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
Activity
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Congratulations Mercedes Korngut, on this amazing opportunity to share your message of finding happiness in everyday life through your partnership…
Congratulations Mercedes Korngut, on this amazing opportunity to share your message of finding happiness in everyday life through your partnership…
Liked by Toshifumi Yokota
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Excited to share that our latest preprint is now live on @PreprintsOrg! 🚀 "Recent Progress of Antisense Oligonucleotide Therapy for SOD1-mutated…
Excited to share that our latest preprint is now live on @PreprintsOrg! 🚀 "Recent Progress of Antisense Oligonucleotide Therapy for SOD1-mutated…
Shared by Toshifumi Yokota
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Excited to be a part of the upcoming Western Canadian Neuromuscular Conference in Calgary, Sept 27-29, 2024! 🧠💡 Join us for a weekend of…
Excited to be a part of the upcoming Western Canadian Neuromuscular Conference in Calgary, Sept 27-29, 2024! 🧠💡 Join us for a weekend of…
Shared by Toshifumi Yokota
Experience
Education
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Supervisors: Drs. Shin'ichi Takeda/ Ryoichi Matsuda
National Institute of Neuroscience, National Center of Neurology and Psychiatry
Awards
• National Institute of Neuroscience Distinguished Thesis Award, National Center of Neurology and Psychiatry, Japan (2003)
• National Institute of Neuroscience Distinguished Presentation Award, National Center of Neurology and Psychiatry, Japan (2003)
• Scholarship of Japan Student Service Organization
Publications
More activity by Toshifumi
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Excited to see the release of abstracts for the Breakthroughs in Muscular Dystrophy conference! Looking forward to discussions on the latest…
Excited to see the release of abstracts for the Breakthroughs in Muscular Dystrophy conference! Looking forward to discussions on the latest…
Shared by Toshifumi Yokota
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Proud to share Sarah's impactful summer project on Fibrodysplasia Ossificans Progressiva (#FOP). Her research offers hope for more effective…
Proud to share Sarah's impactful summer project on Fibrodysplasia Ossificans Progressiva (#FOP). Her research offers hope for more effective…
Shared by Toshifumi Yokota
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Excited to share our latest publication in IJMS, "Oligonucleotide Therapies for Facioscapulohumeral Muscular Dystrophy: Current Preclinical…
Excited to share our latest publication in IJMS, "Oligonucleotide Therapies for Facioscapulohumeral Muscular Dystrophy: Current Preclinical…
Shared by Toshifumi Yokota
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Was fun to bring Aanya to work and hear what she thinks her mom do at illumina. Crime buster!! #illuminaproud.
Was fun to bring Aanya to work and hear what she thinks her mom do at illumina. Crime buster!! #illuminaproud.
Liked by Toshifumi Yokota
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