Modeling Cancer with Pluripotent Stem Cells

Trends in Cancer - Tập 2 Số 9 - Trang 485-494 - 2016
Julian A. Gingold1, Ruoji Zhou2,3, Ihor R. Lemischka4,5,6, Dung‐Fang Lee7,2,3
1Women’s Health Institute, Cleveland Clinic Foundation, Cleveland, OH 44195, USA
2Department of Integrative Biology and Pharmacology, McGovern Medical School, The University of Texas Health Science Center at Houston, Houston, TX 77030, USA
3The University of Texas Graduate School of Biomedical Sciences at Houston, Houston, TX 77030, USA
4Department of Developmental and Regenerative Biology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA
5Department of Pharmacology and System Therapeutics, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA
6The Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA
7Center for Stem Cell and Regenerative Medicine, The Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases, The University of Texas Health Science Center at Houston, Houston, TX 77030, USA

Tóm tắt

Từ khóa


Tài liệu tham khảo

Thomson, 1998, Embryonic stem cell lines derived from human blastocysts, Science, 282, 1145, 10.1126/science.282.5391.1145

Takahashi, 2006, Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors, Cell, 126, 663, 10.1016/j.cell.2006.07.024

Takahashi, 2007, Induction of pluripotent stem cells from adult human fibroblasts by defined factors, Cell, 131, 861, 10.1016/j.cell.2007.11.019

Yu, 2007, Induced pluripotent stem cell lines derived from human somatic cells, Science, 318, 1917, 10.1126/science.1151526

González, 2011, Methods for making induced pluripotent stem cells: reprogramming à la carte, Nat. Rev. Genet., 12, 231, 10.1038/nrg2937

Murry, 2008, Differentiation of embryonic stem cells to clinically relevant populations: lessons from embryonic development, Cell, 132, 661, 10.1016/j.cell.2008.02.008

Lancaster, 2014, Organogenesis in a dish: modeling development and disease using organoid technologies, Science, 345, 1247125, 10.1126/science.1247125

Yin, 2016, Engineering stem cell organoids, Cell Stem Cell, 18, 25, 10.1016/j.stem.2015.12.005

Grskovic, 2011, Induced pluripotent stem cells – opportunities for disease modelling and drug discovery, Nat. Rev. Drug Discov., 10, 915, 10.1038/nrd3577

Dimos, 2008, Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons, Science, 321, 1218, 10.1126/science.1158799

Park, 2008, Disease-specific induced pluripotent stem cells, Cell, 134, 877, 10.1016/j.cell.2008.07.041

Yagi, 2011, Modeling familial Alzheimer's disease with induced pluripotent stem cells, Hum. Mol. Genet., 20, 4530, 10.1093/hmg/ddr394

Brennand, 2011, Modelling schizophrenia using human induced pluripotent stem cells, Nature, 473, 221, 10.1038/nature09915

Carvajal-Vergara, 2010, Patient-specific induced pluripotent stem-cell-derived models of LEOPARD syndrome, Nature, 465, 808, 10.1038/nature09005

Pasca, 2011, Using iPSC-derived neurons to uncover cellular phenotypes associated with Timothy syndrome, Nat. Med., 17, 1657, 10.1038/nm.2576

Itzhaki, 2011, Modelling the long QT syndrome with induced pluripotent stem cells, Nature, 471, 225, 10.1038/nature09747

Moretti, 2010, Patient-specific induced pluripotent stem-cell models for long-QT syndrome, N. Engl. J. Med., 363, 1397, 10.1056/NEJMoa0908679

Rashid, 2010, Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells, J. Clin. Invest., 120, 3127, 10.1172/JCI43122

Liu, 2011, Targeted gene correction of laminopathy-associated LMNA mutations in patient-specific iPSCs, Cell Stem Cell, 8, 688, 10.1016/j.stem.2011.04.019

Smith-Arica, 2003, Infection efficiency of human and mouse embryonic stem cells using adenoviral and adeno-associated viral vectors, Cloning Stem Cells, 5, 51, 10.1089/153623003321512166

Ivanova, 2006, Dissecting self-renewal in stem cells with RNA interference, Nature, 442, 533, 10.1038/nature04915

