Should these treatments prove to be effective and safe, they will radically transform the treatment approach to patients at risk of visual loss. Gene therapies are emerging for several inherited retinal diseases as well. These recent scientific and clinical developments may represent the beginning of an era of approved gene therapies for a wider range of neurologic diseases.
Retinal degenerative diseases are a leading cause of irreversible blindness. The central pathology in many of these conditions is retinal death, induced by a mix of genetic and environmental factors. Inherited conditions are increasingly recognized as an important component of the neuro-ophthalmic differential diagnosis of sub-acute vision loss. For these blinding conditions, innovations in gene and cell therapy approaches offer therapeutic intervention at various disease stages.
A recent review by Dr. Tatiana Bakaeva et.al from Harvard Medical School, Boston sheds light on hereditary optic neuropathies.