Toshifumi Yokota, PhD, FCAHS

Professor, Faculty of Medicine & Dentistry - Medical Genetics Dept


Professor, Faculty of Medicine & Dentistry - Medical Genetics Dept
(780) 492-1102
831 Medical Sciences Building
8613 - 114 St NW
Edmonton AB
T6G 2H7


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


Yokota-lab Website:

Dr. Toshifumi (Toshi) Yokota is a Professor of Medical Genetics at the University of Alberta, holding the title of the Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair. Dr. Yokota's academic journey began with a Ph.D. in cell biology from the University of Tokyo. As a JSPS Research Fellow, Dr. Yokota went on to work at Imperial College London and Children's National Medical Center in Washington DC as a Research Associate before joining the University of Alberta. 

Dr. Yokota's pioneering research has led to significant advancements in the treatment of muscular dystrophy. The study, which was the first of its kind, demonstrated that a cocktail of artificial DNA-like molecules called antisense oligonucleotides can restore gene function and improve muscle strength in a severe animal model of Duchenne muscular dystrophy (DMD) by modulating the splicing of gene transcripts called mRNA. This research directly led to a collaboration with a pharmaceutical company and the development of viltolarsen, an antisense oligonucleotide drug for the treatment of DMD, which was approved by the FDA in 2020 after clinical trials conducted in Japan, Canada, and the United States showing significant improvements in muscle function. More recently, Dr. Yokota's research team developed an approach using synthetic DNA-like molecules called gapmers to interfere with the production of a toxic protein that causes muscle destruction in facioscapulohumeral muscular dystrophy (FSHD). The treatment reduced the toxic gene products called DUX4 by more than 99% and resulted in morphological and functional improvement in patient-derived cells. This is a significant breakthrough in the treatment of FSHD, which is the third most common form of muscular dystrophy. 

With over 100 peer-reviewed articles, editorial roles in several journals, and numerous awards, Dr. Yokota's work is recognized globally, including being elected as a Fellow of the Canadian Academy of Health Sciences, one of the highest honours for researchers in Canada. Dr. Yokota's academic and professional activities reflect a commitment to advancing therapies for rare diseases, particularly muscular dystrophies.

Last updated: 22 January, 2024


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 characterization of AQP4.

Last updated: 6 July 2023

For more information, please visit;



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.

Last updated: 22 January, 2024

Featured Publications

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

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

Antisense Oligonucleotide-Mediated Exon 27 Skipping of Dysferlin for the Treatment of Dysferlinopathy

Molecular Therapy. 2022 May;

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.


Wilton-Clark H., Yokota T.

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

Erkut E., Yokota T.

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

Aslesh T., Yokota T.

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

Sheikh O., Yokota T.

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

Roshmi R.R., Yokota T.

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

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

Wilton-Clark H., Yokota T.

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

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

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

Sheikh O., Yokota T.

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

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

Lim K.R.Q., Yokota T.

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

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

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

Maruyama R., Yokota T.

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

Sheikh O., Yokota T.

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

Aslesh T., Erkut E., Yokota T.

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

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

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

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