Research
Enhancer-targeted CRISPR-A rescues haploinsufficiency and mutant phenotypes in organoid models of autism
Nature · By George T. Chen ·
The Brief
- CRISPR activation directed at enhancers of the autism risk genes CHD8 and SCN2A selectively increased their expression.
- The approach reversed mutant phenotypes in both patient-derived and gene-edited neuron and organoid models.
- The study reports results in organoid models; the sources do not describe effects in animals or humans.
Why it matters
It shows that boosting expression of haploinsufficient autism risk genes through their enhancers can reverse disease-related cellular phenotypes in human models, a possible route for future therapeutic research.
Who’s affected
- autism researchers
- neuroscientists working with brain organoids
- gene regulation and genome-editing researchers
Nature Neuroscience, Published online: 13 November 2026; doi:10.1038/s41593-026-02504-9 CRISPR activation targeted to enhancers of autism risk genes CHD8 and SCN2A selectively increased expression and reversed mutant phenotypes in both patient-derived and gene-edited neuron and…
Timeline
Study published online in Nature Neuroscience (doi:10.1038/s41593-026-02504-9)
nature.com
Enhancer-targeted CRISPR-A rescues haploinsufficiency and mutant phenotypes in organoid models of autism