title: CRISPR/Cas9 for Human Genome Engineering and Disease Research.
abstract: 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.
authors.affiliation: Department of Cellular and Molecular Pharmacology, University of California, San Francisco, California 94158; email: xiongxin1009@gmail.com , wendell.lim@ucsf.edu.
authors.forename: Xin
authors.initials: X
authors.lastname: Xiong
authors.affiliation: Department of Bioengineering, Stanford University, Stanford, California 94305; email: dehua@stanford.edu , stanley.qi@stanford.edu.|Department of Chemical and Systems Biology, Stanford University, Stanford, California 94305.|ChEM-H, Stanford University, Stanford, California 94305.|Gladstone Institute of Cardiovascular Disease, San Francisco, California 94158; email: meng.chen@gladstone.ucsf.edu.
authors.forename: Meng
authors.initials: M
authors.lastname: Chen
authors.affiliation: Department of Cellular and Molecular Pharmacology, University of California, San Francisco, California 94158; email: xiongxin1009@gmail.com , wendell.lim@ucsf.edu.
authors.forename: Wendell A
authors.initials: WA
authors.lastname: Lim
authors.affiliation: Department of Bioengineering, Stanford University, Stanford, California 94305; email: dehua@stanford.edu , stanley.qi@stanford.edu.
authors.forename: Dehua
authors.initials: D
authors.lastname: Zhao
authors.affiliation: Department of Bioengineering, Stanford University, Stanford, California 94305; email: dehua@stanford.edu , stanley.qi@stanford.edu.|Department of Chemical and Systems Biology, Stanford University, Stanford, California 94305.|ChEM-H, Stanford University, Stanford, California 94305.
authors.forename: Lei S
authors.initials: LS
authors.lastname: Qi
mesh_terms: D064113:CRISPR-Cas Systems / Q000235:genetics*; D000072669:Gene Editing / Q000379:methods* / Q000639:trends; D005786:Gene Expression Regulation; D030342:Genetic Diseases, Inborn / Q000628:therapy; D015316:Genetic Therapy; D015894:Genome, Human; D006801:Humans; D000094704:RNA, Guide, CRISPR-Cas Systems / Q000235:genetics / Q000627:therapeutic use
chemical_list: D000094704:RNA, Guide, CRISPR-Cas Systems
keywords: gene editing; gene regulation; gene therapy; genetic screening; human diseases