Neuronal Cell-type Engineering by Transcriptional Activation
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Updated: Oct 15, 2025

Intraventricular Transplantation of Engineered Neuronal Precursors for In Vivo Neuroarchitecture Studies
Published on: May 11, 2019
Songlei Liu1,2, Johannes Striebel3, Giovanni Pasquini3
1Department of Genetics, Blavatnik Institute, Harvard Medical School, Boston, MA, United States.
Abstract:
Gene activation with the CRISPR-Cas system has great implications in studying gene function, controlling cellular behavior, and modulating disease progression. In this review, we survey recent studies on targeted gene activation and multiplexed screening for inducing neuronal differentiation using CRISPR-Cas transcriptional activation (CRISPRa) and open reading frame (ORF) expression. Critical technical parameters of CRISPRa and ORF-based strategies for neuronal programming are presented and discussed. In addition, recent progress on in vivo applications of CRISPRa to the nervous system are highlighted. Overall, CRISPRa represents a valuable addition to the experimental toolbox for neuronal cell-type programming.
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