Progress and prospects of engineered sequence-specific DNA modulating technologies for the management of liver diseases

Figure 1 Potential outcomes of gene therapy. A: Correction of mutations or augmentation of gene function by introduction of a second copy of the gene of interest into a safe harbor locus; B: Changes to transcriptional regulation of a gene of interest, allowing for correction of gene function either by transcriptional activation or suppression. The grey boxes represent exons and linking lines represent introns.