According to the research, injection under the retina may increase light sensitivity in case of inherited blindness.
Researchers from the University of Florida and Pennsylvania took three patients suffering from Leber congenital amaurosis (LCA), a form of blindness caused by changes in the RPE65 gene, for the present study.
These people who were blind since birth were injected with ‘RPE65’ under the retina of the eye, and after one year of treatment they were able to detect dim lights.
"At one year, we have now found that the RPE65 gene therapy appears to be safe and leads to a stable visual improvement in the patients studied,” said study author, Artur V. Cideciyan from the University of Pennsylvania.
Dr. William Hauswirth from University of Florida affirms, “The gene essentially goes into the cells that is missing it, and it starts expressing the protein that it encodes, and that protein is the enzyme they’re missing. So, this enzyme then restores the visual cycle to these cells.”
The finding a significant development in blindness treatment
Dr. Paul A. Sieving, director of the U.S. National Eye Institute said, “These results are very significant because they represent one of the first steps toward the clinical use of gene therapy for an inherited form of blindness.
“I anticipate that it is only a matter of time before similar techniques will be applied to other genetic diseases affecting vision,” he adds.
This was Phase I of the research and only aimed to find how useful the gene therapy was and whether it had any side effects or not.
The success of this phase and improvements in vision in one patient after one year of treatment has encouraged the researchers to further their research for more valid use of the gene therapy.
The present study is specifically beneficial for people suffering from LCA blindness, and more research on varied participants can further prove the benefit of this gene in other forms of blindness.
The findings were published in the online edition of Human Gene Therapy and also in August issue of the New England Journal of Medicine.
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