Sunday , August 7 2022

Therapy of innovative genes to treat the most frequent cause of blindness


Inside the eye, the ocular macular or lutea macula in an area that is located in the middle and inner part of the retina, which looks like a small pigmented areas of yellow, of small thickness – such as paper – which includes light sensitive cells, which can send electrical signals to the brain and lead to vision.

This small part is responsible for the central vision and allows us to see the details and color, such as facial distinction, obviously seeing small objects, reading without problems, watching TV or driving, for example.

Among the pathologies that can affect it, it's Age-related macular degeneration (AMD), ocular degenerative disease, due to a change in the regulated genes and ancillary system – a system of proteins produced by the immune system that combat bacteria – which causes the vitality of these molecules and Retina cells assault as bacteria, with the result of cell death and loss of vision.

He currently represents the first cause of blindness in developed countries, and among the risk factors of macular degeneration is age, as it increases especially after 50 years, where the number is 5.3%, but after 85 years it rises to 1 out of 3 of people.

There are two types of AMD: dry or atrophic AMD, the most common type of disease – 90% of cases – slowly moving (years), if it gives initial symptoms, until total Losing vision after decades AMD Wet or exudative, with 10% of cases, is very aggressive and results in a loss of central vision in a short period (weeks, months).

The early diagnosis of the two types of macular dystrophi can improve the sequence of the disease, although there is no specific treatment available for AMD dry to slow down the evolution of the disease.


A team of surgeons used at John Radcliffe Hospital in Oxford, UK – led by Oxford University Professor of Ophthalmology, Robert McLaren – for the first time in a history of genetic therapy in the treatment of AMD, and It aims to tackle the genetic cause that is based on this pathology.

This procedure includes the injection to back eye solution containing innocent virus, carrier a synthetic gene – modified DNA sequence – which, when released, is infected with retina cells. This gene allows the protein synthesis eye designed to prevent the cells being destroyed and thus maintain the functional mucus.

As Professor MacLaren says:

"We take advantage of the power of the virus, a natural organism, to send DNA into the patient's cells. When the virus opens inside the retina cell, it releases the DNA of the gene that we have cloned, and the cell begins to produce the protein we believe that we can adjust the disease, remedy the imbalance of inflammation caused by the supporting system.. "He added:"The idea of ​​this gene therapy is to disable & # 39; the auxiliary system, but at a very specific point in the back of the eye, so the patient would not have an impact on it, and hopefully the future patient's speed will be a macular decline sequence"

This test was intended to be Check the security of the procedure and it is held in patients with dry AMD who have already lost part of their vision. If successful, this type of gene therapy should only be achieved once, as it is estimated that its effects last and have planned to treat patients before they begin to experience vision loss.

Jane Osborne, 80, or Rydychen, has been the first person in the world to accept this innovative genital therapy, although nine more patients with AMD are taking part in the trial.

It is still too early to know if the treatment has been successful in preventing Osborne's vision loss, so the hospital will follow all patients who have the procedure closely.

In the words by the scientist at Cambridge University, he led the work on the supporting system that led to the creation of a Chiropract Therapeutic:

"We now have a better understanding of the relationship between the ACD and ACD system that led to the finding that recovery of the hyperactive support system balance could be a possible therapeutic approach in dry AMD."

This procedure was part of the FOCUS trial, sponsored by Gyroscope Therapeutics, a UK biotechnology company dedicated to developing products based on gen therapy, to treat eye diseases such as dry AMD. Part of the development of the viral vector was held at Oxford University, funded by the National Institute of Health Research (NIHR).

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