Brain organoids in Parkinson's disease drug development: Human-specific models for translational discovery.
Brain organoids in Parkinson's disease drug development: Human-specific models for translational discovery.
Where did the research take place?
The study site has not been established. Author addresses may differ from where the research occurred.
Luxembourg, LU · Author affiliation
Developmental and Cellular Biology, Luxembourg Centre for Systems Biomedicine (LCSB), University of Luxembourg, 2, place de l'Université, L-4365 Esch-sur-Alzette, Luxembourg; OrganoTherapeutics SARL, 29 Rue Henri Koch, L-4354 Esch-sur-Alzette, Luxembourg.Location evidence
Esch-sur-Alzette, LU · Author affiliation
Developmental and Cellular Biology, Luxembourg Centre for Systems Biomedicine (LCSB), University of Luxembourg, 2, place de l'Université, L-4365 Esch-sur-Alzette, Luxembourg; OrganoTherapeutics SARL, 29 Rue Henri Koch, L-4354 Esch-sur-Alzette, Luxembourg.Location evidence
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Original abstract
Parkinson's disease (PD) poses a major unmet therapeutic challenge, with most drug candidates failing in clinical translation despite promising animal model data. Human induced pluripotent stem cell-derived midbrain organoids recapitulate key PD pathological hallmarks - including dopaminergic neuron loss, α-synuclein aggregation, and neuroinflammation - in a genetically defined, human-specific context. This review summarizes drug screening studies in midbrain organoids across genetic, toxin-based, and α-synuclein preformed fibril models. We highlight therapeutic interventions that rescue PD phenotypes and discuss the personalized medicine potential of patient-derived organoids. We also critically assess current limitations and outline how artificial intelligence integration and assembloid platforms are advancing organoid-based drug discovery towards regulatory acceptance.