Gene therapy used to cure eye disorders

Degenerative eye disorders could soon be treatable.

For a select few, gene therapy is improving sight. Thanks to research out of Oxford University, the vision of six patients has improved significantly. Their research is based on renewing the dying cells along the back of the eye.

At the moment, gene therapy is only being used to treat choroideremia (an X-chromosome-linked degenerative disease in the eye). Nevertheless, it is looking hopeful that it will be able to extend further and have the potential to attack multiple roots of blindness.

The treatment works by injecting working genes into the dying cells that line the back of the eye. Those who have choroideremia have a faulty gene that causes the light detecting cells at the back of the eye to gradually die with time. Gene therapy works by injecting a functioning gene into the cells, and once these new functioning genes are integrated into the cells, further degeneration is prevented.

Patient Jonathan Wyatt said that the surgery was like he had “come to the edge of an abyss.”

The effects are instant. The patient’s sight begins to improve immediately upon completion of the operation.

For many who suffer from vision loss, it is an unthinkable reality to reverse the devastation caused by the lost sense. Over the past twenty-five years of gene therapy, patient Wayne Thompson’s life has been shadowed by an impending blindness. Now after receiving the life altering surgery, he says “I hope I’ll see my grandchildren grow up.”

Those who suffer from choroideremia begin to notice difficulty seeing at night at some point in their teenage years. Their diagnosis is confining. They will gradually lose vision until they lose it all sometime during their forties.

This new research is promising a very different life for those who suffer from the genetic disorder. In the early stages of the disease, the operation is not only stabilizing the patient’s vision but improving it. With a degenerative disorder such as choroideremia, the earlier the treatment, the better the potential recovery is.

The potential of the operation is extending beyond the treatment of choroideremia. Macular degeneration, a common disease that affects one in four over the age of seventy-five, is next to get the gene therapy treatment. Macular degeneration affects the same cells as choroideremia, and although the implanted genes would be different, it is hopeful that this technique will one day cure this and many other causes of blindness.

The Royal National Institute of Blind People claims that this may not be the cure to blindness.

“It is at an early stage at the moment, but it does offer hope for other conditions that have a genetic basis such as macular degeneration and glaucoma,” she said.

While it is still impossible to say where this technology will lead or even whether or not it will cure blindness, the future is looking promising.

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