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Neuronal Cell-type Engineering by Transcriptional Activation.
Liu, Songlei; Striebel, Johannes; Pasquini, Giovanni; Ng, Alex H M; Khoshakhlagh, Parastoo; Church, George M; Busskamp, Volker.
Afiliação
  • Liu S; Department of Genetics, Blavatnik Institute, Harvard Medical School, Boston, MA, United States.
  • Striebel J; Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, United States.
  • Pasquini G; Department of Ophthalmology, Medical Faculty, University of Bonn, Bonn, Germany.
  • Ng AHM; Department of Ophthalmology, Medical Faculty, University of Bonn, Bonn, Germany.
  • Khoshakhlagh P; GC Therapeutics, Inc, Cambridge, MA, United States.
  • Church GM; GC Therapeutics, Inc, Cambridge, MA, United States.
  • Busskamp V; Department of Genetics, Blavatnik Institute, Harvard Medical School, Boston, MA, United States.
Front Genome Ed ; 3: 715697, 2021.
Article em En | MEDLINE | ID: mdl-34713262
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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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Front Genome Ed Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Front Genome Ed Ano de publicação: 2021 Tipo de documento: Article