LONDON: Researchers at Oxford say they have completed the first gene therapy operation in the world to stop Age-Related Macular Degeneration (AMD), the leading cause of incurable blindness in developed countries.
Dry AMD is a slow process of becoming progressively worse of the cells of the macula. It affects the central part of the patient’s vision with gaps or “dots”, which hinders everyday activities such as reading and recognizing faces.
If successful, treatment could have a beneficial impact on patients quality of life and their ability to remain independent.
“Early genetic treatment to preserve the vision of blind patients would be a big step forward, and certainly what I hope to see in the near future,” said Robert MacLaren, a professor at the University of Oxford in the United Kingdom.
The first person to experience the process was Janet Osborne of Oxford, said MacLaren, who treated at John Radcliffe Hospital in the United Kingdom.
Like many people with AMD, Osborne has both eyes but is more advanced than his left eye. As is typical of this disease, the central vision of his left eye has deteriorated and it is very blurry, although his peripheral vision is better.
The 80-year-old woman said that her limited vision makes household chores difficult, such as preparing vegetables and sewing, and she cannot read much. She has trouble recognizing faces.
Osborne said her motivation to participate in the trial was the opportunity to help others with AMD.
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“I did not think of myself, I thought of other people, for me, I hope my vision does not get worse, it would be great, that means it would not be a nuisance to my family,” she said.
The operation consists of separating the retina and injecting a solution containing a virus underneath.
The virus contains an altered DNA sequence, which infects the cells, called retinal pigment epithelium (RPE), and corrects a hereditary imperfection that causes AMD.
Ideally, if successful, gene therapy should be done only once because it is believed that its effects last a long time.
A key factor in AMD is the supplement system, a system of proteins in our immune system that battles bacteria.
In macular degeneration, these proteins are too active and start attacking the cells of the retina in the same way as bacteria.
“We are exploiting the power of the virus, a natural organism, to send DNA to the patient’s cells,” MacLaren said.
“When the virus opens up in the retinal cell, it releases the DNA from the gene we cloned and the cell begins to produce a protein that we believe can alter the disease, correcting the imbalance of the inflammation caused.” Through the complement system, ” He said.
“The idea of this gene therapy is to” disable “the complement system, but at a very precise point in the back of the eye, so that the patient is not affected by it, and we hope that” In the future, this will retard the progression of macular degeneration, “said McLaren.
“We presently see better the connection between the complement system and the DMA, which has driven us to find that restoring the balance of a hyperactive complement system might be a conceivable helpful methodology in AMD.” Peter Lachman, a scientist at the University of Cambridge said.
The goal of therapy is to stop the progression of the disease and preserve the vision of patients.
If successful, it is expected that gene therapy can be used in patients with early AMD in the future and stop the disease before vision begins to deteriorate. PTI