Citation: Lauren N. Randolph, Yuqian Jiang, Xiaojun Lian. Stem Cell Engineering and Differentiation for Disease Modeling and Cell-based Therapies[J]. AIMS Cell and Tissue Engineering, 2017, 1(2): 140-157. doi: 10.3934/celltissue.2017.2.140
[1] | Takahashi K, Tanabe K, Ohnuki M, et al. (2007) Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131: 861-872. doi: 10.1016/j.cell.2007.11.019 |
[2] | Yu J, Vodyanik MA, Smugaotto K, et al. (2007) Induced Pluripotent Stem Cell Lines Derived from Human Somatic Cells. Stem Cell Rev 8: 693-702. |
[3] | Thomson JA, Itskovitz-Eldor J, Shapiro SS, et al. (1998) Embryonic stem cell lines derived from human blastocysts. Science 282: 1145-1147. |
[4] | Takahashi K, Yamanaka S (2006) Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors. Cell 126: 663-676. doi: 10.1016/j.cell.2006.07.024 |
[5] | Okita K, Ichisaka T, Yamanaka S (2007) Generation of germline-competent induced pluripotent stem cells. Nature 448: 313-317. doi: 10.1038/nature05934 |
[6] | Wernig M, Meissner A, Foreman R, et al. (2007) In vitro reprogramming of fibroblasts into a pluripotent ES-cell-like state. Nature 448: 318-324. doi: 10.1038/nature05944 |
[7] | Maherali N, Sridharan R, Xie W, et al. (2017) Directly Reprogrammed Fibroblasts Show Global Epigenetic Remodeling and Widespread Tissue Contribution. Cell Stem Cell 1: 55-70. |
[8] | Sumi T, Tsuneyoshi N, Nakatsuji N, et al. (2007) Apoptosis and differentiation of human embryonic stem cells induced by sustained activation of c-Myc. Oncogene 26: 5564-5576. doi: 10.1038/sj.onc.1210353 |
[9] | Baharvand H, Hassani S (2013) Pluripotent Stem Cells. 997: 13-23. |
[10] | Faravelli I, Bucchia M, Rinchetti, et al. (2014) Motor neuron derivation from human embryonic and induced pluripotent stem cells: experimental approaches and clinical perspectives. Stem Cell Res Ther 5: 87. doi: 10.1186/scrt476 |
[11] | Du ZW, Chen H, Liu H, et al. (2015) Generation and expansion of highly pure motor neuron progenitors from human pluripotent stem cells. Nat Commun 6: 6626. doi: 10.1038/ncomms7626 |
[12] | Wichterle H, Lieberam I, Porter JA, et al. (2002) Directed differentiation of embryonic stem cells into motor neurons. Cell 110: 385-397. doi: 10.1016/S0092-8674(02)00835-8 |
[13] | Takazawa T, Croft GF, Amoroso MW, et al. (2012) Maturation of spinal motor neurons derived from human embryonic stem cells. PLoS One 7: 1-9. |
[14] | Broccoli V, Rubio A, Taverna S, et al. (2015) Overcoming the hurdles for a reproducible generation of human functionally mature reprogrammed neurons. Exp Biol Med 240: 787-794. doi: 10.1177/1535370215577585 |
[15] | Miller JD, Ganat YM, Kishinevsky S, et al. (2013) Human iPSC-Based Modeling of Late-Onset Disease via Progerin-Induced Aging. Cell Stem Cell 13: 691-705. doi: 10.1016/j.stem.2013.11.006 |
[16] | Clevers H (2016) Modeling Development and Disease with Organoids. Cell 165: 1586-1597. doi: 10.1016/j.cell.2016.05.082 |
[17] | Doetschman TC, Eistetter H, Katz M, et al. (1985) The in vitro development of blastocyst-derived embryonic stem cell lines: formation of visceral yolk sac, blood islands and myocardium. J Embryol Exp Morphol 87: 27-45. |
[18] | Bu L, Jiang X, Martinpuig S, et al. (2009) Human ISL1 heart progenitors generate diverse multipotent cardiovascular cell lineages. Nature 460: 113-117. doi: 10.1038/nature08191 |
