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“World’s first” AMD gene therapy carried out by Oxford researchers

The procedure involves detaching the retina and injecting a virus to correct the cause of the eye disease

05 Mar 2019 by Andrew McClean

A professor from the University of Oxford has carried out “the world’s first gene therapy” to tackle the cause of age-related macular degeneration (AMD).

If successful, the treatment could improve patients’ quality of life and their ability to remain independent.

The procedure was carried out at John Radcliffe Hospital by professor of ophthalmology at the University of Oxford, Professor Robert MacLaren.

Speaking about the trial, Professor MacLaren said: “AMD is the number one cause of untreatable blindness in the developed world. A genetic treatment administered early on to preserve the vision in patients who would otherwise lose their sight would be a tremendous breakthrough and certainly something I hope to see in the near future.”

Janet Osborne was the first person to under the procedure. The 80-year-old has AMD in both eyes, but the central vision in her left eye has deteriorated.

Speaking about why she decided to participate in the trial, Ms Osborne said: “I wasn’t thinking of me. I was thinking of other people. For me, I hope my sight doesn’t get any worse. That would be fantastic. It means I wouldn't be such a nuisance to my family.”

The trial was sponsored by British biotech company, Gyroscope Therapeutics, with support provided by the National Institute for Health Research Oxford Biomedical Research Centre.

During the operation, the retina is detached and a solution containing a virus is injected underneath. The virus infects the retinal pigment epithelium and corrects a genetic defect that causes AMD. It is hoped that the procedure only needs to be carried out once.

Professor MacLaren explained: “We’re harnessing the power of the virus, a naturally occurring organism, to deliver the DNA into the patient's cells. When the virus opens up inside the retinal cell it releases the DNA of the gene we have cloned, and the cell starts making a protein that we think can modify the disease, correcting the imbalance of the inflammation caused by the complement system.”

“The idea of this gene therapy is to deactivate the complement system, but at a very specific point at the back of the eye, so the patient would otherwise be unaffected by it, and we hope that in the future it will slow down the progression of macular degeneration,” he added.

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