Toshifumi Yokota, PhD, FCAHS

Area of Study / Keywords

Musculoskeletal Diseases Gene-editing Muscle Pathology Neuromuscular Disease Muscular dystrophy Antisense oligonucleotides CRISPR-Cas9 Neuroscience Neurology Genetics Gene Therapy Water Channel

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About

Dr. Toshifumi (Toshi) Yokota, PhD, FCAHS, holds the prestigious Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair at the University of Alberta, leading transformative research in genetic therapies. The research group led by Dr. Yokota, ranked #2 (top 0.01%) globally for research impact in muscular dystrophy and #4 (top 0.02%) in personalized medicine, is dedicated to advancing understanding and developing treatments for muscular dystrophy and other rare diseases. Their research aims to improve the lives of individuals affected by devastating genetic disorders through innovative therapeutic approaches such as RNA-targeted therapies, CRISPR-mediated genome editing, and patient-customized n-of-1 antisense oligonucleotides.

Dr. Yokota's pioneering research has resulted in the groundbreaking development of novel DNA-like molecules, known as antisense oligonucleotides. This innovation facilitates exon skipping, restoring gene function and enhancing muscle strength in severe animal models of Duchenne muscular dystrophy (DMD) for the first time. The resulting collaboration with pharmaceutical sectors led to the FDA-approved drug Viltolarsen in 2020, marking a significant milestone with clinical trials across Canada, Japan, and the United States showing significant improvements in muscle function.

Additionally. their pioneering work on synthetic DNA-like molecules known as gapmers has shown promise in targeting the production of toxic proteins in facioscapulohumeral muscular dystrophy (FSHD), achieving substantial gene knockdown and functional improvements in patient-derived cells and animal models for the first time. 

Career Highlights
 Pioneering RNA-targeted therapies for muscular dystrophy and rare diseases.
 Over $35M in research funding and 14 patents.
 Co-founder and Chief Scientific Officer of OligomicsTx Inc., merging academic discoveries with pharmaceutical development.

Publications and Patents
Over 100 peer-reviewed articles and numerous patents in genetic therapies.

Recognition
Numerous awards including the BioAlberta Scientific Achievement & Innovation Award and Fellow of the Canadian Academy of Health Sciences (FCAHS), one of the highest honours for researchers in Canada.

Ranked #2 in the world for research impact in muscular dystrophy (Top 0.01%) by ScholarGPS
Ranked #4 in the world for research impact in personalized medicine (Top 0.02%) by ScholarGPS

Lab Website: https://sites.google.com/ualberta.ca/yokota-lab
Google Scholar: https://scholar.google.ca/citations?user=fK0kdbsAAAAJ&hl=en

Last updated: 3 September, 2024

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Research

The research group led by Dr. Toshifumi (Toshi) Yokota is dedicated to advancing knowledge and developing treatments for muscular dystrophy and rare diseases. The goal of their research is to improve the lives of individuals affected by devastating genetic disorders through the use of cutting-edge therapeutics such as CRISPR-mediated genome editing and RNA-targeted antisense oligonucleotides. Currently, the team is actively working on several projects in this field.

1.  Next-Gen Therapeutics for Rare Diseases
The research group is devoted to developing innovative therapeutics for rare diseases, with a specific focus on several devastating genetic disorders. These disorders include Duchenne/Becker muscular dystrophy (DMD/BMD), facioscapulohumeral muscular dystrophy (FSHD), spinal muscular atrophy (SMA), spinal and bulbar muscular atrophy (SBMA), giant axonal neuropathy (GAN),  fibrodysplasia ossificans progressiva (FOP), and dysferlin-deficient muscular dystrophy, which encompasses limb-girdle muscular dystrophy type 2B/R2 (LGMD2B/R2), Miyoshi myopathy, and distal myopathy with anterior tibial onset.

2. Role of Aquaporins in Muscle and Brain
The goal of this project is to investigate the function of Aquaporin-4 (AQP4), a water channel that is specifically expressed in fast-twitch skeletal muscle fibres and the brain. However, it has been observed that the expression of AQP4 is significantly reduced in patients with muscular dystrophy. Despite its known presence in these tissues, the physiological role of AQP4 remains poorly understood. The aim of this project is to gain a better understanding of the role of water channels in muscles and brains through the characterization of AQP4.