[19] | Elliott DA, Braam SR, Koutsis K, et al. (2011) NKX2-5(eGFP/w) hESCs for isolation of human cardiac progenitors and cardiomyocytes. Nat Methods 8: 1037-1040. doi: 10.1038/nmeth.1740 |
[20] | Bao ZZ, Bruneau BG, Seidman JG, et al. (1999) Regulation of chamber-specific gene expression in the developing heart by Irx4. Science 283: 1161-1164. doi: 10.1126/science.283.5405.1161 |
[21] | O'Brien TX, Lee KJ, Chien KR (1993) Positional specification of ventricular myosin light chain 2 expression in the primitive murine heart tube. Proc Natl Acad Sci USA 90: 5157-5161. doi: 10.1073/pnas.90.11.5157 |
[22] | Protze SI, Liu J, Nussinovitch U, et al. (2017) Sinoatrial node cardiomyocytes derived from human pluripotent cells function as a biological pacemaker. Nat Biotechnol 35: 56-68. |
[23] | Xu C, Police S, Rao N, et al. (2002) Characterization and enrichment of cardiomyocytes derived from human embryonic stem cells. Circ Res 91: 501-508. doi: 10.1161/01.RES.0000035254.80718.91 |
[24] | Laflamme MA, Gold J, Xu C, et al. (2005) Formation of human myocardium in the rat heart from human embryonic stem cells. Am J Pathol 167: 663-671. doi: 10.1016/S0002-9440(10)62041-X |
[25] | Laflamme MA, Chen KY, Naumova AV, et al. (2007) Cardiomyocytes derived from human embryonic stem cells in pro-survival factors enhance function of infarcted rat hearts. Nat Biotechnol 25: 1015-1024. doi: 10.1038/nbt1327 |
[26] | Paige SL, Osugi T, Afanasiev OK, et al. (2010) Endogenous Wnt/Beta-Catenin signaling is required for cardiac differentiation in human embryonic stem cells. PLoS One 5: e11134. doi: 10.1371/journal.pone.0011134 |
[27] | Lian X, Palecek SP (2012) Cozzarelli Prize Winner: Robust cardiomyocyte differentiation from human pluripotent stem cells via temporal modulation of canonical Wnt signaling. Proc Natl Acad Sci 109: E1848-E1857. doi: 10.1073/pnas.1200250109 |
[28] | Lian X, Zhang J, Azarin SM, et al. (2013) Directed cardiomyocyte differentiation from human pluripotent stem cells by modulating Wnt/β-catenin signaling under fully defined conditions. Nat Protoc 8: 162-175. |
[29] | Lian X, Bao X, Zilberter M, et al. (2015) Chemically defined, albumin-free human cardiomyocyte generation. Nat Methods 12: 595-596. doi: 10.1038/nmeth.3448 |
[30] | Pei F, Jiang J, Bai S, et al. (2017) Chemical-de fi ned and albumin-free generation of human atrial and ventricular myocytes from human pluripotent stem cells. Stem Cell Res 19: 94-103. doi: 10.1016/j.scr.2017.01.006 |
[31] | Kehat I, Khimovich L, Caspi O, et al. (2004) Electromechanical integration of cardiomyocytes derived from human embryonic stem cells. Nat Biotechnol 22: 1282-1289. doi: 10.1038/nbt1014 |
[32] | Zhang Q, Jiang J, Han P, et al. (2011) Direct differentiation of atrial and ventricular myocytes from human embryonic stem cells by alternating retinoid signals. Cell Res 21: 579-587. doi: 10.1038/cr.2010.163 |
[33] | Cao N, Huang Y, Zheng J, et al. (2016) Conversion of human fibroblasts into functional cardiomyocytes by small molecules. Science 352: 1216-1220. doi: 10.1126/science.aaf1502 |
[34] | Maizels L, Huber I, Arbel G, et al. (2017) Patient-Specific Drug Screening Using a Human Induced Pluripotent Stem Cell Model of Catecholaminergic Polymorphic Ventricular Tachycardia Type 2. Circ Arrhythmia Electrophysiol 10: 1-15. |
