About

Dr. Toshifumi (Toshi) Yokota is a Professor of Medical Genetics at the University of Alberta, holding the esteemed title of the Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair and also serving as the Co-Founder and Chief Scientific Officer (CSO) of OligomicsTx Inc., a company focused on the development of innovative genetic therapies.

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 resulted in the groundbreaking development of a cocktail 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). 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.

More recent developments include an innovative approach using synthetic DNA-like molecules called gapmers to target the production of a toxic protein in facioscapulohumeral muscular dystrophy (FSHD). Demonstrating a reduction in toxic gene products by more than 99%, this approach shows promising functional improvements in patient-derived cells and animal models, heralding a new era in the treatment of this common muscular dystrophy.

Dr. Yokota has earned significant accolades for contributions to the scientific community, recognized as a Fellow of the Canadian Academy of Health Sciences (FCAHS), one of the highest honours for researchers in Canada. Throughout an illustrious career, Dr. Yokota has garnered numerous awards, including the BioAlberta Scientific Achievement & Innovation Award, JSPS Young Scholar Award, and the NIH Ruth L. Kirschstein National Research Service Award. Dr. Yokota is also ranked as the #2 (Top 0.01%) Highly Ranked Scholar in the world in the field of Muscular Dystrophy by ScholarGPS. With a publication record of over 100 peer-reviewed articles and having edited three books, Dr. Yokota also actively contributes as a board member to various journals and organizations. This significant body of work has played a pivotal role in advancing the development of novel therapies for muscular dystrophy and other rare diseases.

Academic Degrees and Training

• BSc: The University of Tokyo  

• Ph.D.: The University of Tokyo (Supervisors: Dr. Shin'ichi Takeda, Dr. Ryoichi Matsuda) 

• Post-doctoral training: Imperial College London (Supervisor: Dr. Terence Partridge)  

• Research Associate: Children’s National Medical Center, Washington DC (Supervisors: Dr. Terence Partridge, Dr. Eric Hoffman)  

Honours

• Fellow, Canadian Academy of Health Sciences (CAHS)

• BioAlberta Scientific Achievement and Innovation Award

• The Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair

• Henri M Toupin Neurological Science Research Chair

• National Institutes of Health (NIH) Ruth L. Kirschstein National Research Service Award (NRSA) 

• Ranked #2 (Top 0.01%) Highly Ranked Scholar in the world for contributions to Muscular Dystrophy Research in the last 5 years by ScholarGPS

• Ranked #4  (Top 0.02%) Highly Ranked Scholar in the world for contributions to Personalized Medicine in the last 5 years by ScholarGPS 

Professional Activities and Membership

• Rare Disease Consortium Japan (Co-Founder/ Executive Board)

• AGADA Biosciences (Advisor)

• Defeat Duchenne Canada (Research Funding Advisory Committee)

• Neuromuscular Diseases Network (Principal Investigator/ Co-Founder/ Executive Board)

• European Cooperation in Science and Technology (COST) Action Network for Delivery of Antisense RNA ThERapeutics (DARTER) (Co-founder)

• Muscular Dystrophy Canada (Research Chair, Medical and Scientific Advisory Committee Member) 

• Patients' Association for Dysferlinopathy Japan (Scientific Advisor)

• Cardiovascular Research Institute (CVRI)

• Neuroscience and Mental Health Institute (NMHI)

• Women and Children’s Health Research Institute (WCHRI) (Social Media Ambassador)

• National Center of Neurology and Psychiatry 

(Visiting Researcher)

Editorial Activities

• Editorial Board Member: 

Genes (MDPI)

International Journal of Molecular Sciences (MDPI)

Frontiers in Genome Editing (Nature Publishing Group) 

Frontiers in Physiology (Nature Publishing Group)

Cells (MDPI)

Nucleic Acid Therapeutics (Mary Ann Liebert)

• Guest Editor: 

Genome and Transcriptome Editing to Understand and Treat Neuromuscular Diseases. Front Genome Ed. (2022)

Genome-Editing Therapies. Int J Mol Sci.  (2019)

Molecular Diagnosis and Novel Therapies for Neuromuscular/ Musculoskeletal Diseases. J Pers Med. (2018)

Muscular Dystrophy: Disease Mechanisms and Therapies. BioMed Res Intl. (2015)

Google Scholar | PubMed Bibliography 

Last updated: 23 May, 2024