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At the clinical level, EDIT-101 is the first use of a genome editor in the CNS and is currently in phase 1/2 clinical trials in adult and pediatric patients ( Modified CRISPR activation Using the traditional CRISPR-Cas9 complex as a basis, a modified version in which the Cas9 enzyme is inactive or dead (dCas9) has been developed [64]

Biosci 3 (6), 237248

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