Breakthrough in Leigh syndrome therapy: research involving IGAB PAS published in Cell (IF: 42,5)

12.03.26

Scientists from the Institute of Genetics and Animal Biotechnology of the Polish Academy of Sciences (IGAB PAS, Jastrzębiec) report in Cell (Zink et al., 2026) the development of a breakthrough therapeutic strategy for Leigh syndrome (LS), a severe neurodevelopmental disease that is lethal in children and previously had no effective treatment.

Using patient-derived stem cells and patient-specific 3D brain organoid models, the international research team developed a new treatment approach that significantly improves disease outcomes. The therapy showed strong benefits in Leigh syndrome patients, where treated children demonstrated improved motor abilities, increased resistance to metabolic crises, and were able to return to normal daily life and attend school.

The work was carried out by stem cell scientist and human genome engineer Paweł Lisowski from IGAB PAS together with outstanding collaborators from leading international research institutions, enabled by Dr. Lisowski’s genome engineering technologies that allowed generation of first and precise patient-specific stem-cell and 3D brain organoid model systems of the disease that formed the basis for this discovery.

Zink et al., 2026, Cell 189, 1–24, March 19, 2026 © https://doi.org/10.1016/j.cell.2026.02.008