Toshifumi Yokota

Summary:

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.