Last updated: 16 May, 2024

For more information, please visit;  https://sites.google.com/ualberta.ca/yokota-lab

 

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Teaching

My teaching philosophy centres around fostering a love for self-learning and equipping students with the skills and concepts needed to continue learning throughout their lives. I believe that my role as a teacher is to facilitate student learning rather than simply impart my own knowledge. I recognize that laboratory research can be a challenging environment for students and make myself highly available to provide guidance and feedback when needed. As a mentor, I hold regular meetings with my students and create a supportive and welcoming atmosphere to encourage them to share their ideas and ask questions.

To be successful in biomedical research, students need a wide range of skills including good research planning, proficiency in experimental techniques, hard work, effective communication, writing, and critical reading. As a teacher, it is my goal to guide students through this process and provide them with hands-on experience that will apply their learning. Through this approach, students will gain a deeper understanding of the research process and develop the skills necessary for success in their future careers.

Classroom-based Teaching/Course Development

MDGEN 601 – Special Topics in Medical Genetics-Musculoskeletal Health

MDGEN 602 – Journal Club

MDGEN 605 –Directed Reading in Medical Genetics

Guest Lectures

GENET 304 - Gene Expression and its Regulation

GENET 418 - Human Genetics

PMCOL 200 - Drugs - An Introduction to Pharmacology 

MDGEN 601 – Special Topics in Medical Genetics

MLSCI 480 - Molecular Genetic Approaches to the Study and Diagnosis of Disease

Research Projects

BIOL 399/498/499 (Research Project)

PHYSL 467 (Undergraduate Research Project)

NEURO 452 (Honors Research Project in Neuroscience)

NEURO 498/499 (Honors Research Project in Neuroscience)

Last updated: 17 May, 2024

Positions Available

Dr. Toshifumi Yokota's laboratory at the University of Alberta is actively seeking applications for various positions including postdoctoral fellows, as well as undergraduate and graduate students. Ideal candidates for the postdoctoral positions should be highly motivated individuals who have earned their Ph.D. within the past three years. We are also looking for eager undergraduate and graduate students who are passionate about advancing their research skills in a dynamic and supportive academic environment. The University of Alberta's Dr. Toshifumi Yokota and the research team have been recognized among the top scholars in the field of muscular dystrophy (ranked #2 in the world) and personalized medicine (ranked #4 in the world) by ScholarGPS. This prestigious recognition highlights the lab's significant contributions and the impactful work being conducted at the University of Alberta.

For more information, please see: https://sites.google.com/ualberta.ca/yokota-lab/positions?authuser=0

Last updated: 22 May, 2024

Harry Wilton-Clark, Eric Yan, Toshifumi Yokota

Genes. 2024 June; 10.3390/genes15070821

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Jamie Leckie, Toshifumi Yokota

Molecules. 2024 June; 10.3390/molecules29112658

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Umme Sabrina Haque, Melissa Kohut, Toshifumi Yokota

Current Research in Toxicology. 2024 June; 10.1016/j.crtox.2024.100182

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Yokota, Toshifumi, Echigoya, Yusuke

2024 February;

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Preprints. 2024 January;

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Tang, Annie, Yokota, Toshifumi

Expert Opinion on Investigational Drugs. 2024 January;

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Yokota *, Jamie Leckie, Toshifumi

Preprints. 2024 January;

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Anwar, Saeed, Yokota, Toshifumi

biomolecules. 2024 January;

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Nakevska, Zorica, Yokota, Toshifumi

European Journal of Cell Biology. 2023 January;

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Shah, Md Nur Ahad, Yokota, Toshifumi

Therapeutic Advances in Neurological Disorders. 2023 January;

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Wilton-Clark, H., Al-aghbari, A., Yang, J., Yokota, T.

