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

Pratiksha I. Thakore; Jennifer B. Kwon; Christopher E. Nelson; Douglas C. Rouse; Matthew P. Gemberling; Matthew L. Oliver; Charles A. Gersbach

doi:10.1038/s41467-018-04048-4

Nature Communications (Apr 2018)

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

Pratiksha I. Thakore,
Jennifer B. Kwon,
Christopher E. Nelson,
Douglas C. Rouse,
Matthew P. Gemberling,
Matthew L. Oliver,
Charles A. Gersbach

Affiliations

Pratiksha I. Thakore: Department of Biomedical Engineering, Duke University
Jennifer B. Kwon: Center for Genomic and Computational Biology, Duke University
Christopher E. Nelson: Department of Biomedical Engineering, Duke University
Douglas C. Rouse: Division of Laboratory Animal Resources, Duke University School of Medicine
Matthew P. Gemberling: Department of Biomedical Engineering, Duke University
Matthew L. Oliver: Department of Biomedical Engineering, Duke University
Charles A. Gersbach: Department of Biomedical Engineering, Duke University

DOI: https://doi.org/10.1038/s41467-018-04048-4
Journal volume & issue: Vol. 9, no. 1
pp. 1 – 9

Abstract

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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.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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