[The contribution of cerebral organoids to the understanding and treatment of rare genetic diseases with neurodevelopmental disorders].
Fatima El ItLaurence FaivreChristel Thauvin-RobinetAntonio VitobelloLaurence DuplombPublished in: Medecine sciences : M/S (2024)
Rare genetic diseases with neurodevelopmental disorders (NDDs) encompass several heterogeneous conditions (autism spectrum disorder (ASD), intellectual disability (ID), attention deficit hyperactivity disorder (ADHD), specific learning disorder (SLD), among others). Currently, few treatments are available for these patients. The difficulty in accessing human brain samples and the discrepancies between human and animal models highlight the need for new research approaches. One promising approach is the use of the cerebral organoids. These 3D, self-organized structures, generated from induced pluripotent stem cells (iPSCs), enable the reproduction of the stages of human brain development, from the proliferation of neural stem cells to their differentiation into neurons, oligodentrocytes, and astrocytes. Cerebral organoids hold great promise in understanding brain development and in the search for treatments.
Keyphrases
- induced pluripotent stem cells
- autism spectrum disorder
- attention deficit hyperactivity disorder
- intellectual disability
- subarachnoid hemorrhage
- neural stem cells
- end stage renal disease
- cerebral ischemia
- newly diagnosed
- ejection fraction
- chronic kidney disease
- genome wide
- signaling pathway
- peritoneal dialysis
- prognostic factors
- high resolution
- endothelial cells
- copy number
- white matter
- big data
- resting state
- functional connectivity
- patient reported outcomes
- multiple sclerosis
- blood brain barrier