A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency.

The article presents a report on a research related to mutation-independent CRISPR-Cas9–mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency. Topics discussed include ornithine transcarbamylase deficiency is an urea cycle disorder associated with high mortality; short-term therapeutic effects as the non-integrated genome gets lost during hepatocyte proliferation; and expression from a non-integrated minigene from the site-specific integration.