Contact
Professor, Faculty of Medicine & Dentistry - Medical Genetics Dept
- toshifum@ualberta.ca
- Phone
- (780) 492-1102
- Address
-
831 Medical Sciences Building
8613 - 114 St NWEdmonton ABT6G 2H7
Overview
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
About
Yokota-lab Website:https://sites.google.com/ualberta.ca/yokota-lab
Dr. Toshifumi Yokota 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. Notably, research into antisense oligonucleotides and exon-skipping technologies led to the FDA-approved drug, viltolarsen, for Duchenne muscular dystrophy. Additionally, groundbreaking work on synthetic DNA-like molecules called gapmers targets the production of toxic proteins in facioscapulohumeral muscular dystrophy (FSHD), showing significant gene knockdown and functional improvements in patient-derived cells and animal models.
Career Highlights
Pioneering RNA-targeted therapies for muscular dystrophy and rare diseases.
Over $34M 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.
Named #2 (Top 0.01%) Highly Ranked Scholar in the world for contributions to the Muscular Dystrophy Research in the last 5 years by ScholarGPS
Named #4 (Top 0.02%) Highly Ranked Scholar in the world for contributions to Personalized Medicine in the last 5 years 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: 30 May, 2024
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
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
Announcements
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
Featured Publications
Harry Wilton-Clark, Eric Yan, Toshifumi Yokota
Genes. 2024 June; 10.3390/genes15070821
Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA
Jamie Leckie, Toshifumi Yokota
Molecules. 2024 June; 10.3390/molecules29112658
Umme Sabrina Haque, Melissa Kohut, Toshifumi Yokota
Current Research in Toxicology. 2024 June; 10.1016/j.crtox.2024.100182
Antisense oligonucleotides that bind to exon 51 of human dystrophin pre-mRNA
Yokota, Toshifumi, Echigoya, Yusuke
2024 February;
Preparing for patient-customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases
Preprints. 2024 January;
Duchenne muscular dystrophy: promising early-stage clinical trials to watch
Tang, Annie, Yokota, Toshifumi
Expert Opinion on Investigational Drugs. 2024 January;
Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA
Yokota *, Jamie Leckie, Toshifumi
Preprints. 2024 January;
The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments
Anwar, Saeed, Yokota, Toshifumi
biomolecules. 2024 January;
Challenges and Future Perspective of Antisense Therapy for Spinal Muscular Atrophy: A Review
Nakevska, Zorica, Yokota, Toshifumi
European Journal of Cell Biology. 2023 January;
Cardiac therapies for Duchenne muscular dystrophy
Shah, Md Nur Ahad, Yokota, Toshifumi
Therapeutic Advances in Neurological Disorders. 2023 January;
Advancing Epidemiology and Genetic Approaches for the Treatment of Spinal and Bulbar Muscular Atrophy (SBMA): Focus on Prevalence in the Indigenous Population of Western Canada
Wilton-Clark, H., Al-aghbari, A., Yang, J., Yokota, T.
