TY  - JOUR
A1  - Agudelo, Daniel
A1  - Carter, Sophie
A1  - Velimirovic, Minja
A1  - Duringer, Alexis
A1  - Rivest, Jean-François
A1  - Levesque, Sébastien
A1  - Loehr, Jeremy
A1  - Mouchiroud, Mathilde
A1  - Cyr, Denis
A1  - Waters, Paula J.
A1  - Laplante, Mathieu
A1  - Moineau, Sylvain
A1  - Goulet, Adeline
A1  - Doyon, Yannick
T1  - Versatile and robust genome editing with Streptococcus thermophilus CRISPR1-Cas9
Y1  - 2020/01/01 
JF  - Genome Research 
JO  - Genome Research 
SP  - 107 
EP  - 117 
DO  - 10.1101/gr.255414.119 
VL  - 30 
IS  - 1 
UR  - http://genome.cshlp.org/content/30/1/107.abstract 
N2  - Targeting definite genomic locations using CRISPR-Cas systems requires a set of enzymes with unique protospacer adjacent motif (PAM) compatibilities. To expand this repertoire, we engineered nucleases, cytosine base editors, and adenine base editors from the archetypal Streptococcus thermophilus CRISPR1-Cas9 (St1Cas9) system. We found that St1Cas9 strain variants enable targeting to five distinct A-rich PAMs and provide a structural basis for their specificities. The small size of this ortholog enables expression of the holoenzyme from a single adeno-associated viral vector for in vivo editing applications. Delivery of St1Cas9 to the neonatal liver efficiently rewired metabolic pathways, leading to phenotypic rescue in a mouse model of hereditary tyrosinemia. These robust enzymes expand and complement current editing platforms available for tailoring mammalian genomes. 
ER  -