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 […]

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