Experimental Gene Therapy [restored Sight To Blind Patients]

2023 Author: Abraham Higgins | [email protected]. Last modified: 2023-05-30 04:59

Experimental gene therapy [restored sight to blind patients]

An international team of researchers reported the effectiveness of experimental gene therapy for a rare hereditary disease that leads to blindness. After treating both eyes, three patients were able to see at dusk, recognize people's faces and shop in the store.

Results of fMRI of the patient before and after treatment when viewing the image from above. The picture after the treatment shows the activation of the visual centers. Illustration of the Children's Hospital of Philadelphia /

An international team of researchers has reported the effectiveness of experimental gene therapy for a rare inherited disease that leads to blindness, according to the BBC.

The first stage of the study involved 12 patients with Leber's congenital amaurosis, a genetic disease of the retina, in which irreparable death of light-sensitive cells occurs, which leads to a sharp deterioration in vision up to complete blindness, lack of pupil response to light and nystagmus.

Each of the participants, who saw practically nothing, was injected into one eye with a viral vector with the RPE65 gene, a defect in which underlies the disease. In 2008, when the results of the first stage were summed up, it turned out that experimental therapy somewhat improved the patients' vision without significant side effects.

After that, three of the study participants injected the gene preparation AAV2-hRPE65v2 into the other eye. According to a report published in the journal Science Translational Medicine, this led to further improvement in vision: patients were able to see at dusk, recognize people's faces and shop in the store.

Manzar Ashtari of the Children's Hospital of Philadelphia said that functional magnetic resonance imaging performed on the volunteers confirmed the activation of the visual centers of the brain when looking at various objects. Study leader Jean Bennett of the University of Pennsylvania said her team now intends to treat the other nine patients with a second eye.