TürkiyeResearch and clinical trials
Gene therapy trials for GM1 gangliosidosis and OTC deficiency at Gazi Hospital
Based on a single news source or an institutional announcement. The level of scientific evidence is low; check the details with a doctor.
Two gene therapy clinical trials are being carried out at Gazi University. They are run by the Phase 1 Clinical Research Center of Gazi University Faculty of Medicine Hospital. The partner is the gene therapy center of the University of Pennsylvania in the USA. The studies cover GM1 gangliosidosis and OTC deficiency. OTC deficiency is a urea cycle disorder. According to the hospital, Türkiye became the fourth country authorized to carry out these studies. The three previous countries were the United Kingdom, the USA and Australia.
What does it mean for patients and families?
There are clinical trials for children with these diseases. Families can consult a pediatric metabolic disease specialist about eligibility requirements.
Source
Gazi Üniversitesi: Gazi Hastanesi'nden Dünyada İlk Kez Uygulanan "İki Gen Tedavisi" Çalışması
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: GM1 gangliosidosis
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.