Researchers have used gene editing in mice to reduce a repetitive behavior linked to autism, the Telegraph reports, adding that this suggests that gene editing could be applied to a number of brain-linked conditions.
In a paper appearing in Nature Biomedical Engineering this week, researchers from the University of Texas Health Science Center at San Antonio report that they were able to use CRISPR–Gold, a non-viral CRISPR–Cas9 delivery method using gold nanoparticles, to edit various types of brain cells. They then designed the machinery to target the metabotropic glutamate receptor 5 (mGluR5) gene, which they reported led to a decrease in mGluR5 in the striatum when they injected it into mouse brains. They noted that mGluR5 has been explored as a potential treatment target for fragile X syndrome and other autism spectrum disorders.
In a mouse model of fragile X syndrome, editing mGluR5 in this way reduced the mice's repetitive behaviors, the researchers report.
"There are no treatments or cures for autism yet, and many of the clinical trials of small-molecule treatments targeting proteins that cause autism have failed," senior author Hye Young Lee from UT Health San Antonio tells the Telegraph. "This is the first case where we were able to edit a causal gene for autism in the brain and show rescue of the behavioral symptoms."