It is one of the very first in Europe of this type and the only one in Abruzzo
The First experimental intervention of gene therapy for maculopathy tied to age. To conduct the operation, unique in its kind in Abruzzo and among the first in Europe, was Professor Rodolfo Mastropasqua, an ordinary of visual apparatus diseases at the Department of Neuroscience, Imaging and Clinical Sciences of the University. The therapeutic approach is revolutionary since it aims to correct genetic defects underlying the disease, through the introduction of a functioning copy of the damaged gene or an alternative gene capable of restoring compromised functions.
Maculopathy and today’s therapies
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Macular degeneration linked to age is a chronic pathology that affects the central part of the retina and which often manifests itself after 50 years. In Italy it hits over a million people And it is the main cause of blindness and hypovision in the population over 65 years. It is a disease that can affect both eyes, even at different times in life, and which initially presents itself with a distorted and blurred vision, until the central view is completely compromised and the patients lose the ability to carry out The most basic daily activities. Maculopathy exists in two forms: dry and wet. The latter is currently treated with anti-vegf drugs, administered via intravitreal injections on a monthly or bimonthly basis. Although effective, therapy has significant limits, primarily the need for frequent administrations in the long run, with an important impact both from a psychological point of view on and logistical patients; To this is added the significant economic burden for the health system.
Maculopathy and experimental gene therapy
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The experimental treatment adopted in Chieti is based on the use of a Benign viral vectorintroduced directly into the eye. A safe virus, engineered to transport genetic information to retina cells, inducing them to independently produce anti-vegf molecules. “With gene therapy we teach the retina to produce anti-vegf independently with the final goal of making a single intervention that will last all life,” explained Mastropasqua. The goal? Replace the current continuous therapeutic regime with a single, potentially definitive procedure.
An important milestone
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The Ocular Genetics Center of the University of Annunzio and the ASL of Chieti is among the very few in Europe to practice this technique and be involved in international experimentation. According to what is reported in an official note, “maintaining and preserving view with a single intervention, especially if carried out early, can not only stop but even improve the quality of the vision and life of these patients and at the same time remarkable economic savings for the state”. The next step is now waiting for the analysis of cumulative data from other centers in the world involved in the experimentation, to thus evaluate the effectiveness of large -scale gene technique and in the long run.
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