The safety of a new gene therapy for vision loss in humans has been proven.
Researchers at Johns Hopkins and their collaborators conducted a small, preliminary clinical study that showed that an experimental gene treatment that uses viruses to deliver a therapeutic gene directly into the eye was safe and could be effective for people suffering from wet age-related macula degeneration (AMD). AMD is the leading cause of blindness in the United States, affecting approximately 1.6 million Americans. The disease is characterized by abnormal blood vessel growth that causes fluid to leak into the central part of the retina, called the macula. This area of the retina is used for reading, driving, and recognizing faces.
The Lancet published a study on May 16, which describes an exciting new method in which a virus similar to the common flu, but modified in the laboratory so that it cannot cause disease, can be used as a vector for a gene, and injected into the eyes. The virus enters the retinal cells, deposits a gene that turns them into factories to produce a therapeutic protein called sFLT01.
Patients with wet AMD have an increased production of the vascular endothelial-growth factor (VEGF) within their retinas. Injections of proteins into the eye are required to bind and inactivate VEGF. This reduces fluid in the macula, and improves vision. The therapeutic proteins leave the eye after a month. Patients with wet AMD need to visit the clinic every 6 to 8 weeks to receive more injections. Many patients are not getting the injections they need because of the discomfort and burden associated with the regimen. This leads to vision loss.
Source:
https://www.hopkinsmedicine.org/news/media/releases/new_gene_therapy_for_vision_loss_proven_safe_in_humans
