Technologies that can edit genes within the brain have the potential to revolutionize how we treat neurological disorders, and also generate new insights into brain function. To date, attempts to use clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing in the brain has relied on viral delivery of the Cas9 enzyme, but this approach is challenging. Researchers at the University of Texas (UT) Health Science Center at San Antonio, and the University of California, Berkeley, have now demonstrated use of nonviral CRISPR-Cas9 delivery system known as CRISPR-Gold to edit different cell types within the brain. The team then used the CRISPR-Gold technology to target a single gene responsible for a form of autism known as fragile X syndrome (FXS), in a mouse model. They found that the treatment effectively rescued animals from the exaggerated repetitive behaviours that are symptomatic of the disorder. The researchers, headed by ...
Original Article: Editing Autism Gene with Nano-CRISPR Curbs Repetitive Behaviors