Lee, 2012, Regulation of embryonic and induced pluripotency by aurora kinase-p53 signaling, Cell Stem Cell, 11, 179, 10.1016/j.stem.2012.05.020

Yusa, 2011, Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells, Nature, 478, 391, 10.1038/nature10424

Zou, 2009, Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells, Cell Stem Cell, 5, 97, 10.1016/j.stem.2009.05.023

Hockemeyer, 2009, Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases, Nat. Biotechnol., 27, 851, 10.1038/nbt.1562

Luo, 2014, Generation of GFP reporter human induced pluripotent stem cells using AAVS1 safe harbor transcription activator-like effector nuclease, Curr. Protoc. Stem Cell Biol., 29, 10.1002/9780470151808.sc05a07s29

Ding, 2013, A TALEN genome-editing system for generating human stem cell-based disease models, Cell Stem Cell, 12, 238, 10.1016/j.stem.2012.11.011

Mali, 2013, RNA-guided human genome engineering via Cas9, Science, 339, 823, 10.1126/science.1232033

Cong, 2013, Multiplex genome engineering using CRISPR/Cas systems, Science, 339, 819, 10.1126/science.1231143

Lee, 2015, Modeling familial cancer with induced pluripotent stem cells, Cell, 161, 240, 10.1016/j.cell.2015.02.045

Mulero-Navarro, 2015, Myeloid dysregulation in a human induced pluripotent stem cell model of PTPN11-associated juvenile myelomonocytic leukemia, Cell Rep., 13, 504, 10.1016/j.celrep.2015.09.019

Kotini, 2015, Functional analysis of a chromosomal deletion associated with myelodysplastic syndromes using isogenic human induced pluripotent stem cells, Nat. Biotechnol., 33, 646, 10.1038/nbt.3178

Funato, 2014, Use of human embryonic stem cells to model pediatric gliomas with H3.3K27M histone mutation, Science, 346, 1529, 10.1126/science.1253799

Duan, 2015, PTEN deficiency reprogrammes human neural stem cells towards a glioblastoma stem cell-like phenotype, Nat. Commun., 6, 10068, 10.1038/ncomms10068

Li, 1988, A cancer family syndrome in twenty-four kindreds, Cancer Res., 48, 5358

Dugimont, 1998, The H19 TATA-less promoter is efficiently repressed by wild-type tumor suppressor gene product p53, Oncogene, 16, 2395, 10.1038/sj.onc.1201742

Roberts, 2013, Noonan syndrome, Lancet, 381, 333, 10.1016/S0140-6736(12)61023-X

Lindsley, 2013, Molecular pathophysiology of myelodysplastic syndromes, Annu. Rev. Pathol., 8, 21, 10.1146/annurev-pathol-011811-132436

Wu, 2012, Somatic histone H3 alterations in pediatric diffuse intrinsic pontine gliomas and non-brainstem glioblastomas, Nat. Genet., 44, 251, 10.1038/ng.1102

Schwartzentruber, 2012, Driver mutations in histone H3.3 and chromatin remodelling genes in paediatric glioblastoma, Nature, 482, 226, 10.1038/nature10833

Furnari, 2007, Malignant astrocytic glioma: genetics, biology, and paths to treatment, Genes Dev., 21, 2683, 10.1101/gad.1596707

Si-Tayeb, 2010, Highly efficient generation of human hepatocyte-like cells from induced pluripotent stem cells, Hepatology, 51, 297, 10.1002/hep.23354

Goldman, 2013, KDR identifies a conserved human and murine hepatic progenitor and instructs early liver development, Cell Stem Cell, 12, 748, 10.1016/j.stem.2013.04.026

Hannan, 2013, Production of hepatocyte-like cells from human pluripotent stem cells, Nat. Protoc., 8, 430, 10.1038/nprot.2012.153

Spence, 2011, Directed differentiation of human pluripotent stem cells into intestinal tissue in vitro, Nature, 470, 105, 10.1038/nature09691

Antonica, 2012, Generation of functional thyroid from embryonic stem cells, Nature, 491, 66, 10.1038/nature11525

