Scientists have for the first time successfully delivered the gene-editing tool CRISPR into mouse brains and altered their behavior.
In the method, a nanoparticle ferries the enzyme used in CRISPR into the brains of a mouse model of fragile X syndrome. The enzyme dampens a signaling pathway related to the syndrome, and lessens the mice’s exaggerated repetitive behaviors.
The results offer hope that the approach might treat fragile X syndrome as well as other conditions related to autism.