Toward curing intractable diseases with genetic mutations

Akitsu Hotta

Center for iPS Cell Research and Application

Many of the diseases that cannot be cured even with modern medicine are caused by genetic mutations. One may think that genetic mutations are somebody else’s problems, but everyone, including healthy individuals, has an average of 17 to 20 disease-related ones. Genes are carefully stored in the nucleus of cells, so it has not been easy to find and repair only the mutated parts. However, this has become possible with the advent of CRISPR genome editing technology, which can alter the DNA sequence of targeted genes. In my laboratory, we are working on developing treatments for muscular dystrophy and other intractable diseases by fully utilizing the genome editing systems of bacteria, the gene delivery mechanisms of viruses, and iPS cell technology.