About

Dr. Toshifumi (Toshi) Yokota, PhD, FCAHS, 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) 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.

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 and is recognized as a Fellow of the Canadian Academy of Health Sciences (FCAHS), one of the highest honours for researchers in Canada. Dr. Yokota has garnered numerous awards, including the BioAlberta Scientific Achievement & Innovation Award, the JSPS Young Scholar Award, and the NIH Ruth L. Kirschstein National Research Service Award. Dr. Yokota is ranked 1st in the world for research impact in muscular dystrophy (Top 0.01%) 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.

Representative original research publications of Dr. Yokota with trainees since 2017 include:

1. Shah MNA et al (2025) DG9-PMO Boosts Nuclear Localization and DMD Exon 44 Skipping, Enhancing Muscle Function and Cardioprotection. Nat Commun. 16(1):4477.(IF=14.7)

2. Irigoien E et al (2025) AOC 1044 Induces Exon 44 Skipping and Restores Dystrophin Protein in Preclinical Models of Duchenne Muscular Dystrophy. Nucleic Acids Res. 53(6):gkaf241. (IF=19.16)

3. Anwar S et al (2025) Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells. Mol Ther Nucleic Acids. 36(1):102443.(IF=10.20)

4. Aslesh et al (2023) DG9 peptide-conjugated morpholino rescues phenotype in SMA model mice by reaching the CNS through a single subcutaneous administration. JCI Insight. e160516. (IF=9.484)

5. Lim et al (2022) Development of DG9 peptide-conjugated single- and multi-exon skipping antisense oligonucleotides for the treatment of Duchenne muscular dystrophy. Proc. Natl. Acad. Sci. U.S.A.119 (9) e2112546119 (IF=12.78)

6. Chiba et al (2021) eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping. Nucleic Acids Res. 49(W1): W193-W198. (IF=19.16)

7. Lim et al (2021) DUX4 transcript knockdown with antisense 2’-O-methoxyethyl gapmers for the treatment of facioscapulohumeral muscular dystrophy. Mol Ther. 29: 848-58. (IF=12.90)

8. Lim et al (2020) Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy. Proc. Natl. Acad. Sci. U.S.A. 117: 16509-16515.

9. Echigoya et al (2019) Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the DMD gene. Mol Ther. 27: 2005-2017.

10. Lim et al (2019) Efficacy of multi-exon skipping treatment in Duchenne muscular dystrophy dog model neonates. Mol Ther. 27: 76-86.

11. Lee et al (2018) Identification of novel antisense-mediated exon skipping targets in DYSF for therapeutic treatment of dysferlinopathy. Mol Ther Nucleic Acids.13: 596-604. (IF=10.20)

12. Echigoya et al (2017) Effects of systemic multi-exon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy. Proc. Natl. Acad. Sci. U.S.A. 114: 4213-8.

13. Echigoya et al (2017) Quantitative antisense screening and optimization for exon 51 skipping in Duchenne muscular dystrophy. Mol Ther. 25: 2561-2572.

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 (FCAHS)

• Parent Project Muscular Dystrophy (USA) Investigator Award

• 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 #1 in the world for research impact in muscular dystrophy (Top 0.01%) by ScholarGPS

• Ranked #3 in the world for research impact in personalized medicine (Top 0.02%) by ScholarGPS 

• Ranked #3 in the world for research impact in oligonucleotides (Top 0.02%) 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: 6 June, 2025