CRISPR/Cas9基因编辑技术在糖尿病细胞治疗中的应用研究进展
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摘要
近几年来,基因编辑技术快速发展,为在特定位置精确操控基因组提供了一个有效而精确的工具。CRISPR/Cas9系统是一种新型的基因编辑工具,可实现对人类基因组的高精度修饰,是疾病建模和治疗的可行选择。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。该文从干细胞分化为β细胞、基因编辑重编程为β细胞以及修饰基因三个方面总结了CRISPR/Cas9技术在糖尿病治疗中的应用。
In recent years, the rapid development of gene editing technology has provided an effective and accurate tool for the manipulation of genomes at specific locations. CRISPR/Cas9 system is a new gene editing tool, which can achieve high-precision modification on human genome and is a feasible strategy for disease modeling and treatment. CRISPR/Cas9 system has been widely applied in many research fields, including diabetes cell therapy. In this review, we summarized recent advances of CRISPR/Cas9 in the treatment of diabetes mellitus, especially in the differentiation of stem cells into β cells, direct reprogramming into beta cells, and genes modification.
In recent years, the rapid development of gene editing technology has provided an effective and accurate tool for the manipulation of genomes at specific locations. CRISPR/Cas9 system is a new gene editing tool, which can achieve high-precision modification on human genome and is a feasible strategy for disease modeling and treatment. CRISPR/Cas9 system has been widely applied in many research fields, including diabetes cell therapy. In this review, we summarized recent advances of CRISPR/Cas9 in the treatment of diabetes mellitus, especially in the differentiation of stem cells into β cells, direct reprogramming into beta cells, and genes modification.
引文
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[27]Qi LS,Larson MH,Gilbert LA,et al.Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression.Cell,2013,152:1173-83
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[29]Perez-Pinera P,Kocak DD,Vockley CM,et al.RNA-guided gene activation by CRISPR-Cas9-based transcription factors.Nat Methods,2013,10:973-6
[30]Chavez A,Scheiman J,Vora S,et al.Highly efficient Cas9-mediated transcriptional programming.Nat Methods,2015,12:326-8
[31]胡依萌,徐焱成.CRISPR/Cas9技术在代谢性疾病研究中的应用进展.山东医药,2017,57:97-100
[32]Long C,Li H,Tiburcy M,et al.Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing.Sci Adv,2018,4:9004
[33]Rogers ZN,McFarland CD,Winters IP,et al.Mapping the in vivo fitness landscape of lung adenocarcinoma tumor suppression in mice.Nat Genet,2018,50:483-6
[34]Kaminski R,Chen Y,Fischer T,et al.Elimination of HIV-1 genomes from human T-lymphoid cells by CRISPR/Cas9 gene editing.Sci Rep,2016,6:28213
[35]Yusa K,Rashid ST,Helene SM,et al.Targeted gene correction ofα1-antitrypsin deficiency in induced pluripotent stem cells.Nature,2011,478:391-4
[36]Kim MY,Yu KR,Kenderian SS,et al.Genetic inactivation of CD33 in hematopoietic stem cells to enable CAR T cell immunotherapy for acute myeloid leukemia.Cell,2018,173:1439-53
[37]Yilmaz A,Peretz M,Aharony A,et al.Defining essential genes for human pluripotent stem cells by CRISPR-Cas9screening in haploid cells.Nat Cell Biol,2018,20:610-9
[38]Liu P,Chen M,Liu Y,et al.CRISPR-based chromatin remodeling of the endogenous Oct4 or Sox2 locus enables reprogramming to pluripotency.Cell Stem Cell,2018,22:252-61
[39]Akcakaya P,Bobbin ML,Guo JA,et al.In vivo CRISPRediting with no detectable genome-wide off-target mutations.Nature,2018,561:416-9
[40]Nishimasu H,Shi X,Ishiguro S,et al.Engineered CRISPR-Cas9 nuclease with expanded targeting space.Science,2018,361:1259-62
[2]Hockemeyer D,Wang H,Kiani S,et al.Genetic engineering of human pluripotent cells using TALEnucleases.Nat Biotechnol,2011,29:731-4
[3]Jackson RN,Golden SM,van Erp PB,et al.Structural biology.Crystal structure of the CRISPR RNA-guided surveillance complex from Escherichia coli.Science,2014,345:1473-9
[4]Yan S,Tu Z,Liu Z,et al.A huntingtin knockin pig model recapitulates features of selective neurodegeneration in Huntington's disease.Cell,2018,173:989-1002
[5]Li Y,Zhang W,Zhao J,et al.Establishing a dual knockout cell line by lentivirus based combined CRISPR/Cas9and Loxp/Cre system.Cytotechnology,2018,70:1595-605
[6]Xu C,Lu Z,Luo Y,et al.Targeting of NLRP3 inflammasome with gene editing for the amelioration of inflammatory diseases.Nat Commun,2018,9:4092
[7]Xiao Y,Li C,Gu M,et al.Protein disulfide isomerase silence inhibits inflammatory functions of macrophages by suppressing reactive oxygen species and NF-κB pathway.Inflammation,2018,41:614-25
[8]Liao HK,Hatanaka F,Araoka T,et al.In vivo target gene activation via CRISPR/Cas9-mediated trans-epigenetic modulation.Cell,2017,171:1495-507
