AI screening in brain organoids uncovers two drug candidates for Leigh syndrome

Mon, 20 Apr 2026 17:20:06 +0200

The need for medical treatments for rare diseases such as Leigh syndrome is high. However, low patient numbers make research into treatments difficult. Together with a team from the University of Luxembourg, researchers from Heinrich Heine University Düsseldorf (HHU) and University Hospital Düsseldorf (UKD) have succeeded in utilizing artificial intelligence (AI) to establish a model that enables a better understanding of Leigh syndrome. In the course of this work, they succeeded in identifying new drug candidates for the treatment of the disease. The study is now published in Nature Communications.