Development and Applications of CRISPR-Cas9 for Genome Engineering

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 g...

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Main Authors: Hsu, Patrick, Lander, Eric Steven, Zhang, Feng
Other Authors: Massachusetts Institute of Technology. Department of Biological Engineering
Format: Article
Language:en_US
Published: Elsevier 2017
Online Access:http://hdl.handle.net/1721.1/111575
https://orcid.org/0000-0003-2782-2509
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author Hsu, Patrick
Lander, Eric Steven
Zhang, Feng
author2 Massachusetts Institute of Technology. Department of Biological Engineering
author_facet Massachusetts Institute of Technology. Department of Biological Engineering
Hsu, Patrick
Lander, Eric Steven
Zhang, Feng
author_sort Hsu, Patrick
collection MIT
description 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.
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spelling mit-1721.1/1115752022-10-01T21:00:28Z Development and Applications of CRISPR-Cas9 for Genome Engineering Hsu, Patrick Lander, Eric Steven Zhang, Feng Massachusetts Institute of Technology. Department of Biological Engineering McGovern Institute for Brain Research at MIT Hsu, Patrick Lander, Eric Steven Zhang, Feng 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. 2017-09-15T20:04:07Z 2017-09-15T20:04:07Z 2014-06 Article http://purl.org/eprint/type/JournalArticle 0092-8674 1097-4172 http://hdl.handle.net/1721.1/111575 Hsu, Patrick D. et al. "Development and Applications of CRISPR-Cas9 for Genome Engineering." Cell 157, 6 (June 2014): 1262-1278 © 2014 Elsevier Inc https://orcid.org/0000-0003-2782-2509 en_US http://dx.doi.org/10.1016/j.cell.2014.05.010 Cell Creative Commons Attribution-NonCommercial-NoDerivs License http://creativecommons.org/licenses/by-nc-nd/4.0/ application/pdf Elsevier PMC
spellingShingle Hsu, Patrick
Lander, Eric Steven
Zhang, Feng
Development and Applications of CRISPR-Cas9 for Genome Engineering
title Development and Applications of CRISPR-Cas9 for Genome Engineering
title_full Development and Applications of CRISPR-Cas9 for Genome Engineering
title_fullStr Development and Applications of CRISPR-Cas9 for Genome Engineering
title_full_unstemmed Development and Applications of CRISPR-Cas9 for Genome Engineering
title_short Development and Applications of CRISPR-Cas9 for Genome Engineering
title_sort development and applications of crispr cas9 for genome engineering
url http://hdl.handle.net/1721.1/111575
https://orcid.org/0000-0003-2782-2509
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