title: [Efficient genome editing in human pluripotent stem cells through CRISPR/Cas9].
abstract: The RNA-guided CRISPR (clustered regularly interspaced short palindromic repeat)-associated Cas9 nuclease has offered a new platform for genome editing with high efficiency. Here, we report the use of CRISPR/Cas9 technology to target a specific genomic region in human pluripotent stem cells. We show that CRISPR/Cas9 can be used to disrupt a gene by introducing frameshift mutations to gene coding region; to knock in specific sequences (e.g. FLAG tag DNA sequence) to targeted genomic locus via homology directed repair; to induce large genomic deletion through dual-guide multiplex. Our results demonstrate the versatile application of CRISPR/Cas9 in stem cell genome editing, which can be widely utilized for functional studies of genes or genome loci in human pluripotent stem cells.
authors.affiliation: Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
authors.forename: Gai-gai
authors.initials: GG
authors.lastname: Liu
authors.affiliation: Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
authors.forename: Shuang
authors.initials: S
authors.lastname: Li
authors.affiliation: Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
authors.forename: Yu-da
authors.initials: YD
authors.lastname: Wei
authors.affiliation: Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
authors.forename: Yong-xian
authors.initials: YX
authors.lastname: Zhang
authors.affiliation: Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
authors.forename: Qiu-rong
authors.initials: QR
authors.lastname: Ding
mesh_terms: D064113:CRISPR-Cas Systems / Q000235:genetics*; D016368:Frameshift Mutation; D015894:Genome, Human / Q000235:genetics*; D006801:Humans; D039904:Pluripotent Stem Cells / Q000378:metabolism*; D017393:RNA Editing; D000094704:RNA, Guide, CRISPR-Cas Systems / Q000235:genetics
chemical_list: D000094704:RNA, Guide, CRISPR-Cas Systems