A revolutionary medical procedure called gene therapy gives those with hereditary blindness fresh hope. Clinical studies are also being conducted to expand these therapies to treat additional diseases. Recent scientific advancements have shown techniques to treat various types of blindness using gene therapy.
The development of gene therapy medications for hereditary eye illnesses is being spearheaded by Jean Bennett, a gene therapy expert and retired professor of ophthalmology at the University of Pennsylvania. The first gene therapy treatment for a hereditary condition to receive U.S. approval was created by Bennett and her colleagues. Patients with biallelic RPE65 mutation-associated retinal dystrophy, a rare genetic condition that results in early blindness and visual deficits, can be treated with luxturna.
Bennett recently discussed gene therapy and the accomplishments her team has made in enabling the blind to sight at the 2023 Imagine Solutions Conference in Naples, Florida. Bennett described how gene therapy addresses illness by switching on or off genes, introducing a new or modified gene into the body, and replacing a disease-causing gene with a healthy new or modified copy of that gene.
different organizations all around the world have started clinical studies aimed at different hereditary kinds of blindness, such as choroideremia, achromatopsia, retinitis pigmentosa, and age-related macular degeneration, as a result of the discovery of gene therapy for inherited illness.
Thanks to the pioneering work of doctors like Bennett, there are at least 40 clinical studies enrolling patients with additional hereditary types of blinding condition. Even while not all types of blindness can be healed, gene therapy is giving millions of individuals across the world who are visually impaired fresh hope. With continuing clinical studies, scientists are investigating new applications for this cutting-edge method to cure a variety of genetic illnesses, giving patients all around the world fresh hope.