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Modeling striatal development and disease with human pluripotent stem cells - PubMed

3 hours ago
  • #striatum
  • #medium spiny neurons
  • #pluripotent stem cells
  • The striatum is crucial for motor control, cognition, reward processing, and habit formation, with dysfunction linked to neurological and psychiatric disorders.
  • Human pluripotent stem cells (PSCs) offer a valuable platform for modeling human striatal development and disease, overcoming limitations of animal models due to species-specific differences.
  • Current approaches focus on generating medium spiny neurons (MSNs) from PSCs, emphasizing dorsal and ventral striatal specification and the protracted maturation of human MSNs.
  • Advances in differentiation strategies, including small molecule patterning, transcription factor induction, and 3D organoid/assembloid systems, enhance model efficiency and complexity.
  • PSC-derived striatal models are primarily used in Huntington's disease research, revealing early developmental, transcriptional, synaptic, and network abnormalities.
  • Recent studies extend these models to other neurological conditions and incorporate circuit-level analyses using cortico-striatal assembloids.
  • Single-cell and single-nucleus transcriptomic datasets provide reference frameworks for benchmarking PSC-derived striatal cell identity and maturation.
  • Current challenges include incomplete maturation, limited non-neuronal cell representation, and restricted applicability to psychiatric disorders.
  • Future directions involve integrating developmental biology, multi-omics resources, and advanced in vitro strategies to improve models for translational neuroscience.