The Development of an AAV-Based CRISPR SaCas9 Genome Editing System That Can Be Delivered to Neurons and Regulated via Doxycycline and Cre-Recombinase.

The RNA-guided Cas9 nuclease, from the type II prokaryotic clustered regularly interspersed short palindromic repeats (CRISPR) adaptive immune system, has been adapted by scientists to enable site specific genome editing of eukaryotic cells both and . Previously, we reported the development of an adeno-associated virus (AAV)-mediated CRISPR (Sp) Cas9 system, in which the genome editing function can be regulated by controlling the expression of the guide RNA (sgRNA) in a doxycycline (Dox)-dependent manner. Here, we report the development of an AAV vector tool kit utilizing the Cas9 from (SaCas9).