Host: The Japanese Society of Toxicology
Approximately 50 clinical trials of genome editing therapies have already been conducted overseas. In addition to issues, such as gene transfer efficiency and immune response to vectors and therapeutic genes, for conventional gene therapy, genome editing therapy has unique challenges including off-target mutations caused by and immune responses against artificial nucleases. In particular, in vivo off-target mutations are difficult to evaluate in preclinical studies due to differences in nucleotide sequences between humans and model animals. In order to establish genome editing as a treatment for a wider range of diseases, it is essential to develop technologies to solve these issues.
