In a ray of hope for many across the world who suffer from LCA1, a team of US researchers have given patients with the rare, inherited blindness, functional eyesight for the first time.LCA1 is an eye disease that causes severe vision loss and is caused by mutations in the GUCY2D gene.
Individuals who have the disease typically have very poor vision, which makes it hard or impossible for them to read, drive or use their eyes to navigate the environment.
The treatment which was developed by University of Florida scientists, essentially gene therapy, had minimal side effects, except inflammation which was rectified using steroids.
The individuals who were administered the maximum dosage of the gene therapy demonstrated a remarkable increase in their vision.
According to the researchers, for many patients, this treatment was like a light had been switched on after long.
These outcomes open the door for the therapy’s advancement in clinical trials and eventual commercialisation, noted Shannon Boye, chief of UF’s Division of Cellular and Molecular Therapy, co-author of the study, and co-founder of Atsena Therapeutics, the UF branch that created the gene therapy.
In order to compare the patients’ eyesight in the treated and untreated eyes, researchers monitored the patients for a full year, so that they could have permanent evidence to back their claims.
The patients’ vision improved more when they received bigger doses.