Researchers Discover Possible Cure for Rare Form of Blindness
New Delhi: In a breakthrough for those affected by LCA1 worldwide, a team of US researchers has restored functional eyesight to patients with the rare inherited blindness for the first time.
LCA1 is an eye disease that causes severe vision loss and is caused due to the 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 of the therapy.
Gene therapy should require only one treatment per eye and persist for a sufficiently long duration of time to have any tangible effects, according to the researchers.
They have so far observed optical gains that endure for at least five years, a promising remark to say the least.
LCA1 is one of a rare type of blindness that can permanently damage any seeing faculties but it does not remain such an impossible condition after a treatment like this has been discovered.
