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Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase

文献类型：期刊论文

作者Yuan, Juanjuan3; Ma, Yunqing3; Huang, Tao3; Peng, Yuanzheng3; Li, Jia3; Zhang, Yuchen3; Chang, Xing3; Chen, Yanhao4; Ding, Qiurong4; Song, Bing1
刊名MOLECULAR CELL
出版日期2018
卷号72期号:2页码:380-+
ISSN号1097-2765
关键词Phylogenetic tree Triplet cover Tree-distances Hall's theorem Ample patchwork Shellability
DOI10.1016/j.molcel.2018.09.002
文献子类Article
英文摘要RNA splicing is a critical mechanism by which to modify transcriptome, and its dysregulation is the underlying cause of many human diseases. It remains challenging, however, to genetically modulate a splicing event in its native context. Here, we demonstrate that a CRISPR-guided cytidine deaminase (i.e., targeted-AID mediated mutagenesis [TAM]) can efficiently modulate various forms of mRNA splicing. By converting invariant guanines to adenines at either 5' or 3' splice sites (SS), TAM induces exon skipping, activation of alternative SS, switching between mutually exclusive exons, or targeted intron retention. Conversely, TAM promotes downstream exon inclusion by mutating cytidines into thymines at the polypyrimidine tract. Applying this approach, we genetically restored the open reading frame and dystrophin function of a mutant DMD gene in patient-derived induced pluripotent stem cells (iPSCs). Thus, the CRISPR-guided cytidine deaminase provides a versatile genetic platform to modulate RNA splicing and to correct mutations associated with aberrant splicing in human diseases.
学科主题Biochemistry & Molecular Biology ; Cell Biology
WOS关键词PLURIPOTENT STEM-CELLS ; MUSCULAR-DYSTROPHY ; INTRON RETENTION ; MAMMALIAN-CELLS ; PYRUVATE-KINASE ; GENOME ; DIFFERENTIATION ; EXPRESSION ; REGULATOR ; NUCLEASES
语种英语
出版者CELL PRESS
WOS记录号WOS:000447630200018
版本出版稿
源URL[http://202.127.25.144/handle/331004/986]  
专题中国科学院上海生命科学研究院营养科学研究所
作者单位1.Guangzhou Med Univ, Key Lab Major Obstet Dis Guangdong Prov, Key Lab Reprod & Genet Guangdong Higher Educ Inst, Affiliated Hosp 3, Guangzhou 510510, Guangdong, Peoples R China;
2.Univ Calif San Diego, Dept Cellular & Mol Med, La Jolla, CA 92037 USA,
3.Univ Chinese Acad Sci, Chinese Acad Sci, CAS Key Lab Tissue Microenvironm & Tumor, Shanghai Inst Nutr & Hlth,Shanghai Inst Biol Sci, Shanghai 200031, Peoples R China;
4.Univ Chinese Acad Sci, Shanghai Inst Nutr & Hlth, Chinese Acad Sci, Shanghai Inst Biol Sci,CAS Key Lab Nutr Metab & F, Shanghai 200031, Peoples R China;
推荐引用方式
GB/T 7714		 Yuan, Juanjuan,Ma, Yunqing,Huang, Tao,et al. Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase[J]. MOLECULAR CELL,2018,72(2):380-+.
APA Yuan, Juanjuan.,Ma, Yunqing.,Huang, Tao.,Peng, Yuanzheng.,Li, Jia.,...&,.(2018).Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase.MOLECULAR CELL,72(2),380-+.
MLA Yuan, Juanjuan,et al."Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase".MOLECULAR CELL 72.2(2018):380-+.

入库方式： OAI收割

来源：上海营养与健康研究所

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