It is assumed that more than 50% of the most enfeebling ocular diseases have their origin in
the posterior segment. Furthermore, most of these diseases lead to partial or complete …

Highlights•Luxturna, a viral gene therapy product, is approved for rare Leber's congenital
amaurosis.•Ocular gene therapies are now being developed for non-inherited retinal …

Mutations in the photoreceptor-specific flippase ABCA4 are associated with Stargardt
disease and many other forms of retinal degeneration that currently lack curative therapies …

Recent advances in optogenetics hold promise for vision restoration in degenerative eye
diseases. Optogenetics refers to techniques that use light to control the cellular activity of …

For patients with hereditary retinal diseases, retinal gene therapy offers significant promise
for the prevention of retinal degeneration. While adeno-associated virus (AAV)-based …

Retinal gene therapy is a rapidly growing field with numerous clinical trials underway, and
route of delivery is a critical contributor to its success. Subretinal administration, which …

Purpose.: Diabetic retinopathy (DR) is the leading cause of blindness among working age
adults and does not have any curative treatments. Although chemical-and injury-induced …

Mutations in the photoreceptor-specific flippase ABCA4 lead to accumulation of the toxic
bisretinoid A2E, resulting in atrophy of the retinal pigment epithelium (RPE) and death of the …

Gene therapy is a critical tool for the treatment of monogenic retinal diseases. However, the
limited vector capacity of the current benchmark delivery strategy, adeno-associated virus …

Mutations in genes in the retinal pigment epithelium (RPE) cause or contribute to debilitating
ocular diseases, including Leber's congenital amaurosis (LCA). Genetic therapies …