The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes

The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes - Gene editing

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 […]