Nature Communications (May 2021)
Tissue-specific activation of gene expression by the Synergistic Activation Mediator (SAM) CRISPRa system in mice
- Charleen Hunt,
- Suzanne A. Hartford,
- Derek White,
- Evangelos Pefanis,
- Timothy Hanna,
- Clarissa Herman,
- Jarrell Wiley,
- Heather Brown,
- Qi Su,
- Yurong Xin,
- Dennis Voronin,
- Hien Nguyen,
- Judith Altarejos,
- Keith Crosby,
- Jeffery Haines,
- Sarah Cancelarich,
- Meghan Drummond,
- Sven Moller-Tank,
- Ryan Malpass,
- Jacqueline Buckley,
- Maria del Pilar Molina-Portela,
- Gustavo Droguett,
- David Frendewey,
- Eric Chiao,
- Brian Zambrowicz,
- Guochun Gong
Affiliations
- Charleen Hunt
- Regeneron Pharmaceuticals, Inc.
- Suzanne A. Hartford
- Regeneron Pharmaceuticals, Inc.
- Derek White
- Regeneron Pharmaceuticals, Inc.
- Evangelos Pefanis
- Regeneron Pharmaceuticals, Inc.
- Timothy Hanna
- Regeneron Pharmaceuticals, Inc.
- Clarissa Herman
- Regeneron Pharmaceuticals, Inc.
- Jarrell Wiley
- Regeneron Pharmaceuticals, Inc.
- Heather Brown
- Regeneron Pharmaceuticals, Inc.
- Qi Su
- Regeneron Pharmaceuticals, Inc.
- Yurong Xin
- Regeneron Pharmaceuticals, Inc.
- Dennis Voronin
- Regeneron Pharmaceuticals, Inc.
- Hien Nguyen
- Regeneron Pharmaceuticals, Inc.
- Judith Altarejos
- Regeneron Pharmaceuticals, Inc.
- Keith Crosby
- Regeneron Pharmaceuticals, Inc.
- Jeffery Haines
- Regeneron Pharmaceuticals, Inc.
- Sarah Cancelarich
- Regeneron Pharmaceuticals, Inc.
- Meghan Drummond
- Regeneron Pharmaceuticals, Inc.
- Sven Moller-Tank
- Regeneron Pharmaceuticals, Inc.
- Ryan Malpass
- Regeneron Pharmaceuticals, Inc.
- Jacqueline Buckley
- Regeneron Pharmaceuticals, Inc.
- Maria del Pilar Molina-Portela
- Regeneron Pharmaceuticals, Inc.
- Gustavo Droguett
- Regeneron Pharmaceuticals, Inc.
- David Frendewey
- Regeneron Pharmaceuticals, Inc.
- Eric Chiao
- Regeneron Pharmaceuticals, Inc.
- Brian Zambrowicz
- Regeneron Pharmaceuticals, Inc.
- Guochun Gong
- Regeneron Pharmaceuticals, Inc.
- DOI
- https://doi.org/10.1038/s41467-021-22932-4
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 14
Abstract
CRISPR-Cas9 is a gene editing tool that can be used to modulate gene expression. Here, the authors report the generation of a mouse model that express all components of the CRISPR-Cas9 guide directed Synergistic Activation Mediator (SAM), demonstrate that gene activation can be achieved with various delivery methods and include generation of a disease model of hypercholesterolemia