Toshifumi Yokota


Dr. Yokota's research focuses on understanding and developing treatments for muscular dystrophies and other genetic disorders. In particular, his research aims to develop and apply new methods, including Precision Health and Personalized Genetic Medicine, to correct mutations in a precise and targeted manner. Examples of technologies used in his research include genome editing and synthetic DNA-like molecules called antisense oligonucleotides. He demonstrated for the first time that synthetic antisense oligonucleotides can restore gene function by modulating splicing of gene transcripts, called exon skipping, and improved muscle strength in a severe animal model of Duchenne muscular dystrophy (DMD). His study directly led to a collaboration with a pharmaceutical company and the development of viltolarsen, an antisense oligonucleotide drug to treat DMD, which was approved by the FDA in 2020. Viltolarsen is the first and only FDA-approved drug that clearly restored dystrophin, a protein lacking in DMD patients, and improved muscle function in clinical trials.

Stories this researcher is featured in:

December 13, 2023

5 inspiring stories of 2023

As the year comes to a close, we want to reflect on our progress at the Women and Children’s Health Research Institute (WCHRI). 

Featuring Toshifumi Yokota Read more
October 13, 2023

Developing a “weed killer” to treat a type of muscular dystrophy

A WCHRI student is researching a DNA-like molecule to prevent muscle damage

Featuring Toshifumi Yokota Read more
February 23, 2022

New treatment could benefit up to 45 per cent of patients with Duchenne muscular dystrophy

Cocktail of drugs acts like a Band-Aid for gene mutations, allowing the body to produce the protein needed to rebuild degenerated muscle tissue.

Featuring Toshifumi Yokota Read more
June 30, 2021

Online resource will speed up development of new approach to treating genetic diseases

U of A research team leads international effort to advance exon-skipping therapy with the help of their machine-learning-based eSkip-Finder tool and database.

Featuring Toshifumi Yokota Read more
May 15, 2020

Seeking a cure for muscular dystrophy

An Edmonton team is closing in on effective treatments for two devastating genetic diseases.

Featuring Toshifumi Yokota Read more