25. Web site: 2020-10-28 . A New Precision-Medicine Therapy for Duchenne Muscular Dystrophy Recently Approved by the FDA, Nearing Approval in Canada . 2021-08-20 . Patient Worthy. en-US.
  26. Web site: 2020-08-19. Viltepso (viltolarsen) injection Now Commercially Available in the U.S.. 2021-08-22. The Associated Press . en.
  27. Web site: A DNA 'Stitch' as a Therapy for Duchenne Muscular Dystrophy . 2021-08-25. Labroots.
  28. Web site: Researchers developing new 'DNA stitch' to treat muscular dystrophy: Experimental treatment has potential to help almost half of patients with Duchenne muscular dystrophy. 2021-08-19 . ScienceDaily . en.
  29. Lim . Kenji Rowel Q. . Woo . Stanley . Melo . Dyanna . Huang . Yiqing . Dzierlega . Kasia . Shah . Md Nur Ahad . Aslesh . Tejal . Roshmi . Rohini Roy . Echigoya . Yusuke . Maruyama . Rika . Moulton . Hong M. . 2022-03-01 . Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy . Proceedings of the National Academy of Sciences of the United States of America . 119 . 9 . e2112546119 . 10.1073/pnas.2112546119 . free . 1091-6490 . 8892351 . 35193974. 2022PNAS..11912546L .
  30. Echigoya. Yusuke. Nakamura. Akinori. Nagata. Tetsuya. Urasawa. Nobuyuki. Lim. Kenji Rowel Q.. Trieu. Nhu. Panesar. Dharminder. Kuraoka. Mutsuki. Moulton. Hong M.. Saito. Takashi. Aoki. Yoshitsugu. 2017-04-18. Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy. Proceedings of the National Academy of Sciences. en. 114. 16. 4213–4218. 10.1073/pnas.1613203114. 0027-8424. 28373570. 5402437. 2017PNAS..114.4213E . free.
  31. Web site: eSkip-Finder. 2021-08-19. eskip-finder.org.
  32. Chiba . Shuntaro . Lim . Kenji Rowel Q. . Sheri. Narin. Anwar. Saeed. Erkut. Esra. Shah. Md Nur Ahad . Aslesh. Tejal. Woo. Stanley. Sheikh. Omar. Maruyama. Rika. Takano . Hiroaki. 2021-07-02. eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping. Nucleic Acids Research. 49. W1. W193–W198. 10.1093/nar/gkab442. 1362-4962. 8265194. 34104972.
  33. News: Health Innovation Roundup. en-CA. Health Cities. 2021-08-19.
  34. Web site: Canadian Institutes of Health Research. 2008-11-20. Funding Decisions Database. 2021-08-20. webapps.cihr-irsc.gc.ca.
  35. Web site: 2021-04-28 . Enhancing the efficacy of antisense oligonucleotide therapy for FSHD Muscular Dystrophy Canada . 2022-04-23 . en-CA.
  36. 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. 10.1073/pnas.1909649117 . 32601200 . 7368245 . 2020PNAS..11716509L . free .
  37. Web site: 2020-06-29. Researchers develop potential new therapy for common form of muscular dystrophy. 2021-08-23. News-Medical.net. en.
  38. Lee . Joshua J. A. . Maruyama . Rika . Duddy . William . Sakurai . Hidetoshi . Yokota . Toshifumi . 2018-12-07 . Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy . Molecular Therapy. Nucleic Acids . 13 . 596–604 . 10.1016/j.omtn.2018.10.004 . 2162-2531 . 6234522 . 30439648.
  39. Maruyama . Rika . Nguyen . Quynh . Roshmi . Rohini Roy . Touznik . Aleksander . Yokota . Toshifumi . June 2022 . Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1R206H Transcript and Inhibit Osteogenic Differentiation . Nucleic Acid Therapeutics . 32 . 3 . 185–193 . 10.1089/nat.2021.0009 . 2159-3345 . 35085461. 246360464 .
  40. Aslesh . Tejal . Erkut . Esra . Ren . Jun . Lim . Kenji Rowel Q. . Woo . Stanley . Hatlevig . Susan . Moulton . Hong M. . Gosgnach . Simon . Greer . John . Maruyama . Rika . Yokota . Toshifumi . 2023-01-31 . DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration . JCI Insight . 8 . 5 . e160516 . 10.1172/jci.insight.160516 . 2379-3708 . 36719755. 10077475 . 256415367 .
  41. Web site: Toshifumi Yokota elected 2023 Fellow of the Canadian Academy of Health Sciences . 2024-11-04 . www.ualberta.ca . en.
  42. Web site: Holley . Claire . 2019-08-01 . LO student chosen as a Fulbright Scholar . 2024-11-04 . LakeOswegoReview.com . en.
  43. Web site: scholargps .
  44. Web site: Federal RePORTER - Project Details. 2021-08-18. federalreporter.nih.gov.
  45. Web site: Canadian Institutes of Health Research. 2008-11-20. Funding Decisions Database. 2021-08-20. webapps.cihr-irsc.gc.ca.
  46. Web site: 2022-09-28 . BioAlberta Recognizes Alberta Innovators and Entrepreneurs at Annual Health and Life Sciences Showcase & Awards Ceremony . 2022-09-28 . www.businesswire.com . en.
  47. Web site: Toshifumi Yokota elected 2023 Fellow of the Canadian Academy of Health Sciences . 2024-01-10 . www.ualberta.ca . en.
  48. Web site: Gallant . Colin . 2024-03-15 . OligomicsTx wins at Startup TNT summit finale . 2024-03-26 . Taproot Edmonton . en.
  49. Web site: Fast . Darren . 2024 Innovation Awards recipients . 2024-11-02 . www.ualberta.ca . en.

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