The approach, which combines CRISPR with single-cell analyses of organoids, suggests that intermediate progenitor cells are especially vulnerable to mutations associated with autism.
Spectrum: Autism Research News
Tag: gene expression
Voles reared primarily by their fathers show altered synapse density.
The inhibitory cells misfire and contribute to social difficulties in mice that model the syndrome.
The discovery could help clinicians diagnose children who carry mutations in the gene, called SCN2A, and gauge their responses to potential therapies.
The approach prompts cultured cells to correct the genetic mutation in fragile X syndrome using their own DNA repair system, but it still needs to be tested further.
But the alternatives, including living-brain biopsies, raise logistical and ethical questions, experts say.
The findings add to the growing evidence that genes with disparate functions can play similar roles in brain development.