Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy

Figure 3

Schematic representation of a double-stranded break (DSB; red arrowheads), which can be repaired through nonhomologous end-joining (NHEJ) or homology-directed repair (HDR) pathways. The introduction of a double-strand break in the DNA will typically undergo the error-prone NHEJ repair pathway, which results in insertions and deletions (INDELs) of variable length that will lead to premature stop codon formation. HDR, an error-free repair pathway, occurs using a wild-type donor template with homology to the target site, which serves as a template for precise gene correction of the host’s DNA.