Key result
An intein-mediated split-Cas9 system demonstrated nuclease activity comparable to wild-type Cas9 across 3 endogenous genes and was efficiently delivered and reconstituted via rAAV.
An intein-mediated split-Cas9 system successfully bypasses rAAV packaging limits while maintaining nuclease activity comparable to wild-type Cas9.
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Supports split-Cas9 AAV delivery in animal models; leaves open human translation and safety.
Truong et al. (2015) studied Gene therapy. Intein-mediated split-Cas9 system vs. Wild-type Cas9 was evaluated on Nuclease activity. An intein-mediated split-Cas9 system demonstrated nuclease activity comparable to wild-type Cas9 across 3 endogenous genes and was efficiently delivered and reconstituted via rAAV.
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