Two trials in gene therapy on Leber’s congenital amaurosis, an inherited form of blindness, have shown signs of temporary vision improvement, according to scientists.
Laber’s congenital amaurosis is a childhood-onset autosomal recessive blindness, known to be caused by mutations in at least 19 different genes. This condition was considered to be untreatable until 2008, when gene therapy was successfully developed for patients with the disease caused by mutated RPE65 gene.
The study published in the New England Journal of Medicine observed the effects of injecting healthy genes into the retinas of 12 young patients in a period of six years.
The scientists found that fifty percent of patients showed signs of improvement in their night vision, peaking at six to twelve months after the treatment. In these patients, the treated retina showed improved visual sensitivity, which slowly increased in area and then contracted. Unfortunately their daytime vision did not improve.
[quote text_size=”small” author=”– Robin Ali” author_title”Head of genetics at UCL’s Institute of Ophthalmology”]
Gene therapy improves night vision but there is no evidence that it slows progression of the disorder. (…) We now need a more potent gene therapy vector.
[/quote]
Scientists from the University of Pennsylvania led another small trial and have found a similar pattern of improving eyesight lasting between one to three years after the treatment, according to BBC News.
Dr. Samuel Jacobson, from the University of Pennsylvania’s Scheie Eye Institute, in Philadelphia, said that these results are an important step towards efficient cure for both inherited blindness and other diseases of the retina, even though the therapy does not appear to be a permanent treatment.
“The gain in knowledge resulting from these trials is an opportunity to improve the therapy so that restored vision could be sustained for longer periods in patients,” Dr. Jacobson concluded.
In other eyesight and retina related news here on Immortal News, comedian Roseanne Barr revealed that she is slowly going blind due to macular degeneration and glaucoma.
