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Back in September, a breakthrough gene therapy was found to treat inherited retinal dystrophies. Now, people in the UK are being treated with it.
This gene therapy is meant for people who are born with retinal dystrophy. It happens when the patient inherits a faulty copy of the RPE65 gene from both parents. Babies lose sight as they grow older since this gene is crucial for providing the requirements that light-sensitive cells need to absorb light.
The trouble of sight starts with poor night vision and leads to complete blindness with the death of the current cells.
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NHS therapy is expected to halt sight loss and even improve vision in the process. In order to do this, working copies of the RPE65 gene is injected into the back of the eye. The copies are put inside a harmless virus and the virus penetrates the retinal cells.
After the virus is inside the nucleus, the gene provides the instructions to make the RPE65 protein. This provides a healthier vision.
The treatment is sadly only suitable for patients who have some vision remaining. It is expected to be beneficial to the children who have this eye disorder since it can stop sight loss before any permanent damage is done.
Matthew Wood is one of the first patients to receive the therapy. He started losing his sight as a child, and only had peripheral vision currently. His surgery was carried out by Professor Robert MacLaren.
In a BBC interview, MacLaren said, “This is very exciting – this is the first approved NHS gene therapy for an eye disease, but there are opportunities to use gene therapy to treat other diseases in the future, not only in the eye.”
One downside of the treatment is the price point with £600,000. However, NHS England has stated that they have agreed on a discount with the manufacturer Novartis.