RRC ID 44664
著者 Horii T, Tamura D, Morita S, Kimura M, Hatada I.
タイトル Generation of an ICF syndrome model by efficient genome editing of human induced pluripotent stem cells using the CRISPR system.
ジャーナル Int J Mol Sci
Abstract Genome manipulation of human induced pluripotent stem (iPS) cells is essential to achieve their full potential as tools for regenerative medicine. To date, however, gene targeting in human pluripotent stem cells (hPSCs) has proven to be extremely difficult. Recently, an efficient genome manipulation technology using the RNA-guided DNase Cas9, the clustered regularly interspaced short palindromic repeats (CRISPR) system, has been developed. Here we report the efficient generation of an iPS cell model for immunodeficiency, centromeric region instability, facial anomalies syndrome (ICF) syndrome using the CRISPR system. We obtained iPS cells with mutations in both alleles of DNA methyltransferase 3B (DNMT3B) in 63% of transfected clones. Our data suggest that the CRISPR system is highly efficient and useful for genome engineering of human iPS cells.
巻・号 14(10)
ページ 19774-81
公開日 2013-9-30
DOI 10.3390/ijms141019774
PII ijms141019774
PMID 24084724
PMC PMC3821585
MeSH Alleles Base Sequence Cell Line Clustered Regularly Interspaced Short Palindromic Repeats / genetics* DNA (Cytosine-5-)-Methyltransferases / genetics Gene Targeting / methods Genetic Engineering / methods Genome / genetics* Humans Hypertelorism / genetics* Induced Pluripotent Stem Cells / metabolism* Intellectual Disability / genetics* Kyphosis / genetics* Megalencephaly / genetics* Molecular Sequence Data RNA Editing / genetics* Tongue / abnormalities*
IF 4.556
引用数 63
WOS 分野 BIOCHEMISTRY & MOLECULAR BIOLOGY CHEMISTRY, MULTIDISCIPLINARY
リソース情報
ヒト・動物細胞