Gene therapy can correct inherited retinal eye diseases

Researchers at the American Academy of Ophthalmology have found that Leber’s congenital Amaurosis (LCA), a very severe form of retinal disease
can be improved with the help of gene therapy.

Not only that, but the improvements were also found to be stable for two years.

Gene Therapy Can Correct Retinal Eye Diseases: LCA can be diagnosed in children at a very early age, sometimes even at infancy.

The main symptoms
of LCA are severity in vision loss and nystagmus(involuntary eye movement). By the time a person reaches his thirties or forties LCA develops into blindness.

* The impact of gene therapy could mainly be observed in children. The visual acuity and light sensitivity was tremendous in these children.
* Not only in children, researchers were also able to observe improvement in adults.

The Research and Theory: LCA is due to the mutations caused in any of the 13 genes in our body. Researchers were studying a Type 2 LCA which is due to mutation in the RPE65 gene.

* The doctors injected a normal functioning RPE65 gene which was joined with a virus into the sub-retinal space upon performing a surgery.
* The altered virus places the normally functioning RPE65 gene into the diseased cells and modifies the defective enzymes.
* After 2 weeks from the date of surgery, doctors observed that the patients reported an improvement in vision even in dim light.
* There were a few patients who also said that their visual acuity improved.
* A few even experienced massive improvement in nystagmus.
* The most encouraging result was that none of the patients experienced adverse effects.

Gene therapy does not improve sight on a permanent basis. But, gene therapy can correct inherited retinal eye diseases and help people restore sight.

People who undergo gene therapy need not be classified as blind any more. Researchers are trying hard to implement this technique in a safer way in younger children.