A platform bridging human biology and artificial intelligence: Deep machine learning–powered genome engineering technology brain organoid platform accelerates drug discovery for lethal childhood disorders

21.04.26

Dr Paweł Lisowski, a genome engineer and human stem cell researcher from the Institute of Genetics and Animal Biotechnology of the Polish Academy of Sciences (Jastrzębiec), together with an international team of researchers, report in Nature Communications the development of a new drug discovery platform that combines human genome engineering, stem cell–based brain organoids, and deep machine learning to identify therapeutic candidates for severe neurodevelopmental and mitochondrial diseases.

Built on human genome engineering and brain organoid technologies, the platform enables direct screening of drugs in patient-specific human brain models. The researchers demonstrate its power using rare, lethal childhood disorders with no effective treatment - as a model system.

Further reading at:  Menacho C et al. Accelerating Leigh syndrome drug discovery through deep learning screening in brain organoids. Nature Communications 2026 DOI:10.1038/s41467-026-71391-2