NGS data from ‘Treatment of a metabolic liver disease by in vivo genome base editing in adult mice’

CRISPR/Cas9-mediated base editing allows to interchange purine bases or pyrimidine bases independent of homology directed repair and independent of double stranded DNA breaks. We used a dual AAV system to deliver base editing agents to a murine disease model in vivo. We reliably corrected the Phenylketonuria phenotye in our mouse model, where the readout was, reversion of the fair hair phenotype, restoration of in vitro enzyme activity NGS of the locus to quantify correction on genomic DNA and mRNA level. We further evaluated off-target effects on genomic DNA.