TY - JOUR
T1 - CRISPR/Cas9 for Human Genome Engineering and Disease Research
AU - Xiong, Xin
AU - Chen, Meng
AU - Lim, Wendell A.
AU - Zhao, Dehua
AU - Qi, Lei S.
N1 - Publisher Copyright:
Copyright © 2016 by Annual Reviews. All rights reserved.
PY - 2016
Y1 - 2016
N2 - The clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated 9 (Cas9) system, a versatile RNA-guided DNA targeting platform, has been revolutionizing our ability to modify, manipulate, and visualize the human genome, which greatly advances both biological research and therapeutics development. Here, we review the current development of CRISPR/Cas9 technologies for gene editing, transcription regulation, genome imaging, and epigenetic modification. We discuss the broad application of this system to the study of functional genomics, especially genome-wide genetic screening, and to therapeutics development, including establishing disease models, correcting defective genetic mutations, and treating diseases.
AB - The clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated 9 (Cas9) system, a versatile RNA-guided DNA targeting platform, has been revolutionizing our ability to modify, manipulate, and visualize the human genome, which greatly advances both biological research and therapeutics development. Here, we review the current development of CRISPR/Cas9 technologies for gene editing, transcription regulation, genome imaging, and epigenetic modification. We discuss the broad application of this system to the study of functional genomics, especially genome-wide genetic screening, and to therapeutics development, including establishing disease models, correcting defective genetic mutations, and treating diseases.
KW - gene editing
KW - gene regulation
KW - gene therapy
KW - genetic screening
KW - human diseases
UR - http://www.scopus.com/inward/record.url?scp=84984911537&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84984911537&partnerID=8YFLogxK
U2 - 10.1146/annurev-genom-083115-022258
DO - 10.1146/annurev-genom-083115-022258
M3 - Review article
C2 - 27216776
AN - SCOPUS:84984911537
SN - 1527-8204
VL - 17
SP - 131
EP - 154
JO - Annual Review of Genomics and Human Genetics
JF - Annual Review of Genomics and Human Genetics
ER -