The first successful gene therapy in Poland that inhibits vision loss takes place

The first successful gene therapy in Poland that inhibits vision loss and even restores vision was carried out by doctors from Poznań. This is an opportunity for patients with genetic retinal diseases.

The Ophthalmology Department of the Clinical Hospital Heliodora Święcicki in Poznań is the only centre in the country that received the certification required by the European Medicines Agency, authorising the use of retinal gene therapy.

There are nearly 280 types of retinal degeneration. These are rare ophthalmic diseases with a genetic background.

“Damage to one of the genes leads to degenerative changes in the retina, which causes the patient to see worse at an early age, have a narrowed field of view, difficulty in moving, abnormal twilight vision, and over time, there is a very large degeneration of cells responsible for seeing light,” explains professor Marcin Stopa, Head of the Clinical Department of Ophthalmology at the Clinical Hospital H. Święcicki in Poznań.

The vast majority of these diseases are incurable. The exception is Leber's congenital blindness - an inherited genetic disease caused by a defect of several genes. This degeneration was the first in Poland to be treated by doctors from Poznań, who replaced the damaged gene with a healthy DNA thread.

“Using very small incisions, you need to enter the eye and administer the medicine containing viral particles directly under the retina. In their centre there is a DNA strand that will produce a healthy protein that will replace the malfunctioning one in a given patient, " emphasized Professor Stopa.

“The treatments performed by the doctors from Poznań save from blindness. We hope to restore function to these remaining photoreceptors and to ensure that the patient, who is already on the verge of blindness, will improve his vision, visual independence and halt further disease progression,” professor Maciej Krawczyński from the Medical University of Karol Marcinkowski in Poznań highlighted.

So far, about 40 such procedures have been performed in Europe, and about 140 in the world. Eyesight defects treatable with the above-described method are diagnosed in three out of 100,000 patients. Research into the use of gene therapies in other retinal diseases is ongoing.