Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
10145047 | Current Opinion in Biomedical Engineering | 2018 | 17 Pages |
Abstract
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.
Keywords
Related Topics
Physical Sciences and Engineering
Engineering
Biomedical Engineering
Authors
Christina Marie Fuentes, David Vernon Schaffer,