Dye, 2015, In vitro generation of human pluripotent stem cell derived lung organoids, Elife, 4, e05098, 10.7554/eLife.05098

Huang, 2014, Efficient generation of lung and airway epithelial cells from human pluripotent stem cells, Nat. Biotechnol., 32, 84, 10.1038/nbt.2754

Huang, 2015, The in vitro generation of lung and airway progenitor cells from human pluripotent stem cells, Nat. Protoc., 10, 413, 10.1038/nprot.2015.023

Xia, 2013, Directed differentiation of human pluripotent cells to ureteric bud kidney progenitor-like cells, Nat. Cell. Biol., 15, 1507, 10.1038/ncb2872

Takasato, 2014, Directing human embryonic stem cell differentiation towards a renal lineage generates a self-organizing kidney, Nat. Cell Biol., 16, 118, 10.1038/ncb2894

Osakada, 2009, Stepwise differentiation of pluripotent stem cells into retinal cells, Nat. Protoc., 4, 811, 10.1038/nprot.2009.51

Nakano, 2012, Self-formation of optic cups and storable stratified neural retina from human ESCs, Cell Stem Cell, 10, 771, 10.1016/j.stem.2012.05.009

Mellough, 2012, Efficient stage-specific differentiation of human pluripotent stem cells toward retinal photoreceptor cells, Stem Cells, 30, 673, 10.1002/stem.1037

Lamba, 2006, Efficient generation of retinal progenitor cells from human embryonic stem cells, Proc. Natl. Acad. Sci. U.S.A., 103, 12769, 10.1073/pnas.0601990103

Yang, 2014, Generation of folliculogenic human epithelial stem cells from induced pluripotent stem cells, Nat. Commun., 5, 3071, 10.1038/ncomms4071

Kidwai, 2014, Differentiation of epidermal keratinocytes from human embryonic stem cells, Methods Mol. Biol., 1195, 13, 10.1007/7651_2013_46

Grigoriadis, 2010, Directed differentiation of hematopoietic precursors and functional osteoclasts from human ES and iPS cells, Blood, 115, 2769, 10.1182/blood-2009-07-234690

Kennedy, 2007, Development of the hemangioblast defines the onset of hematopoiesis in human ES cell differentiation cultures, Blood, 109, 2679, 10.1182/blood-2006-09-047704

Chambers, 2009, Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling, Nat. Biotechnol., 27, 275, 10.1038/nbt.1529

Lian, 2007, Derivation of clinically compliant MSCs from CD105+, CD24– differentiated human ESCs, Stem Cells, 25, 425, 10.1634/stemcells.2006-0420

Fang, 2006, Defining the conditions for the generation of melanocytes from human embryonic stem cells, Stem Cells, 24, 1668, 10.1634/stemcells.2005-0414

Lancaster, 2013, Cerebral organoids model human brain development and microcephaly, Nature, 501, 373, 10.1038/nature12517

McCracken, 2014, Modelling human development and disease in pluripotent stem-cell-derived gastric organoids, Nature, 516, 400, 10.1038/nature13863

Matano, 2015, Modeling colorectal cancer using CRISPR-Cas9-mediated engineering of human intestinal organoids, Nat. Med., 21, 256, 10.1038/nm.3802

Takebe, 2013, Vascularized and functional human liver from an iPSC-derived organ bud transplant, Nature, 499, 481, 10.1038/nature12271

Takebe, 2014, Generation of a vascularized and functional human liver from an iPSC-derived organ bud transplant, Nat. Protoc., 9, 396, 10.1038/nprot.2014.020

Boj, 2015, Organoid models of human and mouse ductal pancreatic cancer, Cell, 160, 324, 10.1016/j.cell.2014.12.021

Xia, 2014, The generation of kidney organoids by differentiation of human pluripotent cells to ureteric bud progenitor-like cells, Nat. Protoc., 9, 2693, 10.1038/nprot.2014.182

Freedman, 2015, Modelling kidney disease with CRISPR-mutant kidney organoids derived from human pluripotent epiblast spheroids, Nat. Commun., 6, 8715, 10.1038/ncomms9715

