Development and Applications of CRISPR-Cas9 for Genome Engineering
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Lander_Development and.pdf
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Author(s) • •
Hsu, Patrick
Lander, Eric Steven
Zhang, Feng
Date Issued
June 2014
Journal
Cell
Publisher
Elsevier
Citation
Hsu, Patrick D. et al. "Development and Applications of CRISPR-Cas9 for Genome Engineering." Cell 157, 6 (June 2014): 1262-1278 © 2014 Elsevier Inc
Version
Author's final manuscript
Abstract
Recent advances in genome engineering technologies based on the CRISPR-associated RNA-guided endonuclease Cas9 are enabling the systematic interrogation of mammalian genome function. Analogous to the search function in modern word processors, Cas9 can be guided to specific locations within complex genomes by a short RNA search string. Using this system, DNA sequences within the endogenous genome and their functional outputs are now easily edited or modulated in virtually any organism of choice. Cas9-mediated genetic perturbation is simple and scalable, empowering researchers to elucidate the functional organization of the genome at the systems level and establish causal linkages between genetic variations and biological phenotypes. In this Review, we describe the development and applications of Cas9 for a variety of research or translational applications while highlighting challenges as well as future directions. Derived from a remarkable microbial defense system, Cas9 is driving innovative applications from basic biology to biotechnology and medicine.
MIT Department
Massachusetts Institute of Technology. Department of Biological Engineering
McGovern Institute for Brain Research at MIT
Terms of Use
Creative Commons Attribution-NonCommercial-NoDerivs License
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DOI of Published Version
https://doi.org/10.1016/j.cell.2014.05.010