Preprints. 2023 January;

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Wilton-Clark, Harry, Yokota, Toshifumi

Molecular Therapy-Methods & Clinical Development. 2023 January;

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Haque, Umme Sabrina, Yokota, Toshifumi

2023 January;

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Tejal Aslesh, 1, Esra Erkut, 2, Jun Ren, 3, Kenji Rowel Q. Lim, 2, Stanley Woo, 2, Susan Hatlevig, 4, Hong M. Moulton, 4, Simon Gosgnach, 3, John Greer, 3, Rika Maruyama, 2, and others

JCI Insight. 2023 January;

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Yokota, Toshifumi Toshi, Maruyama, Rika, Duan, Dongsheng, Yokota, Toshifumi

Frontiers in Genome Editing. 2023 January;

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Haque, Umme Sabrina, Yokota, Toshifumi

Cells. 2023 January;

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Anwar, Saeed, Mir, Farin, Yokota, Toshifumi

Pharmaceutics. 2023 January;

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y Alex Zhu 1,2,Shuntaro Chiba 3ORCID,Yuki Shimizu 4,Katsuhiko Kunitake 5ORCID,Yasushi Okuno 3,4,Yoshitsugu Aoki 5ORCID andToshifumi Yokota 2, *ORCID

Pharmaceutics. 2023 January;

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He, Merry, Yokota, Toshifumi

Skeletal Muscle Stem Cells Methods and Protocols. 2023 January;

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Shirakaki, Satomi, Roshmi, Rohini Roy, Yokota,, Toshifumi

J. Pers. Med.. 2023 January;

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Maruyama, Rika, Fiorillo, Alyson, Heier, Christopher, Duan, Dongsheng, Yokota, Toshifumi

Frontiers in Genome Editing. 2023 January;

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Alex Zhu, Shuntaro Chiba, Yuki Shimizu, Katsuhiko Kunitake, Yasushi Okuno, Yoshitsugu Aoki, Yokota *, Toshifumi

Preprints. 2023 January;

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1, Narin Sheri, Yokota, Toshifumi

Skeletal Muscle Stem Cells Methods and Protocols. 2023 January;

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Sheri, Narin, Yokota, Toshifumi

Skeletal Muscle Stem Cells: Methods and Protocols. 2023 January;

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Anwar, Saeed, Yokota, Toshifumi

Genes. 2023 January;

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Wilton-Clark H, T., Yokota

Pharmaceutics. 2023 January;

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Wilton-Clark, Harry, Yokota, Toshifumi

2023 January;

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Wilton-Clark, Harry, Yokota, Toshifumi

Med. 2023 January;

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Anwar, Saeed, Yokota, Toshifumi

2023 January;

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Alex Zhu, Shuntaro Chiba, Yuki Shimizu, Katsuhiko Kunitake, Yasushi Okuno, Yoshitsugu Aoki, Yokota *, Toshifumi

Preprints. 2023 January;

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Wilton-Clark, Harry, Al-aghbari, Ammar, Yang, Jessica, Yokota, Toshifumi

Genes. 2023 January;

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Aslesh, Tejal, Al-aghbari, Ammar, yokota, toshifumi

International Journal of Molecular Sciences. 2023 January;

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logo, Kasia Dzierlega ORCID, Chakraborty, Mainak, logo, Megan Lee ORCID, Soliman, Amro M., Parker, Derek, Khan, Saad, Chan, Yi Tao, logo, Masoud Akbari ORCID, Yokota, Toshifumi, Winer, Shawn, others

J Immunol. 2023 January;

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Lin C., Han G., Jia L., Zhao Y., Song J., Ran N., Yokota T., Seow Y., Yin H.F.

EMBO REPORTS. 2022 June; 23 (6) 10.15252/embr.202153955

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Maruyama R., Nguyen Q., Roshmi R.R., Touznik A., Yokota T.

Nucleic Acid Therapeutics. 2022 June; 32 (3):185-193 10.1089/nat.2021.0009

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Molecular Therapy. 2022 May;

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Lim K.R.Q., Woo S., Melo D., Huang Y., Dzierlega K., Shah M.N.A., Aslesh T., Roshmi R.R., Echigoya Y., Maruyama R., Moulton H.M., Yokota T.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA. 2022 March; 119 (9) 10.1073/pnas.2112546119

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Wilton-Clark H., Yokota T.

