Gene Therapy for Ophthalmic Diseases

Abstract

In the field of ophthalmology, gene therapy has focused on hereditary retinal dystrophy, including retinitis pigmentosa. Hereditary retinal dystrophy is a group of intractable diseases for which there is currently no effective treatment, and expectations are focused on gene therapy and retinal regeneration therapy using iPS cells. In recent years, results of a number of clinical trials of gene therapies for hereditary retinal dystrophy have been reported from Europe and the United States. Since 2017, gene therapy drugs for Leber congenital amaurosis have been approved after being found to be safe and therapeutically efficacious. In this article, we will discuss the current state of gene therapy for retinitis pigmentosa and the use of adeno-associated virus (AAV) vectors in gene therapy.