RPE65 gene therapy

RPE65 gene therapy is a treatment designed for certain genetic forms of blindness caused by mutations in the RPE65 gene. This gene is crucial for the functioning of photoreceptor cells in the retina, which are essential for vision. The therapy involves delivering a functional copy of the RPE65 gene directly to the retinal cells using a modified virus. By restoring the proper gene function, this therapy can help improve vision or slow down vision loss in affected individuals. It represents a significant advance in treating genetic eye diseases and has been shown to provide meaningful benefits for patients.