Figure 3.

Schematic depicting the gene therapy delivery approach for inherited retinal diseases. (A) Gene editing components, such as prime editors, will be packaged into the delivery vector, such as an adeno-associated virus (AAV). (B) Subretinal delivery involves injection into the space between the retinal pigment epithelium (RPE) and photoreceptors to directly target those cells, while intravitreal injection delivers the viral vector into the vitreous body and can best target the inner retina, optic nerve, and lens. (C) After injection, gene editing will occur in the targeted cell (photoreceptor shown in image), and the mutated deoxyribonucleic acid (DNA) will be edited to keep the cell healthy and functional.