Morizane, 2015, Nephron organoids derived from human pluripotent stem cells model kidney development and injury, Nat. Biotechnol., 33, 1193, 10.1038/nbt.3392

Takasato, 2015, Kidney organoids from human iPS cells contain multiple lineages and model human nephrogenesis, Nature, 526, 564, 10.1038/nature15695

Eiraku, 2011, Self-organizing optic-cup morphogenesis in three-dimensional culture, Nature, 472, 51, 10.1038/nature09941

Völkner, 2016, Retinal organoids from pluripotent stem cells efficiently recapitulate retinogenesis, Stem Cell Reports, 6, 525, 10.1016/j.stemcr.2016.03.001

Kuwahara, 2015, Generation of a ciliary margin-like stem cell niche from self-organizing human retinal tissue, Nat. Commun., 6, 6286, 10.1038/ncomms7286

Rouhani, 2016, Mutational history of a human cell lineage from somatic to induced pluripotent stem cells, PLoS Genet., 12, e1005932, 10.1371/journal.pgen.1005932

Somers, 2010, Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette, Stem Cells, 28, 1728, 10.1002/stem.495

Fukawatase, 2014, Ataxia telangiectasia derived iPS cells show preserved x-ray sensitivity and decreased chromosomal instability, Sci. Rep., 4, 5421, 10.1038/srep05421

Garçon, 2013, Ribosomal and hematopoietic defects in induced pluripotent stem cells derived from Diamond Blackfan anemia patients, Blood, 122, 912, 10.1182/blood-2013-01-478321

Ge, 2015, Dysregulation of the transforming growth factor β pathway in induced pluripotent stem cells generated from patients with Diamond Blackfan anemia, PLoS ONE, 10, e0134878, 10.1371/journal.pone.0134878

Li, 2012, Trisomy correction in Down syndrome induced pluripotent stem cells, Cell Stem Cell, 11, 615, 10.1016/j.stem.2012.08.004

Agarwal, 2010, Telomere elongation in induced pluripotent stem cells from dyskeratosis congenita patients, Nature, 464, 292, 10.1038/nature08792

Batista, 2011, Telomere shortening and loss of self-renewal in dyskeratosis congenita induced pluripotent stem cells, Nature, 474, 399, 10.1038/nature10084

Itoh, 2011, Generation of keratinocytes from normal and recessive dystrophic epidermolysis bullosa-induced pluripotent stem cells, Proc. Natl. Acad. Sci. U.S.A., 108, 8797, 10.1073/pnas.1100332108

Tolar, 2011, Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa, J. Invest. Dermatol., 131, 848, 10.1038/jid.2010.346

Raya, 2009, Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells, Nature, 460, 53, 10.1038/nature08129

Ye, 2009, Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders, Blood, 114, 5473, 10.1182/blood-2009-04-217406

Tulpule, 2013, Pluripotent stem cell models of Shwachman-Diamond syndrome reveal a common mechanism for pancreatic and hematopoietic dysfunction, Cell Stem Cell, 12, 727, 10.1016/j.stem.2013.04.002

Cheung, 2014, Telomerase protects werner syndrome lineage-specific stem cells from premature aging, Stem Cell Reports, 2, 534, 10.1016/j.stemcr.2014.02.006

Shimamoto, 2014, Reprogramming suppresses premature senescence phenotypes of Werner syndrome cells and maintains chromosomal stability over long-term culture, PLoS ONE, 9, e112900, 10.1371/journal.pone.0112900

Thatava, 2011, Successful disease-specific induced pluripotent stem cell generation from patients with kidney transplantation, Stem Cell Res. Ther., 2, 48, 10.1186/scrt89

Komor, 2016, Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage, Nature, 533, 420, 10.1038/nature17946

Menon, 2015, Lymphoid regeneration from gene-corrected SCID-X1 subject-derived iPSCs, Cell Stem Cell, 16, 367, 10.1016/j.stem.2015.02.005

Xie, 2014, Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac, Genome. Res., 24, 1526, 10.1101/gr.173427.114

Bellin, 2013, Isogenic human pluripotent stem cell pairs reveal the role of a KCNH2 mutation in long-QT syndrome, EMBO J., 32, 3161, 10.1038/emboj.2013.240