Modeling Phenotypic Heterogeneity of Glycogen Storage Disease Type 1a Liver Disease in Mice by Somatic CRISPR/CRISPR-associated protein 9-Mediated Gene Editing.
Martijn G S RuttenTerry G J DerksNicolette C A HuijkmanTrijnie BosNiels J KloosterhuisKees C W A van de KolkJustina Clarinda WoltersMirjam H KosterLaura BongiovanniRachel E ThomasAlain de BruinBart van de SluisMaaike H OosterveerPublished in: Hepatology (Baltimore, Md.) (2021)
In conclusion, we show that somatic CRISPR/Cas9-mediated gene editing allows for the modeling of a spectrum of hepatocyte-borne GSD-1a disease symptoms in mice and to efficiently study GGIs in the liver. This approach opens perspectives for translational research and will likely contribute to personalized treatments for GSD-1a and other genetic liver diseases.