Adeno-associated virus-mediated delivery of CRISPR-Cas9 for genome editing in the central nervous system.

The emergence of CRISPR-Cas9 as a powerful genome editing tool has led to several studies exploring its potential to treat neurological disorders. Cas9 and its sgRNA can be readily engineered to target any gene and can be multiplexed to target several genes at once. Furthermore, the use of adeno-associated virus (AAV) to deliver with Cas9 and its sgRNA is a promising therapeutic combination with strong potential to reach the clinic. Here we discuss how Cas9 editing has been utilized for gene insertion, knockout, and deletion in vivo for applications in the central nervous system (CNS). Furthermore, we highlight major challenges that remain for AAV-Cas9-sgRNA clinical translation.