An Oxford woman first subjected to gene therapy



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An Oxford woman became the first in the world to undergo an operation that could prevent the most common cause of blindness in the UK.

Janet Osborne suffers from age-related macular degeneration (AMD), which normally affects more than 600,000 people.

It affects the central part of the patient's vision with gaps or "spots", making everyday activities such as reading and recognizing faces difficult.

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The 80-year-old woman, who has both eyes, said that her restricted vision made housework such as vegetable preparation and sewing more difficult, and often had trouble recognizing faces.

Dr. Robert MacLaren, professor of ophthalmology at Oxford University, has developed an innovative gene therapy operation at John Radcliffe Hospital to prevent long-term vision loss.

Professor MacLaren said, "AMD is the leading cause of incurable blindness in developed countries.

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"Genetic therapy administered early to preserve the vision of patients who would otherwise lose their sight would be a major step forward and certainly what I hope to see in the near future."

This operation was part of the FOCUS trial, sponsored by Gyroscope Therapeutics, a British biotechnology company that developed gene therapy products for eye diseases such as dry AMD.

The procedure, a world first, consisted of detaching the retina and injecting a solution containing a virus.

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The virus contains a modified DNA sequence, which infects cells, called retinal pigment epithelium (RPE), and corrects a genetic defect causing AMD.

If successful, gene therapy should be used only once, as it is thought that its effects last a long time.

The goal of the therapy is to stop the progression of the disease and preserve the vision of the patients.

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It is hoped that gene therapy can be used in patients with early AMD in the future and stop the disease before their vision begins to deteriorate.

Ms. Osborne stated that she was motivated to participate in the trial to help others, stating, "I did not think about myself. I thought of other people.

"For me, I hope my eyesight will not get worse, it would be fantastic, which means I would not be so embarrassing for my family."

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