Modeling of hematologic malignancies by iPS technology

Experimental Hematology

Volumn 43, Issue 8, 2015, Pages 654-660

  Arai, Shunya ^a,b   Miyauchi, Masashi ^a,b   Kurokawa, Mineo ^a,b  

^a UNIVERSITY OF TOKYO   (Japan)

^b JAPAN SCIENCE AND TECHNOLOGY AGENCY   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

LENTIVIRUS VECTOR; RETROVIRUS VECTOR;

ACUTE MYELOBLASTIC LEUKEMIA; ASXL1 GENE; BCR ABL GENE; BIOTECHNOLOGY; BONE MARROW DEPRESSION; CARCINOGENESIS; CELL DIFFERENTIATION; CELL ISOLATION; CHROMOSOME 7Q; CHROMOSOME DELETION; CHROMOSOME LOSS; CHRONIC MYELOID LEUKEMIA; DNA CROSS LINKING; DYSKERATOSIS CONGENITA; FANCONI ANEMIA; GENE; GENETIC TRANSDUCTION; HEMATOLOGIC MALIGNANCY; HUMAN; JAK2 GENE; MUTATIONAL ANALYSIS; MYELODYSPLASTIC SYNDROME; MYELOPROLIFERATIVE NEOPLASM; NONHUMAN; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; REVIEW; RUNX1 GENE; SHWACHMAN SYNDROME; STEM CELL CULTURE; THROMBOCYTE DISORDER; TUMOR DIFFERENTIATION; TUMOR MODEL; VIRAL GENE DELIVERY SYSTEM; ANIMAL; BIOLOGICAL MODEL; CANCER STEM CELL; GENETICS; HEMATOLOGIC DISEASE; HEMATOPOIETIC STEM CELL; HUMAN CHROMOSOME; METABOLISM; PATHOLOGY;

IPS;

ANIMALS; CHROMOSOMES, HUMAN; HEMATOLOGIC NEOPLASMS; HEMATOPOIETIC STEM CELLS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MODELS, BIOLOGICAL; NEOPLASTIC STEM CELLS;

EID: 84939190535     PISSN: 0301472X     EISSN: 18732399     Source Type: Journal    
DOI: 10.1016/j.exphem.2015.06.006     Document Type: Review

References (47)

