University College London – The New England Journal of Medicine has published a study report stating that the gene therapy can provide only temporary relief to the patients of Leber Congenital Amaurosis (LCA). In this inherited disorder, the patients start losing vision from childhood.
The study shows that the gene therapy improves the eyesight of the patients and retina’s sensitivity in a few weeks from the treatment.
Both these benefits last for around three years and later on start diminishing. The results were deduced from an ongoing clinical trial, which is being funded by the National Institutes of Health’s National Eye Institute (NEI). The in-depth examinations have revealed that the treated retinal areas gained the visual sensitivity rapidly during the treatment. It first expanded and then slowly contracted.
According to the University of Pennsylvania Scheie Eye Institute, Philadelphia’s Samuel G. Jacobson, MD, PhD, it is possible to improve the vision in the LCA patients who previously were not treated. He has said that gene therapy can be used to treat the patients suffering from incurable retinal problems. Although the current gene therapy provides only temporarily relief, and is not a permanent treatment as expected by the health experts earlier, the researchers are hoping to gain more knowledge in due course.
More Research Needed for Permanent Treatment
Repeated research in this field is expected to provide more permanent and improved method for treating LCA and other retina damaging disorders. Such a treatment is expected to restore the vision and sustain it for longer periods in the patients. The trials were conducted in two sets on the children suffering from blindness due to LCA.
The children suffering from LCA do not have night vision and slowly lose their daylight vision. The Leber’s congenital amaurosis directly hampers the retina’s functioning attacking the light-sensitive tissue present at the back of the eye. Around 10% people suffer from LCA and carry RPE65 gene in its mutated form.