Genetic Therapy Improves Sight of Near Blind Patients

A new genetic therapy has demonstrated the ability to improve the vision of patients who would otherwise have gone blind.

It is hoped that this could have a massive benefit for the partially sighted in the future.

And a clinical study has shown that the improvement is long-lasting and so the therapy is suitable to be offered as a treatment.

Researchers are now set to apply for approval for trials to begin next year.

These will assess some of the more common forms of blindness, such as macular degeneration.

Meanwhile, the results have been published in the New England Journal of Medicine.

The therapy involves injecting a working copy of the gene into the back of patients’ eyes to prevent more cells from dying.

Researchers found that the treatment revives some of the dying cells and improves the patient’s vision.

There is early promise from the research, although it should be emphasised that the clinical study carried out was certainly small.

Only 32 patients have been engaged with the treatment over the last four and a half years.

Initially beginning with older patients, researchers have gained confidence in the efficacy of the technique, and have since tried it on progressively younger patients with reasonably good vision.

The study has also indicated that the treatment has the potential to last for a significant period of time.

This is based on the fact that the first patient received the therapy four and a half years ago and his treated eye has shown no diminution in vision.

Prof Robert MacLaren of Oxford University commented that the new gene treatment has huge potential.

“The concept of gene therapy is that it corrects gene defects. Ideally we should only have to do that once, because once the DNA is corrected and inserted into the correct cell, that cell should be able to continue its function as normal. We seem to have achieved this concept of one single treatment that does not need to be repeated which is unlike traditional medicines.”

MacLaren believes that a gene therapy for choroideremia could be licensed within three years, and that the existing research has demonstrated that gene therapy for blindness is safe and effective.

“When I started my career as an eye surgeon when we had these patients that had inherited diseases, not only did we tell them nothing could be done but we would actually discharge them from the clinics. We are now calling them back in to test them, to look at them in great detail because potential treatments are available. To treat a disease at the genetic level is surely the most efficient way of treating a disease, to prevent it from happening in the first place.”


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