Open Seminar by Prof Alessandro Prigione and Prof Jürgen Knoblich

Wednesday 26 August 2026, 9 am - 11 am
Lecture Speakers: Prof Alessandro Prigione , Prof Jürgen Knoblich
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Presented by the Hands-on Summer Course: Human Stem Cell-Derived Neuronal Networks in Health & Disease of the Medical Neuroscience Department, Radboudumc, DCMN

9:00 – 09:50 | Professor Dr. Alessandro Prigione, M.D., Ph.D., Düsseldorf, Germany

Accelerating mitochondrial disease drug discovery with brain organoid models

Abstract: Mitochondrial diseases, caused by pathogenic variants in the nuclear or mitochondrial DNA, represent a major therapeutic challenge due to the paucity of effective model systems. One of the most severe forms of mitochondrial disease is Leigh syndrome (LS), a devastating neurological disease with no cure that leads to early death in children. New Approach Methodologies (NAMs), such as induced pluripotent stem cells (iPSCs) and derived organoids, offer unprecedented opportunities for building alternative human-relevant models. In this talk, I will present our ongoing efforts in advancing the understanding and therapies for LS using patient-derived iPSCs and brain organoids as NAMs. I will show examples of innovative interventions for LS that we identified through iPSC-driven deep learning screening or neuronal high-content screening. 

A repurposable drug that we discovered has received the designation of Orphan Drug from the European Medicines Agency (EMA) for the treatment of LS, and for this a clinical trial is now under development. Through collaboration with clinical scientists, we hope to translate our experimental findings into concrete therapies for incurable pediatric mitochondrial diseases with highly unmet medical needs.

Alessandro Prigione

Alessandro Prigione

Dr. Alessandro Prigione is a tenured Associate Professor of Pediatric Metabolic Medicine in the Department of General Pediatrics at Heinrich Heine University in Düsseldorf, Germany. He received a MD from the University of Milan Italy in 2002 and a PhD from the San Raffaele University in Italy in 2008. His lab employs induced pluripotent stem cells (iPSCs) and derived neurons and brain organoids for disease modeling and drug discovery of rare mitochondrial neurological disorders, with a specific focus is on Leigh syndrome. Dr. Prigione is a member of the scientific council of AFM Telethon, of the scientific committees of Mitocon and Cure Mito, and the current Editor-in-Chief of the journal Stem Cell Research (Elsevier). He is the coordinator of international consortia focusing on the use of iPSCs for advancing mitochondrial disease treatments (CureMILS from EJPRD, SildeMITO from AFM, SynLeigh from ERDERA). A repurposable drug identified by the Prigione group following an iPSC-based approach has recently received the designation of Orphan Drug from the European Medicines Agency (EMA) for the treatment of Leigh syndrome, and for this a clinical trial is now under development.

10:00-10:50 | Professor Dr. Jürgen A. Knoblich, Ph.D., Vienna, Austria

Cerebral Organoids: Growing human brain tissue from stem cells to study development and disease

Abstract:The human brain is unique in its size and complexity. We have developed cerebral organoids, 3D cell cultures derived from patient stem cells that recapitulate the development of the human brain. Using this technology, we have identified developmental processes unique to humans, studied the mechanistic basis for brain diseases and reconstituted human neural network activity (Lancaster et al., Nature 2013; Esk et al., Science 2020; Eichmüller et al., Science 2022; Li et al., Nature 2023). Our efforts to recapitulate human specific processes of brain development and to replicate disease pathology on the circuit level will be presented. Specifically, I will cover our recent efforts to use electrophysiology and barcoded connectome analysis with single-cell resolution in order to determine and understand how neural network activity and network architecture change in patients that develop epilepsy

Jürgen Knoblich

Jürgen Knoblich

Jürgen Knoblich is deputy scientific director at the Institute of Molecular Biotechnology of the Austrian Academy of Sciences (IMBA) and Professor at the Medical University in Vienna. Originally trained as a Drosophila researcher, his research focuses on the development of the human brain and the study of neurodevelopmental disorders. He received his PhD from the University of Tübingen and postdoctoral training in the laboratory of Lily and Yuh-Nung Jan at UCSF, San Francisco. In 2013, the Knoblich group has established cerebral organoids, a groundbreaking new technology that allows reconstitution of human brain development starting from patient iPS cells at unprecedented detail. They have used this system for modelling various neurodevelopmental disorders human brain tissue. They were able to screen through entire sets of disease genes relevant for autism spectrum disorders and could demonstrate that neurodevelopmental disorders can arise from cell types not found in animal models.

When
Wednesday 26 August 2026, 9 am - 11 am
Speakers
Prof Alessandro Prigione , Prof Jürgen Knoblich
Location
Red Room, Trigon building (Kapittelweg 29, Nijmegen)
Organisation
Donders Institute for Brain, Cognition and Behaviour