规律簇集间断的短回文重复序列及其相关核酸酶9系统应用于糖尿病领域的研究进展
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摘要
规律簇集间断的短回文重复序列及其相关核酸酶9(CRISPR/Cas9)系统是一种新型的基因编辑工具,可同时研究多个基因的功能及相互关系,已在多种疾病中显示出巨大的潜在价值。与其他基因编辑技术,如锌指核酸酶(ZFNs)和转录激活样核酸酶(TALENs)相比,CRISPR/Cas9更简单、高效。随着CRISPR/Cas9系统技术的广泛应用,在糖尿病领域有不少相关研究出现。本文从基础到临床对CRISPR/Cas9在糖尿病领域的研究进展作文献回顾,期待为相关人员提供参考。
Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPRassociated nuclease 9(Cas9)(CRISPR/Cas9),a revolutionary gene editing technology,has been applied in the researches of many diseases such as anemia,HIV,cardiovascular diseases and so on.Compared to other gene editing techniques such as zinc-finger nucleases(ZFNs)and transcription activator-like effector nucleases(TALENs),CRISPR/Cas9 is much easier and effective to use.In the study field of diabetes,several researchers have tried to set up new diabetic animal models,modify diabetic associated gens or explore new diabetic therapy using CRISPR/Cas9.We review literatures related to the use of CRISPR/Cas9 in diabetes studies.
Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPRassociated nuclease 9(Cas9)(CRISPR/Cas9),a revolutionary gene editing technology,has been applied in the researches of many diseases such as anemia,HIV,cardiovascular diseases and so on.Compared to other gene editing techniques such as zinc-finger nucleases(ZFNs)and transcription activator-like effector nucleases(TALENs),CRISPR/Cas9 is much easier and effective to use.In the study field of diabetes,several researchers have tried to set up new diabetic animal models,modify diabetic associated gens or explore new diabetic therapy using CRISPR/Cas9.We review literatures related to the use of CRISPR/Cas9 in diabetes studies.
引文
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[18]Schmid-Burgk JL,Chauhan D,Schmidt T,et al.A Genomewide CRISPR(Clustered Regularly Interspaced Short Palindromic Repeats)screen identifies NEK7as an essential component of NLRP3inflammasome activation.J Biol Chem,2016,291:103-109.
[19]Oslowski CM,Hara T,O'sullivan-Murphy B,et al.Thioredoxin-interacting protein mediates ER stress-induced beta cell death through initiation of the inflammasome.Cell Metab,2012,16:265-273.
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[24]Kok FO,Lawson ND.A platform for reverse genetics in endothelial cells.Circ Res,2015,117:107-108.
[2]Jinek M,Chylinski K,Fonfara I,et al.A programmable dualRNA-guided DNA endonuclease in adaptive bacterial immunity.Science,2012,337:816-821.
[3]Mali P,Esvelt KM,Church GM.Cas9as a versatile tool for engineering biology.Nat Methods,2013,10:957-963.
[4]Gonzalez F,Zhu Z,Shi ZD,et al.An iCRISPR platform for rapid,multiplexable,and inducible genome editing in human pluripotent stem cells.Cell Stem Cell,2014,15:215-226.
[5]Konermann S,Brigham MD,Trevino AE,et al.Genome-scale transcriptional activation by an engineered CRISPR-Cas9complex.Nature,2015,517:583-588.
[6]Chavez A,Scheiman J,Vora S,et al.Highly efficient Cas9-mediated transcriptional programming.Nat Methods,2015,12:326-328.
[7]Zalatan JG,Lee ME,Almeida R,et al.Engineering complex synthetic transcriptional programs with CRISPR RNA scaffolds.Cell,2015,160:339-350.
[8]Fitzgerald K,Frank-Kamenetsky M,Shulga-Morskaya S,et al.Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9(PCSK9)and the concentration of serum LDL cholesterol in healthy volunteers:a randomised,single-blind,placebo-controlled,phase 1trial.Lancet,2014,383:60-68.
[9]Fellmann C,Lowe SW.Stable RNA interference rules for silencing.Nat Cell Biol,2014,16:10-18.
[10]Ma Y,Shen B,Zhang X,et al.Heritable multiplex genetic engineering in rats using CRISPR/Cas9.PLoS One,2014,9:e89413.
[11]Li D,Qiu Z,Shao Y,et al.Heritable gene targeting in the mouse and rat using a CRISPR-Cas system.Nat Biotechnol,2013,31:681-683.
[12]Hai T,Teng F,Guo R,et al.One-step generation of knockout pigs by zygote injection of CRISPR/Cas system.Cell Res,2014,24:372-375.
[13]Kang Y,Zheng B,Shen B,et al.CRISPR/Cas9-mediated Dax1knockout in the monkey recapitulates human AHC-HH.Hum Mol Genet,2015,24:7255-7264.
[14]马元武,马婧,路迎冬,et al.利用CRISPR/Cas9敲除大鼠胰岛素受体底物1(Irs1)基因.中国比较医学杂志,2014,3:55-60.
[15]Bao D,Ma Y,Zhang X,et al.Preliminary characterization of a leptin receptor knockout rat created by CRISPR/Cas9system.Sci Rep,2015,5:15942.
[16]Wang X,Tang Y,Lu J,et al.Characterization of novel cytochrome P450 2E1knockout rat model generated by CRISPR/Cas9.Biochem Pharmacol,2016,105:80-90.
[17]Claussnitzer M,Dankel SN,Kim KH,et al.FTO obesity variant circuitry and adipocyte browning in humans.N Engl J Med,2015,373:895-907.
[18]Schmid-Burgk JL,Chauhan D,Schmidt T,et al.A Genomewide CRISPR(Clustered Regularly Interspaced Short Palindromic Repeats)screen identifies NEK7as an essential component of NLRP3inflammasome activation.J Biol Chem,2016,291:103-109.
[19]Oslowski CM,Hara T,O'sullivan-Murphy B,et al.Thioredoxin-interacting protein mediates ER stress-induced beta cell death through initiation of the inflammasome.Cell Metab,2012,16:265-273.
[20]Bompada P,Atac D,Luan C,et al.Histone acetylation of glucose-induced thioredoxin-interacting protein gene expression in pancreatic islets.Int J Biochem Cell Biol,2016,81:82-91.
[21]Wadden TA,Hollander P,Klein S,et al.Weight maintenance and additional weight loss with liraglutide after low-calorie-dietinduced weight loss:the SCALE Maintenance randomized study.Int J Obes,2013,37:1443-1451.
[22]Naylor J,Suckow AT,Seth A,et al.Use of CRISPR/Cas9-engineered INS-1pancreatic beta cells to define the pharmacology of dual GIPR/GLP-1R agonists.Biochem J,2016,473:2881-2891.
[23]Cox DB,Platt RJ,Zhang F.Therapeutic genome editing:prospects and challenges.Nat Med,2015,21:121-131.
[24]Kok FO,Lawson ND.A platform for reverse genetics in endothelial cells.Circ Res,2015,117:107-108.