[9]Wang H,Xu X,Nguyen CM,et al.CRISPR-mediated programmable 3D genome positioning and nuclear organization.Cell,2018,175:1405-17
[10]Adli M.The CRISPR tool kit for genome editing and beyond.Nat Commun,2018,9:1911
[11]Kondo Y,Toyoda T,Inagaki N,et al.iPSC technologybased regenerative therapy for diabetes.J Diabetes Investig,2018,9:234-43
[12]Lilly MA,Davis MF,Fabie JE,et al.Current stem cell based therapies in diabetes.Am J Stem Cell,2016,5:87-98
[13]McGrath PS,Watson CL,Ingram C,et al.The basic helixloop-helix transcription factor NEUROG3 is required for development of the human endocrine pancreas.Diabetes,2015,64:2497-505
[14]Zhu Z,Li QV,Lee K,et al.Genome editing of lineage determinants in human pluripotent stem cells reveals mechanisms of pancreatic development and diabetes.Cell Stem Cell,2016,18:755-68
[15]González 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,2014,15:215-26
[16]Shi ZD,Lee K,Yang D,et al.Genome editing in hPSCs reveals GATA6 haploinsufficiency and a genetic interaction with GATA4 in human pancreatic development.Cell Stem Cell,2017,20:675-88
[17]Zeng H,Guo M,Zhou T,et al.An isogenic human ESCplatform for functional evaluation of genome-wideassociation-study-identified diabetes genes and drug discovery.Cell Stem Cell,2016,19:326-40
[18]Saarim?ki-Vire J,Balboa D,Russell MA,et al.An activating STAT3 mutation causes neonatal diabetes through premature induction of pancreatic differentiation.Cell Rep,2017,19:281-94
[19]Blyszczuk P,Czyz J,Kania G,et al.Expression of Pax4 in embryonic stem cells promotes differentiation of nestinpositive progenitor and insulin-producing cells.Proc Natl Acad Sci USA,2003,100:998-1003
[20]张迪,翁孝刚,王涛,等.不同途径移植人脐带间充质干细胞治疗糖尿病大鼠.新乡医学院学报,2013,30:347-52
[21]Gerace D,Martiniello-Wilks R,Nassif NT,et al.CRISPR-targeted genome editing of mesenchymal stem cellderived therapies for type 1 diabetes:a path to clinical success?Stem Cell Res Therapy,2017,8:62
[22]Yue J,Gou X,Li Y,et al.Engineered epidermal progenitor cells can correct diet-induced obesity and diabetes.Cell Stem Cell,2017,21:256-63
[23]Akinci E,Banga A,Tungatt K,et al.Reprogramming of various cell types to aβ-like state by Pdx1,Ngn3 and MafA.PLoS One,2013,8:82424
[24]Banga A,Akinci E,Greder LV,et al.In vivo reprogramming of Sox9+cells in the liver to insulin-secreting ducts.Proc Natl Acad Sci USA,2012:109,15336-41
[25]Hickey RD,Galivo F,Schug J,et al.Generation of isletlike cells from mouse gall bladder by direct ex vivo reprogramming.Stem Cell Res,2013,11:503-15
[26]Hanna J,Saha K,Pando B,et al.Direct cell reprogramming is a stochastic process amenable to acceleration.Nature,2009,462:595-601
[27]Qi LS,Larson MH,Gilbert LA,et al.Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression.Cell,2013,152:1173-83
[28]Maeder ML,Linder SJ,Cascio VM.et al.CRISPR RNA-guided activation of endogenous human genes.Nat Methods,2013,10:977-9
[29]Perez-Pinera P,Kocak DD,Vockley CM,et al.RNA-guided gene activation by CRISPR-Cas9-based transcription factors.Nat Methods,2013,10:973-6
[30]Chavez A,Scheiman J,Vora S,et al.Highly efficient Cas9-mediated transcriptional programming.Nat Methods,2015,12:326-8
[31]胡依萌,徐焱成.CRISPR/Cas9技术在代谢性疾病研究中的应用进展.山东医药,2017,57:97-100
[32]Long C,Li H,Tiburcy M,et al.Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing.Sci Adv,2018,4:9004
[33]Rogers ZN,McFarland CD,Winters IP,et al.Mapping the in vivo fitness landscape of lung adenocarcinoma tumor suppression in mice.Nat Genet,2018,50:483-6
[34]Kaminski R,Chen Y,Fischer T,et al.Elimination of HIV-1 genomes from human T-lymphoid cells by CRISPR/Cas9 gene editing.Sci Rep,2016,6:28213
[35]Yusa K,Rashid ST,Helene SM,et al.Targeted gene correction ofα1-antitrypsin deficiency in induced pluripotent stem cells.Nature,2011,478:391-4
[36]Kim MY,Yu KR,Kenderian SS,et al.Genetic inactivation of CD33 in hematopoietic stem cells to enable CAR T cell immunotherapy for acute myeloid leukemia.Cell,2018,173:1439-53
[37]Yilmaz A,Peretz M,Aharony A,et al.Defining essential genes for human pluripotent stem cells by CRISPR-Cas9screening in haploid cells.Nat Cell Biol,2018,20:610-9
[38]Liu P,Chen M,Liu Y,et al.CRISPR-based chromatin remodeling of the endogenous Oct4 or Sox2 locus enables reprogramming to pluripotency.Cell Stem Cell,2018,22:252-61
[39]Akcakaya P,Bobbin ML,Guo JA,et al.In vivo CRISPRediting with no detectable genome-wide off-target mutations.Nature,2018,561:416-9
[40]Nishimasu H,Shi X,Ishiguro S,et al.Engineered CRISPR-Cas9 nuclease with expanded targeting space.Science,2018,361:1259-62