Genes. 2022 February; 13 (2) 10.3390/genes13020257

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Erkut E., Yokota T.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES. 2022 February; 23 (3) 10.3390/ijms23031832

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Aslesh T., Yokota T.

Cells. 2022 February; 11 (3) 10.3390/cells11030417

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Sheikh O., Yokota T.

ARCHIVES OF TOXICOLOGY. 2022 January; 96 (1) 10.1007/s00204-021-03184-z

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Roshmi R.R., Yokota T.

CLINICAL PHARMACOLOGY & THERAPEUTICS. 2021 December; 13 10.2147/CPAA.S288842

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Echigoya Y., Trieu N., Duddy W., Moulton H.M., Yin H., Partridge T.A., Hoffman E.P., Kornegay J.N., Rohret F.A., Rogers C.S., Yokota T.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES. 2021 December; 22 (23) 10.3390/ijms222313065

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Wilton-Clark H., Yokota T.

DRUGS OF TODAY. 2021 December; 57 (12):707-717 10.1358/dot.2021.57.12.3352740

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Lim K.R.Q., Shah M.N.A., Woo S., Wilton-Clark H., Zhabyeyev P., Wang F., Maruyama R., Oudit G.Y., Yokota T.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES. 2021 December; 22 (23) 10.3390/ijms222312617

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Chiba S., Lim K.R.Q., Sheri N., Anwar S., Erkut E., Shah M.N.A., Aslesh T., Woo S., Sheikh O., Maruyama R., Takano H., Kunitake K., Duddy W., Okuno Y., Aoki Y., Yokota T.

NUCLEIC ACIDS RESEARCH. 2021 July; 49 (W1):W193-W198 10.1093/nar/gkab442

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Sheikh O., Yokota T.

BIODRUGS. 2021 July; 35 (4):389-399 10.1007/s40259-021-00486-7

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Tone Y., Mamchaoui K., Tsoumpra M.K., Hashimoto Y., Terada R., Maruyama R., Gait M.J., Arzumanov A.A., McClorey G., Imamura M., Takeda S., Yokota T., Wood M.J.A., Mouly V., Aoki Y.

Nucleic Acid Therapeutics. 2021 April; 31 (2):172-181 10.1089/nat.2020.0907

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Lim K.R.Q., Yokota T.

Frontiers in Pharmacology. 2021 March; 12 10.3389/fphar.2021.642858

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Lim K.R.Q., Bittel A., Maruyama R., Echigoya Y., Nguyen Q., Huang Y., Dzierlega K., Zhang A., Chen Y.W., Yokota T.

MOLECULAR THERAPY. 2021 February; 29 (2):848-858 10.1016/j.ymthe.2020.10.010

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Anwar S., He M., Lim K.R.Q., Maruyama R., Yokota T.

Journal of Personalized Medicine. 2021 January; 11 (1):1-14 10.3390/jpm11010046

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Maruyama R., Yokota T.

Methods in Molecular Biology. 2021 January; 2224 10.1007/978-1-0716-1008-4_15

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Sheikh O., Yokota T.

EXPERT OPINION ON INVESTIGATIONAL DRUGS. 2021 January; 30 (2):167-176 10.1080/13543784.2021.1868434

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Aslesh T., Erkut E., Yokota T.

EXPERT OPINION ON BIOLOGICAL THERAPY. 2021 January; 21 (8):1049-1061 10.1080/14712598.2021.1872539

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van Westering T.L.E., Johansson H.J., Hanson B., Coenen-Stass A.M.L., Lomonosova Y., Tanihata J., Motohashi N., Yokota T., Takeda S., Lehtiö J., Wood M.J.A., Andaloussi S.E.L., Aoki Y., Roberts T.C.

MOLECULAR & CELLULAR PROTEOMICS. 2020 December; 19 (12):2047-2067 10.1074/mcp.RA120.002345

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Lim K.R.Q., Nguyen Q., Yokota T.

Journal of Personalized Medicine. 2020 November; 10 (4):1-18 10.3390/jpm10040241

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