1. Takahashi K., Tanabe K., Ohnuki M., et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007, 131:861-872.
2. Yu J., Vodyanik M.A., Smuga-Otto K., et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007, 318:1917-1920.
3. Gao J., Yan X.L., Li R., et al. Characterization of OP9 as authentic mesenchymal stem cell line. J Genet Genomics 2010, 37:475-482.
4. Weisel K.C., Gao Y., Shieh J.H., et al. Stromal cell lines from the aorta-gonado-mesonephros region are potent supporters of murine and human hematopoiesis. Exp Hematol 2006, 34:1505-1516.
5. Takayama N., Nishikii H., Usui J., et al. Generation of functional platelets from human embryonic stem cells in vitro via ES-sacs, VEGF-promoted structures that concentrate hematopoietic progenitors. Blood 2008, 111:5298-5306.
6. Amabile G., Welner R.S., Nombela-Arrieta C., et al. In vivo generation of transplantable human hematopoietic cells from induced pluripotent stem cells. Blood 2013, 121:1255-1264.
7. Suzuki N., Yamazaki S., Yamaguchi T., et al. Generation of engraftable hematopoietic stem cells from induced pluripotent stem cells by way of teratoma formation. Mol Ther 2013, 21:1424-1431.
8. Kee Y., D'Andrea A.D. Molecular pathogenesis and clinical management of Fanconi anemia. J Clin Invest 2012, 122:3799-3806.
9. Raya A., Rodríguez-Pizà I., Guenechea G., et al. Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature 2009, 460:53-59.
10. Tulpule A., Kelley J.M., Lensch M.W., et al. Pluripotent stem cell models of Shwachman-Diamond syndrome reveal a common mechanism for pancreatic and hematopoietic dysfunction. Cell Stem Cell 2013, 12:727-736.
11. Agarwal S., Loh Y.H., McLoughlin E.M., et al. Telomere elongation in induced pluripotent stem cells from dyskeratosis congenita patients. Nature 2010, 464:292-296.
12. Batista L.F., Pech M.F., Zhong F.L., et al. Telomere shortening and loss of self-renewal in dyskeratosis congenita induced pluripotent stem cells. Nature 2011, 474:399-402.
13. Song W.J., Sullivan M.G., Legare R.D., et al. Haploinsufficiency of CBFA2 causes familial thrombocytopenia with propensity to develop acute myelogenous leukaemia. Nat Genet 1999, 23:166-175.
14. Harada Y., Harada H. Molecular pathways mediating MDS/AML with focus on AML1/RUNX1 point mutations. J Cell Physiol 2009, 220:16-20.
15. Sakurai M., Kunimoto H., Watanabe N., et al. Impaired hematopoietic differentiation of RUNX1-mutated induced pluripotent stem cells derived from FPD/AML patients. Leukemia 2014, 28:2344-2354.
16. Connelly J.P., Kwon E.M., Gao Y., et al. Targeted correction of RUNX1 mutation in FPD patient-specific induced pluripotent stem cells rescues megakaryopoietic defects. Blood 2014, 124:1926-1930.
17. Antony-Debré I., Manchev V.T., Balayn N., et al. Level of RUNX1 activity is critical for leukemic predisposition but not for thrombocytopenia. Blood 2015, 125:930-934.
18. Iizuka H., Kagoya Y., Kataoka K., et al. Targeted gene correction of RUNX1 in induced pluripotent stem cells derived from familial platelet disorder with propensity to myeloid malignancy restores normal megakaryopoiesis. Exp Hematol 2015, Epub ahead of print.
19. Zou J., Mali P., Huang X., et al. Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease. Blood 2011, 118:4599-4608.
20. Ye L., Chang J.C., Lin C., et al. Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases. Proc Natl Acad Sci U S A 2009, 106:9826-9830.
21. Wang Y., Zheng C.G., Jiang Y., et al. Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice. Cell Res 2012, 22:637-648.
22. Papapetrou E.P., Lee G., Malani N., et al. Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol 2011, 29:73-78.
23. Varela I., Karagiannidou A., Oikonomakis V., et al. Generation of human β-thalassemia induced pluripotent cell lines by reprogramming of bone marrow-derived mesenchymal stromal cells using modified mRNA. Cell Reprogram 2014, 16:447-455.
24. Kralovics R., Teo S., Li S., et al. Acquisition of the V617F mutation of JAK2 is a late genetic event in a subset of patients with myeloproliferative disorders. Blood 2006, 108:1377-1380.
25. Maclean G.A., Menne T.F., Guo G., et al. Altered hematopoiesis in trisomy 21 as revealed through in vitro differentiation of isogenic human pluripotent cells. Proc Natl Acad Sci U S A 2012, 109:17567-17572.
26. Park I.H., Arora N., Huo H., et al. Disease-specific induced pluripotent stem cells. Cell 2008, 134:877-886.
27. Chou S.T., Byrska-Bishop M., Tober J.M., et al. Trisomy 21-associated defects in human primitive hematopoiesis revealed through induced pluripotent stem cells. Proc Natl Acad Sci U S A 2012, 109:17573-17578.
28. Mou X., Wu Y., Cao H., et al. Generation of disease-specific induced pluripotent stem cells from patients with different karyotypes of Down syndrome. Stem Cell Res Ther 2012, 3:14.
29. Byrska-Bishop M., VanDorn D., Campbell A.E., et al. Pluripotent stem cells reveal erythroid-specific activities of the GATA1 N-terminus. J Clin Invest 2015, 125:993-1005.
30. Ye Z., Liu C.F., Lanikova L., et al. Differential sensitivity to JAK inhibitory drugs by isogenic human erythroblasts and hematopoietic progenitors generated from patient-specific induced pluripotent stem cells. Stem Cells 2014, 32:269-278.
31. Hosoi M., Kumano K., Taoka K., et al. Generation of induced pluripotent stem cells derived from primary and secondary myelofibrosis patient samples. Exp Hematol 2014, 42:816-825.
32. Yamamoto S., Otsu M., Matsuzaka E., et al. Screening of drugs to treat 8p11 myeloproliferative syndrome using patient-derived induced pluripotent stem cells with fusion gene CEP110-FGFR1. PLoS One 2015, 10:e0120841.
33. Saliba J., Hamidi S., Lenglet G., et al. Heterozygous and homozygous JAK2(V617F) states modeled by induced pluripotent stem cells from myeloproliferative neoplasm patients. PLoS One 2013, 8:e74257.
34. Kotini A.G., Chang C.J., Boussaad I., et al. Functional analysis of a chromosomal deletion associated with myelodysplastic syndromes using isogenic human induced pluripotent stem cells. Nat Biotechnol 2015, 33:646-655.
35. Hu K., Yu J., Suknuntha K., et al. Efficient generation of transgene-free induced pluripotent stem cells from normal and neoplastic bone marrow and cord blood mononuclear cells. Blood 2011, 117:e109-e119.
36. Carette J.E., Pruszak J., Varadarajan M., et al. Generation of iPSCs from cultured human malignant cells. Blood 2010, 115:4039-4042.
37. Kumano K., Arai S., Hosoi M., et al. Generation of induced pluripotent stem cells from primary chronic myelogenous leukemia patient samples. Blood 2012, 119:6234-6242.
38. Bedel A., Pasquet J.M., Lippert E., et al. Variable behavior of iPSCs derived from CML patients for response to TKI and hematopoietic differentiation. PLoS One 2013, 8:e71596.
39. Ye Z., Zhan H., Mali P., et al. Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders. Blood 2009, 114:5473-5480.
40. Sebastiano V., Maeder M.L., Angstman J.F., et al. In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases. Stem Cells 2011, 29:1717-1726.
41. Sakuma T., Hosoi S., Woltjen K., et al. Efficient TALEN construction and evaluation methods for human cell and animal applications. Genes Cells 2013, 18:315-326.
42. Horii T., Tamura D., Morita S., et al. Generation of an ICF syndrome model by efficient genome editing of human induced pluripotent stem cells using the CRISPR system. Int J Mol Sci 2013, 14:19774-19781.
43. Hou Z., Zhang Y., Propson N.E., et al. Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis. Proc Natl Acad Sci U S A 2013, 110:15644-15649.
44. Bauer D.E., Kamran S.C., Orkin S.H. Reawakening fetal hemoglobin: prospects for new therapies for the β-globin disorders. Blood 2012, 120:2945-2953.
45. Sandler V.M., Lis R., Liu Y., et al. Reprogramming human endothelial cells to haematopoietic cells requires vascular induction. Nature 2014, 511:312-318.
46. Elcheva I., Brok-Volchanskaya V., Kumar A., et al. Direct induction of haematoendothelial programs in human pluripotent stem cells by transcriptional regulators. Nat Commun 2014, 5:4372.
47. Doulatov S., Vo L.T., Chou S.S., et al. Induction of multipotential hematopoietic progenitors from human pluripotent stem cells via respecification of lineage-restricted precursors. Cell Stem Cell 2013, 13:459-470.