LCA1 is an eye disease that causes severe vision loss and is caused by mutations in the GUCY2D gene.
Individuals who have this disease usually have very poor vision, making it difficult or impossible for them to read, drive, or use their eyes to navigate in the environment.
The treatment, essentially gene therapy developed by scientists at the University of Florida, had minimal side effects except inflammation, which was resolved using steroids.
Individuals who were given the maximum dose of gene therapy saw a significant improvement in vision.
According to researchers, for many patients this treatment was as if a light had been switched on after a long time.
Shannon Boye, chief of UF's Cellular and Molecular Therapy division, co-author of the study and co-founder of Atsena Therapeutics, the UF branch that created it, said these results will help advance the therapy into clinical trials and ultimately commercialization. Open the door for. gene therapy.
To compare patients' vision in treated and untreated eyes, researchers monitored patients for a full year so they could have permanent evidence to support their claims.
Patients' vision improved more when they received larger doses.
According to the researchers, gene therapy should require only one treatment per eye and should be continued over a long enough period to have any tangible effects.
They have so far seen optical benefits that persist for at least five years, so at least that's a promising note.
LCA1 is a rare type of blindness that can permanently damage one's ability to see, but with treatments like this one, it is no longer an impossible condition.
Individuals who have this disease usually have very poor vision, making it difficult or impossible for them to read, drive, or use their eyes to navigate in the environment.
The treatment, essentially gene therapy developed by scientists at the University of Florida, had minimal side effects except inflammation, which was resolved using steroids.
Individuals who were given the maximum dose of gene therapy saw a significant improvement in vision.
According to researchers, for many patients this treatment was as if a light had been switched on after a long time.
Shannon Boye, chief of UF's Cellular and Molecular Therapy division, co-author of the study and co-founder of Atsena Therapeutics, the UF branch that created it, said these results will help advance the therapy into clinical trials and ultimately commercialization. Open the door for. gene therapy.
To compare patients' vision in treated and untreated eyes, researchers monitored patients for a full year so they could have permanent evidence to support their claims.
Patients' vision improved more when they received larger doses.
According to the researchers, gene therapy should require only one treatment per eye and should be continued over a long enough period to have any tangible effects.
They have so far seen optical benefits that persist for at least five years, so at least that's a promising note.
LCA1 is a rare type of blindness that can permanently damage one's ability to see, but with treatments like this one, it is no longer an impossible condition.