[35] | Ackermann M, Liebhaber S, Klusmann JH, et al. (2015) Lost in translation: pluripotent stem cell-derived hematopoiesis. EMBO Mol Med 7: 1388-1402. doi: 10.15252/emmm.201505301 |
[36] | Chadwick K, Wang L, Li L, et al. (2003) Cytokines and BMP-4 promote hematopoietic differentiation of human embryonic stem cells. Blood 102: 906-915. doi: 10.1182/blood-2003-03-0832 |
[37] | Woods NB, Parker AS, Moraghebi R, et al. (2011) Brief report: efficient generation of hematopoietic precursors and progenitors from human pluripotent stem cell lines. Stem Cells 29: 1158-1164. doi: 10.1002/stem.657 |
[38] | Ng ES, Azzola L, Bruveris FF, et al. (2016) Differentiation of human embryonic stem cells to HOXA+ hemogenic vasculature that resembles the aorta-gonad-mesonephros. Nat Biotechnol 34: 1168-1179. doi: 10.1038/nbt.3702 |
[39] | Galat Y, Dambaeva S, Elcheva I, et al. (2017) Cytokine-free directed differentiation of human pluripotent stem cells efficiently produces hemogenic endothelium with lymphoid potential. Stem Cell Res Ther 8: 67. doi: 10.1186/s13287-017-0519-0 |
[40] | Lian X, Bao X, Ai-Ahmad A, et al. (2014) Efficient Differentiation of Human Pluripotent Stem Cells to Endothelial Progenitors via Small-Molecule Activation of WNT Signalling. Stem Cell Rep 3: 804-816. doi: 10.1016/j.stemcr.2014.09.005 |
[41] | Sugimura R, Jha DK, Han A, et al. (2017) Haematopoietic stem and progenitor cells from human pluripotent stem cells. Nature 545: 432-438. doi: 10.1038/nature22370 |
[42] | Lumelsky N, Blondel O, Laeng P, et al. (2001) Differentiation of Embryonic Stem Cells to Insulin-Secreting Structures Similar to Pancreatic Islets. Science 292: 1389-1394. doi: 10.1126/science.1058866 |
[43] | Shi Y, Hou L, Tang F, et al. (2005) Inducing Embryonic Stem Cells to Differentiate into Pancreatic β Cells by a Novel Three-Step Approach with Activin A and All- Trans Retinoic Acid. Stem Cells 23: 656-662. doi: 10.1634/stemcells.2004-0241 |
[44] | D'Amour KA, Agulnick AD, Eliazer S, et al. (2005) Efficient differentiation of human embryonic stem cells to definitive endoderm. Nat Biotechnol 23: 1534-1541. doi: 10.1038/nbt1163 |
[45] | D'Amour KA, Bang AG, Eliazer S, et al. (2006) Production of pancreatic hormone–expressing endocrine cells from human embryonic stem cells. Nat Biotechnol 24: 1392-1401. doi: 10.1038/nbt1259 |
[46] | Jiang J, Au M, Lu K, et al. (2007) Generation of Insulin-Producing Islet-Like Clusters from Human Embryonic Stem Cells. Stem Cells 25: 1940-1953. doi: 10.1634/stemcells.2006-0761 |
[47] | Nostro MC, Sarangi F, Ogawa S, et al. (2011) Stage-specific signaling through TGFβ family members and WNT regulates patterning and pancreatic specification of human pluripotent stem cells. Development 138: 861-871. doi: 10.1242/dev.055236 |
[48] | Pagliuca FW, Millman JR, Gurtler M, et al. (2014) Generation of functional human pancreatic β cells in vitro. Cell 159: 428-439. doi: 10.1016/j.cell.2014.09.040 |
[49] | Millman JR, Xie C, Van DA, et al. (2016) Generation of stem cell-derived β-cells from patients with type 1 diabetes. Nat Commun 7: 11463. doi: 10.1038/ncomms11463 |
[50] | Kim YG, Cha J, Chandrasegaran S (1996) Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci USA 93: 1156-1160. doi: 10.1073/pnas.93.3.1156 |
[51] | Porteus MH, Carroll D (2005) Gene targeting using zinc finger nucleases. Nat Biotechnol 23: 967-973. doi: 10.1038/nbt1125 |