Preprints. 2023 January;
CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy
Wilton-Clark, Harry, Yokota, Toshifumi
Molecular Therapy-Methods & Clinical Development. 2023 January;
DG9, a Versatile Cell-Penetrating Peptide to Enhance Delivery of Antisense Oligonucleotide-Based Therapeutics
Haque, Umme Sabrina, Yokota, Toshifumi
2023 January;
DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration
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;
Editorial for ‘Genome and Transcriptome Editing to Understand and Treat Neuromuscular Diseases’
Yokota, Toshifumi Toshi, Maruyama, Rika, Duan, Dongsheng, Yokota, Toshifumi
Frontiers in Genome Editing. 2023 January;
Enhancing Antisense Oligonucleotide-Based Therapeutic Delivery with DG9, a Versatile Cell-Penetrating Peptide
Haque, Umme Sabrina, Yokota, Toshifumi
Cells. 2023 January;
Enhancing the Effectiveness of Oligonucleotide Therapeutics Using Cell-Penetrating Peptide Conjugation, Chemical Modification, and Carrier-Based Delivery Strategies
Anwar, Saeed, Mir, Farin, Yokota, Toshifumi
Pharmaceutics. 2023 January;
Ensemble-Learning and Feature Selection Techniques for Enhanced Antisense Oligonucleotide Efficacy Prediction in Exon Skipping
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;
Exons 45--55 Skipping Using Antisense Oligonucleotides in Immortalized Human DMD Muscle Cells
He, Merry, Yokota, Toshifumi
Skeletal Muscle Stem Cells Methods and Protocols. 2023 January;
Genetic Approaches for the Treatment of Giant Axonal Neuropathy
Shirakaki, Satomi, Roshmi, Rohini Roy, Yokota,, Toshifumi
J. Pers. Med.. 2023 January;
Genome and transcriptome editing to understand and treat neuromuscular diseases
Maruyama, Rika, Fiorillo, Alyson, Heier, Christopher, Duan, Dongsheng, Yokota, Toshifumi
Frontiers in Genome Editing. 2023 January;
Improved Prediction of Antisense Oligonucleotide Efficacy for Exon Skipping Using Ensemble Learning and Feature Selection
Alex Zhu, Shuntaro Chiba, Yuki Shimizu, Katsuhiko Kunitake, Yasushi Okuno, Yoshitsugu Aoki, Yokota *, Toshifumi
Preprints. 2023 January;
In Vivo Evaluation of Exon 51 Skipping in hDMD/Dmd-null Mice
1, Narin Sheri, Yokota, Toshifumi
Skeletal Muscle Stem Cells Methods and Protocols. 2023 January;
In Vivo Evaluation of Exon 51 Skipping in hDMD/Dmd-null Mice
Sheri, Narin, Yokota, Toshifumi
Skeletal Muscle Stem Cells: Methods and Protocols. 2023 January;
Navigating the Complex Landscape of Fibrodysplasia Ossificans Progressiva: From Current Paradigms to Therapeutic Frontiers
Anwar, Saeed, Yokota, Toshifumi
Genes. 2023 January;
Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy
Wilton-Clark H, T., Yokota
Pharmaceutics. 2023 January;
Recent Trends in Antisense Therapies for Duchenne muscular dystrophy
Wilton-Clark, Harry, Yokota, Toshifumi
2023 January;
Safety concerns surrounding AAV and CRISPR therapies in neuromuscular treatment
Wilton-Clark, Harry, Yokota, Toshifumi
Med. 2023 January;
The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments
Anwar, Saeed, Yokota, Toshifumi
2023 January;
Use of 3-Way Voting of Machine Learning Algorithms Improves Prediction Performance of the Efficacy of Antisense-Mediated Exon Skipping and Reduces the Computational Burden
Alex Zhu, Shuntaro Chiba, Yuki Shimizu, Katsuhiko Kunitake, Yasushi Okuno, Yoshitsugu Aoki, Yokota *, Toshifumi
Preprints. 2023 January;
Advancing Epidemiology and Genetic Approaches for the Treatment of Spinal and Bulbar Muscular Atrophy: Focus on Prevalence in the Indigenous Population of Western Canada
Wilton-Clark, Harry, Al-aghbari, Ammar, Yang, Jessica, Yokota, Toshifumi
Genes. 2023 January;
Assessing the Role of Aquaporin 4 in Skeletal Muscle Function
Aslesh, Tejal, Al-aghbari, Ammar, yokota, toshifumi
International Journal of Molecular Sciences. 2023 January;
Activin A--Expressing Polymorphonuclear Myeloid-Derived Suppressor Cells Infiltrate Skeletal and Cardiac Muscle and Promote Cancer Cachexia
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;
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.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA. 2022 March; 119 (9) 10.1073/pnas.2112546119
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
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
Wilton-Clark H., Yokota T.
DRUGS OF TODAY. 2021 December; 57 (12):707-717 10.1358/dot.2021.57.12.3352740
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
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
Restoration of dystrophin expression and correction of Duchenne muscular dystrophy by genome editing
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
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