• 21/04/2024 17:57

Gene therapy against dangerous hereditary diseases was carried out for the first time in Turkey


Mar 5, 2024

For the first time in the world, Turkish scientists have successfully used gene therapy against phenylketonuria – a disease that can cause permanent brain damage.

In addition, for the first time in Turkey, doctors used a non-surgical method of intracerebral gene therapy to treat GM1 gangliosidosis – dangerous hereditary disease of children, Ukrinform reports with reference to TRT Haber.

"Clinical Research Center "Phase-1" Faculty of Medicine at Gazi University was the first in the world to successfully use gene therapy against phenylketonuria, which can cause permanent brain damage, and for the first time in Turkey – a non-surgical method of intracerebral gene therapy for GM1 gangliosidosis, which is life-threatening for children,” – reports the TV channel.

Phenylketonuria – This is a hereditary disease characterized mainly by damage to the nervous system. If left untreated, it leads to mental retardation.

“We started this study as an international center, the only one in the world… This is important not only for many patients in our country, but throughout the world. We have now successfully used gene therapy in two of our PKU patients and will soon use it in a third patient. We will watch the results together in the coming days,” – Turkish scientists reported.

GM1 gangliosidosis – a fatal hereditary disease associated with lipid metabolism disorders that causes serious damage to the brain and various organs in children and can lead to death.

“Gene therapy for this disease was developed by a foreign sponsor; three centers in the world, including ours, were selected for its use. We use gene therapy for this disease directly in the brain, rather than intravenously. It involves injecting medication directly into the brain using a needle under tomography without surgery,” – scientists explained.

For Turkey, this procedure was performed for the first time; two Turkish patients became the 5th and 6th patients in the world who received such treatment.

As noted in the publication , Ghazi University also conducts other unique research. “In addition, we are trying to make a local gene therapy drug for another disease in our own laboratory. If we succeed, we will conduct clinical studies of this drug in the coming years.” said the deputy head of the center, Professor Fatih Ezgu.

As Ukrinform reported, in March of this year, a unique unit was opened at the Western Ukrainian Specialized Children's Medical Center, which will perform immunogenetic examinations for patients before and after transplantation, as well as for children with oncological and nephrological diseases.

Photo: Anadolu Ajansı


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