In this article, Merienne et al. designed a smart lentiviral vector system to improve the safety for genome editing of CNS disease gene. They characterized their CRISPR self-inactivating system (KamiCas9) for permanent disruption of huntingtin gene in the context of Huntington’s disease (HD).
The potency of LV-KamiCas9 was demonstrated in mouse primary neuronal/glial cultures, in the striatum of mice, and in patient-specific iPSC neuronal derivatives. They showed that the kinetics of Cas9 expression shortly followed by self-inactivation provided high on-target editing while progressively inactivating the nuclease, therefore preventing further on- and off-target activities.
R&D GEG Tech team works for providing high performance CRISPR/Cas9 lentiviral vector systems with smart designs for CRISPR: inducible systems, self-inactivating systems, non-integrating LVs, RNA LVs (LV which remains in RNA form), multiple gRNAs expression, specific pseudotypes etc…
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