Review Article

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

Figure 2

Therapeutic approaches for treating retinal dystrophies. For an in vivo approach (indicated in blue), the patient’s DNA is isolated, and genetic screening is carried out to identify the pathogenic mutation causing the retinal phenotype. Delivery of the CRISPR/Cas9 components to correct the pathogenic mutation in vivo is achieved via AAV vectors administrated directly to the retina of the patients. For an ex vivo approach (in green), patient’s fibroblasts with a known mutation in an IRD gene are isolated and reprogrammed to patient-specific iPSC. Genome editing of iPSCs is carried out using the CRISPR/Cas9 system. The corrected iPSCs are further differentiated into retinal cells, which can then be reimplanted into the patient’s retina.