Gene Therapy for Other Ophthalmic Conditions

Advances in the future may include viral vectors, non-viral vectors, and delivery methods that allow treatment of a greater retinal expanse and an expanded approach to technical challenges related to gene size and disease mechanisms. Genetic therapies for non-retinal ophthalmic diseases are also being tested with gene-augmentation therapy for Leber hereditary optic neuropathy as one example. 

Efforts are also underway to develop genetic therapies for ophthalmic conditions with multifactorial causation such as age-related macular degeneration (AMD). Rather than targeting a specific disease-causing gene, as can be done for IRDs, gene therapies in this scenario are used to increase the levels of proteins that may reduce or alter disease progression. Clinical trials of genetic therapies for wet and dry AMD are currently underway.