This month’s Going on Trial newsletter explores a new tool for communicating with clinical trial participants and their families, among other drug development news.
Spectrum: Autism Research News
The approach, which combines CRISPR with single-cell analyses of organoids, suggests that intermediate progenitor cells are especially vulnerable to mutations associated with autism.
The newfound DNA-cutting enzyme, called Fanzor, can be programmed to edit the human genome and could prove easier to deliver to cells than current CRISPR tools.
The discovery could help clinicians diagnose children who carry mutations in the gene, called SCN2A, and gauge their responses to potential therapies.
The gene-editing advances make it easier to target specific tissues in mice and detect off-target effects.
The findings add to the growing evidence that genes with disparate functions can play similar roles in brain development.
UBE3A, a key gene associated with both autism-linked conditions, can explain most — but not all — of the syndromes’ atypical neuronal properties.
CRISPR-edited prairie voles that lack receptors for the so-called “social hormone” still bond with their mate and pups, raising questions about the molecule’s role.
The model enables the study of autism-linked genes at the earliest stages of neural development.