Hematopoietic stem cell expansion and generation: the ways to make a breakthrough

다국어 초록 (Multilingual Abstract)

Hematopoietic stem cell transplantation (HSCT) is the first field where human stem cell therapy was successful. Flooding interest on human stem cell therapy to cure previously incurable diseases is largely indebted to HSCT success. Allogeneic HSCT has been an im-portant modality to cure various diseases including hematologic malignancies, various non-malignant hematologic diseases, primary immunodeficiency diseases, and inborn errors of metabolism, while autologous HSCT is generally performed to rescue bone mar-row aplasia following high-dose chemotherapy for solid tumors or multiple myeloma. Recently, HSCs are also spotlighted in the field of regenerative medicine for the amelio-ration of symptoms caused by neurodegenerative diseases, heart diseases, and others. Although the demand for HSCs has been growing, their supply often fails to meet the demand of the patients needing transplant due to a lack of histocompatible donors or a limited cell number. This review focuses on the generation and large-scale expansion of HSCs, which might overcome current limitations in the application of HSCs for clinical use. Furthermore, current proof of concept to replenish hematological homeostasis from non-hematological origin will be covered.

참고문헌 (Reference)

1 김창성, "iPSC technology-Powerful hand for disease modeling and therapeutic screen" 생화학분자생물학회 48 (48): 256-265, 2015

2 Gaj T, "ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering" 31 : 397-405, 2013

3 Geron Corporation, "World's first clinical trial of human embryonic stem cell therapy cleared" 4 : 161-, 2009

4 Sturgeon CM, "Wnt signaling controls the specification of definitive and primitive hematopoiesis from human pluripotent stem cells" 32 : 554-561, 2014

5 Gori JL, "Vascular niche promotes hematopoietic multipotent progenitor formation from pluripotent stem cells" 125 : 1243-1254, 2015

6 Oran B, "Umbilical cord blood transplantation : a maturing technology" 2012 : 215-222, 2012

7 Horwitz ME, "Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment" 124 : 3121-3128, 2014

8 Kanemura H, "Tumorigenicity studies of induced pluripotent stem cell (iPSC)-derived retinal pigment epithelium (RPE) for the treatment of age-related macular degeneration" 9 : e85336-, 2014

9 Hanna J, "Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin" 318 : 1920-1923, 2007

10 Song WK, "Treatment of macular degeneration using embryonic stem cell-derived retinal pigment epithelium : preliminary results in Asian patients" 4 : 860-872, 2015

11 Meuwissen HJ, "Treatment of lymphopenic hypogammaglobulinemia and bonemarrow aplasia by transplantation of allogeneic marrow. Crucial role of histocompatiility matching" 281 : 691-697, 1969

12 de Lima M, "Transplantation of ex vivo expanded cord blood cells using the copper chelator tetraethylenepentamine : a phase I/II clinical trial" 41 : 771-778, 2008

13 Barker JN, "Transplantation of 2partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy" 105 : 1343-1347, 2005

14 Liu Z, "Transdifferentiation of Human Hair Follicle Mesenchymal Stem Cells into Red Blood Cells by OCT4" 2015 : 389628-, 2015

15 Lu R, "Tracking single hematopoietic stem cells in vivo using high-throughput sequencing in conjunction with viral genetic barcoding" 29 : 928-933, 2011

16 Joung JK, "TALENs : a widely applicable technology for targeted genome editing" 14 : 49-55, 2013

17 Kennedy M, "T lymphocyte potential marks the emergence of definitive hematopoietic progenitors in human pluripotent stem cell differentiation cultures" 2 : 1722-1735, 2012

18 Galic Z, "T lineage differentiation from human embryonic stem cells" 103 : 11742-11747, 2006

19 Wada H, "Successful differentiation to T cells, but unsuccessful B-cell generation, from B-cell-derived induced pluripotent stem cells" 23 : 65-74, 2011

20 Real PJ, "SCL/TAL1 regulates hematopoietic specification from human embryonic stem cells" 20 : 1443-1453, 2012

21 Sandler VM, "Reprogramming human endothelial cells to haematopoietic cells requires vascular induction" 511 : 312-318, 2014

22 Riddell J, "Reprogramming committed murine blood cells to induced hematopoietic stem cells with defined factors" 157 : 549-564, 2014

