Recent Success of In Utero Gene Editing

In a recent Popular Science article, author Kat Eschner explains the content and future impacts of the Nature paper "In utero CRISPR-mediated therapeutic editing of metabolic genes".  For the first time researchers used in utero base editing to treat a genetic disorder. The experiment was restricted to mice and many more successful studies need to be completed before researchers are able to begin human trials, but there were hopeful results. The researchers found that the mice who were treated in utero for their rare liver disease (HT1) with base editing, thrived in comparison to those mice not treated and even those treated after birth with nitisinone. Furthermore, the edited cells persisted through development. They discovered that Pcsk9 targeting decreased cholesterol levels and Hpd targeting resulted in 'rescuing' the deadly phenotype of HT1. The long term goal for this research is for base editing to be utilized in humans to prevent life-threatening genetic disorders.