Genetic Engineering Publications - GEG Tech top picks

Here, the scientists report a strategy utilizing the CRISPR system in conjunction with piggyBac technology to target and reincorporate exons 7 and 8 at the specific Arg1 locus in attempts to restore the function of arginase-1 in induced pluripotent stem cell (iPSC)-derived hepatocyte-like cells (iHLCs) and macrophages in vitro. Their study provides proof-of-concept for gene-editing at the Arg1 locus and highlights the challenges that lie ahead to restore sufficient liver-based urea cycle function in patients with urea cycle disorders.