RNA-guided transcriptional silencing in vivo with S. aureus CRISPR-Cas9 repressors

Repression of gene transcription using CRISPR-Cas9 has been achieved in vitro but not for delivery into adult animal models. Here, the authors use AAV8 to deliver the transcriptional repressor dSaCas9KRAB to the cholesterol regulator Pcsk9, and show repression up to 24 weeks and reduced cholesterol...

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Auteurs principaux: Pratiksha I. Thakore, Jennifer B. Kwon, Christopher E. Nelson, Douglas C. Rouse, Matthew P. Gemberling, Matthew L. Oliver, Charles A. Gersbach
Format: article
Langue:EN
Publié: Nature Portfolio 2018
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Accès en ligne:https://doaj.org/article/46f1559feeb54a51902bab3a11b455bc
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Résumé:Repression of gene transcription using CRISPR-Cas9 has been achieved in vitro but not for delivery into adult animal models. Here, the authors use AAV8 to deliver the transcriptional repressor dSaCas9KRAB to the cholesterol regulator Pcsk9, and show repression up to 24 weeks and reduced cholesterol levels in mice.