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.

Drug Research could lead to AMD therapy

By Adrian Galbreth

New drug research may pave the way for more effective treatments of age-related macular degeneration - the leading cause of blindness in the western world.

Those are the claims being made by researchers at Tufts University School of Medicine, who say that a protein known as galectin-3 promotes the growth of new blood vessels, and that targeting the protein can "significantly reduce" angiogenesis.

The findings have been published in the Journal of Experimental Medicine and may lead to treatments for diseases caused by excessive angiogenesis, which include AMD, said Dr Noorjahan Panjwani, who led the project.

She explained: "Our study shows that galectin-3 protein binds to glycans of specific cell-adhesion proteins to activate the signaling pathways that bring about angiogenesis. This improved understanding may provide a more targeted approach to preventing harmful angiogenesis."

Meanwhile, another team of researchers at Tufts have found that that non-viral gene therapy can delay the onset of some forms of eye disease and offer hope to retinal degeneration sufferers.ADNFCR-1853-ID-800028486-ADNFCR

Visudyne therapy

Age-related macular degeneration (AMD) is a progressive disease of the eye, meaning that it can’t be cured. The good news is that AMD can be controlled. Treatments available today can slow the vision loss associated with AMD and even restore some vision.

Wet and dry age-related macular degeneration (AMD) differ in several important respects, including treatment options. The only way to find out which AMD treatment regimen or management plan is right for you is to talk to your ophthalmologist or retinal specialist.

Visudyne therapy treats a form of AMD called predominantly classic subfoveal CNV due to AMD.

In this form of AMD, abnormal blood vessels leak fluid and/or blood under the macula—the part of the eye responsible for central vision—causing serious vision loss. During therapy, Visudyne is injected into the patient (generally through the arm). Next, light from a low-energy laser activates Visudyne, causing it to destroy the leaky vessels. As a part of a wet AMD treatment regimen, Visudyne therapy may help slow its progression.

Ask your ophthalmologist or retinal specialist if Visudyne therapy may be right for you.

Other treatment options
Anti-VEGF (anti-vascular endothelial growth factor) treatments block the formation of new abnormal blood vessels