Guide To Diagnosing And Treating Leber Congenital Amaurosis

Gene Therapy

Gene therapy can be used to treat some individuals affected by Leber congenital amaurosis. If a patient's LCA is caused by mutations in both versions of their RPE65 gene, a gene therapy product is available to help treat vision loss caused by the disorder. This type of therapy works when the gene product is injected into the area underneath the retina, allowing for a new and working version of the RPE65 gene to diffuse into the affected cells.

In order for this product to be injected, a vitrectomy or a surgical procedure to remove vitreous humor gel from the eye cavity is required. This procedure allows the ophthalmologist to have better retinal access. While gene therapy does not cure vision loss caused by Leber congenital amaurosis, it is successful at making improvements in the patient's vision. It is vital for affected individuals to undergo this therapy soon after diagnosis, and before their retina has degenerated past a certain point. In order for a patient to undergo gene therapy for LCA, they have to have enough functional retina present for gene repair.

Keep reading to discover more about treating Leber congenital amaurosis now.