Development of a next-generation in vivo human gene-editing therapeutic platform - Year two

The over-arching goal of our project is to develop a robust next-generation gene-editing platform to repair the deleterious mutations that are responsible for genetic diseases such as Cystic Fibrosis and cancer. First-generation precision endonuclease technologies have been tremendous for in vitro gene disruption studies and ex vivo treatments, but there has been limited success at developing safe and effective in vivo human gene-editing therapies.