[52] | Sanjana NE, Cong L, Zhou Y, et al. (2012) A transcription activator-like effector toolbox for genome engineering. Nat Protoc 7: 171-192. doi: 10.1038/nprot.2011.431 |
[53] | Ishino Y, Shinagawa H, Makino K, et al. (1987) Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product. J Bacteriol 169: 5429-5433. doi: 10.1128/jb.169.12.5429-5433.1987 |
[54] | Jansen R, Embden JDA, Gaastra W, at al. (2002) Identification of genes that are associated with DNA repeats in prokaryotes. Mol Microbiol 43: 1565-1575. doi: 10.1046/j.1365-2958.2002.02839.x |
[55] | Mojica FJM, Díez-Villaseñor C, García-Martínez J, et al. (2005) Intervening Sequences of Regularly Spaced Prokaryotic Repeats Derive from Foreign Genetic Elements. J Mol Evol 60: 174-182. doi: 10.1007/s00239-004-0046-3 |
[56] | Hsu PD, Lander ES, Zhang, F (2014) Development and Applications of CRISPR-Cas9 for Genome Engineering. Cell 157: 1262-1278. doi: 10.1016/j.cell.2014.05.010 |
[57] | Cong L, Ran FA, Cox D, et al. (2013) Multiplex Genome Engineering Using CRISPR/Cas Systems. Science 339: 819-823. doi: 10.1126/science.1231143 |
[58] | Zhang JP, Li XL, Li GH, et al. (2017) Efficient precise knockin with a double cut HDR donor after CRISPR / Cas9-mediated double-stranded DNA cleavage. Genome Biol 18: 35. doi: 10.1186/s13059-017-1164-8 |
[59] | Zhang XH, Tee LY, Wang XG, et al. (2015) Off-target Effects in CRISPR/Cas9-mediated Genome Engineering. Mol Ther Nucleic Acids 4: e264. doi: 10.1038/mtna.2015.37 |
[60] | Tsai SQ, Wyvekens N, Khayter C, et al. (2014) Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing. Nat Biotechnol 32: 569-576. doi: 10.1038/nbt.2908 |
[61] | Davis RP, Ng ES, Costa M, et al. (2008) Targeting a GFP reporter gene to the MIXL1 locus of human embryonic stem cells identifies human primitive streak – like cells and enables isolation of primitive hematopoietic precursors. Blood 111: 1876-1884. doi: 10.1182/blood-2007-06-093609 |
[62] | Lian X, Xu J, Bao X, et al. (2016) Interrogating Canonical Wnt Signaling Pathway in Human Pluripotent Stem Cell Fate Decisions Using CRISPR-Cas9. Cell Mol Bioeng 9: 325-334. doi: 10.1007/s12195-016-0453-8 |
[63] | Wang H, Luo X, Yao L, et al. (2015) Improvement of Cell Survival During Human Pluripotent Stem Cell Definitive Endoderm Differentiation. Stem Cells Dev 24: 2536-2546. doi: 10.1089/scd.2015.0018 |
[64] | Clarke RL, Yzaguirre AD, Yashiro-Ohtani Y, et al. (2013) The expression of Sox17 identifies and regulates haemogenic endothelium. Nat Cell Biol 15: 502-510. doi: 10.1038/ncb2724 |
[65] | Wang P, Rodriguez RT, Wang J, et al. (2011) Targeting SOX17 in human embryonic stem cells creates unique strategies for isolating and analyzing developing endoderm. Cell Stem Cell 8: 335-346. doi: 10.1016/j.stem.2011.01.017 |
[66] | Hinson JT, Chopra A, Nafissi N, et al. (2015) Titin mutations in iPS cells define sarcomere insufficiency as a cause of dilated cardiomyopathy. Science 349: 982-987. doi: 10.1126/science.aaa5458 |
[67] | Nelson CE, Hakim CH, Ousterout DG, et al. (2016) In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystropy. Science 351: 403-408. doi: 10.1126/science.aad5143 |
[68] | Chen Y, Yu J, Niu Y, et al. (2017) Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys. Cell 169: 945-955. doi: 10.1016/j.cell.2017.04.035 |