Yokota Lab

Welcome to the Yokota Lab

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.

As a global leader in genetic therapeutics, Dr. Yokota directs a research program that is internationally recognized for its excellence and innovation in rare disease research. According to ScholarGPS (2024-25), the Yokota Lab ranks:

#1 in the world for research impact in Muscular Dystrophy (top 0.01%)
#3 in the world in Oligonucleotide Research (top 0.02%)
#3 in the world in Personalized Medicine (top 0.02%)

The lab is dedicated to improving the lives of individuals affected by devastating genetic conditions through the development of cutting-edge therapeutics—including RNA-targeted antisense oligonucleotides (ASOs), CRISPR-based genome editing, and personalized “n-of-1” medicines tailored to the unique genetic mutations of individual patients.

1. Next-Gen Therapeutics for Rare Diseases

The Yokota Lab is passionate about creating next-generation therapies for rare neuromuscular and genetic diseases. The team of scientists, clinicians, and students work at the intersection of molecular biology, bioinformatics, and translational medicine to develop precision treatments for:

Duchenne and Becker Muscular Dystrophy (DMD/BMD)
Dysferlinopathies (DYSF), including
Limb-Girdle Muscular Dystrophy Type 2B/R2 (LGMD2B/R2)
Miyoshi Myopathy
Distal Myopathy with Anterior Tibial Onset
Facioscapulohumeral Muscular Dystrophy (FSHD)
Fibrodysplasia Ossificans Progressiva (FOP)
Friedreich’s Ataxia (FRDA)
Giant Axonal Neuropathy (GAN)
Spinal and Bulbar Muscular Atrophy (SBMA)
—with a special focus on its prevalence among Indigenous communities in Western Canada
Spinal Muscular Atrophy (SMA)

The lab is also pioneering machine learning approaches to optimize the design and efficacy of antisense oligonucleotides, accelerating the discovery of next-gen treatments.

2. Role of Aquaporins in Muscle

This project investigates the function of water channels in muscle fibers. Notably, research has shown that the expression of water channel aquaporin-4 (AQP4) is significantly reduced in patients with muscular dystrophy, suggesting a potential link between water channel dysfunction and disease progression. Dr. Yokota's study uncovered the mechanisms by which AQP4 is recruited and localized at the muscle membrane, offering new insights into its role in muscle physiology. However, despite its well-documented presence in these tissues, the precise physiological function of AQP4 remains unclear. This project aims to elucidate the role of water channels in muscle function. By improving our understanding of water channel dynamics, we hope to reveal their broader physiological significance and its potential impact on neuromuscular disorders.

Last updated: 1 July 2025