A remarkable new study has successfully used the CRISPR-Cas9 gene editing technique to edit a specific gene in mice engineered to have fragile X syndrome (FXS), a single-gene disorder often related to autism.
The single gene edit in the live mice resulted in significant improvements in repetitive and obsessive behaviors, making this the first time gene editing has been used to effectively target behavioral symptoms related to autism spectrum disorder (ASD).
FXS is a genetic disorder associated with intellectual disability, seizures and exaggerated repetitive behavior.
Previous studies have shown that the repetitive behaviors associated with FXS are related to a specific excitatory receptor in the brain that, when dysregulated, causes exaggerated signaling between cells.
The CRISPR technique homes in on the gene that controls that excitatory receptor, the metabotropic glutamate receptor 5 (mGluR5), and essentially disables it, dampening the excessive signaling the corresponds with repetitive behaviors.
In mice treated with the new system, obsessive digging behavior was reduced by 30 percent and repetitive leaping actions dropped by 70 percent.
An even more fascinating element of this new research was the novel CRISPR-Cas9 delivery method, pioneered by a team at the University of California, Berkeley.
The most commonly used gene delivery technique for CRISPR harnesses the power of viruses to ferry the Cas9 enzyme to a targeted cell.
But viral gene delivery has its limitations. As well as battling the potential for a person’s immune system to develop antibodies against the virus, this delivery system makes it difficult to control how much Cas9 is ultimately delivered.
The effectiveness of this CRISPR-Gold system in accurately editing specific genes in brain cells could in the future be potentially applied to a broad assortment of different genetic diseases, as well as targeting other social interaction symptoms associated with ASD.
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