Pathogenic windows and rescue thresholds

Define developmental windows and minimum repair thresholds for ASD risk mutations using isogenic iPSC-derived brain organoids and assembloids.

Using isogenic iPSC-derived brain organoids and assembloids, we define pathogenic windows and minimum rescue thresholds for representative ASD risk mutations, integrating time-series single-cell multi-omics and RNA base editing.

Research aims

  • Generate isogenic iPSC lines carrying representative ASD risk mutations.
  • Build time-series single-cell transcriptomic and epigenomic atlases of brain organoids and assembloids.
  • Map developmental stages at which mutations exert causal effects.
  • Test RNA base-editing rescue at different developmental windows.

This project establishes a parameter framework to support rational intervention design for neurodevelopmental disorders.