23 Ran D, "RUNX1a enhances hematopoietic lineage commitment from human embryonic stem cells and inducible pluripotent stem cells" 121 : 2882-2890, 2013

24 Lu SJ, "Platelets generated from human embryonic stem cells are functional in vitro and in the microcirculation of living mice" 21 : 530-545, 2011

25 Brehm MA, "Overcoming current limitations in humanized mouse research" 208 : S125-S130, 2013

26 Petersdorf EW, "Optimizing outcome after unrelated marrow transplantation by comprehensive matching of HLA class I and II alleles in the donor and recipient" 92 : 3515-3520, 1998

27 Wagner JE Jr EM, "One-unit versus two-unit cord-blood transplantation for hematologic cancers" 371 : 1685-1694, 2014

28 Delaney C, "Notch-mediated expansion of human cord blood progenitor cells capable of rapid myeloid reconstitution" 16 : 232-236, 2010

29 Peled T, "Nicotinamide, a SIRT1inhibitor, inhibits differentiation and facilitates expansion of hematopoietic progenitor cells with enhanced bone marrow homing and engraftment" 40 : 342-355.e1, 2012

30 Reardon S, "Japan stem-cell trial stirs envy" 513 : 287-288, 2014

31 Young JC, "Inhibitors of histone deacetylases promote hematopoietic stem cell self-renewal" 6 : 328-336, 2004

32 Takahashi K, "Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors" 126 : 663-676, 2006

33 Doulatov S, "Induction of multipotential hematopoietic progenitors from human pluripotent stem cells via respecification of lineage-restricted precursors" 13 : 459-470, 2013

34 Amabile G, "In vivo generation of transplantable human hematopoietic cells from induced pluripotent stem cells" 121 : 1255-1264, 2013

35 Larochelle A, "Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow : implications for gene therapy" 2 : 1329-1337, 1996

36 Tanner A, "Humanized mice as a model to study human hematopoietic stem cell transplantation" 23 : 76-82, 2014

37 Carpenter L, "Human induced pluripotent stem cells are capable of B-cell lymphopoiesis" 117 : 4008-4011, 2011

38 Wang L, "Human embryonic stem cells maintained in the absence of mouse embryonic fibroblasts or conditioned media are capable of hematopoietic development" 105 : 4598-4603, 2005

39 Woll PS, "Human embryonic stem cells differentiate into a homogeneous population of natural killer cells with potent in vivo antitumor activity" 113 : 6094-6101, 2009

40 Vodyanik MA, "Human embryonic stem cell-derived CD34+ cells : efficient production in the coculture with OP9 stromal cells and analysis of lymphohematopoietic potential" 105 : 617-626, 2005

41 Matsuoka Y, "Human cord blood-derived primitive CD34-negative hematopoietic stem cells (HSCs) are myeloid-biased long-term repopulating HSCs" 5 : e290-, 2015

42 Weidner CI, "Hematopoietic stem and progenitor cells acquire distinct DNA-hypermethylation during in vitro culture" 3 : 3372-, 2013

43 Ramos-Mejía V, "HOXA9promotes hematopoietic commitment of human embryonic stem cells" 124 : 3065-3075, 2014

44 Berges BK, "HIV-1infection and CD4 T cell depletion in the humanized Rag2-/-gamma c-/-(RAG-hu)mouse model" 3 : 76-, 2006

45 Lang J, "Generation of hematopoietic humanized mice in the newborn BALB/c-Rag2null Il2rγnull mouse model : a multivariable optimization approach" 140 : 102-116, 2011

46 Suzuki N, "Generation of engraftable hematopoietic stem cells from induced pluripotent stem cells by way of teratoma formation" 21 : 1424-1431, 2013

47 Taylor CJ, "Generating an iPSC bank for HLA-matched tissue transplantation based on known donor and recipient HLA types" 11 : 147-152, 2012

48 Nakamura S, "Expandable megakaryocyte cell lines enable clinically applicable generation of platelets from human induced pluripotent stem cells" 14 : 535-548, 2014

49 Xie J, "Ex vivo expansion of hematopoietic stem cells" 58 : 839-853, 2015

50 Chaurasia P, "Epigenetic reprogramming induces the expansion of cord blood stem cells" 124 : 2378-2395, 2014

51 Lee K, "Engraftment of human iPS cells and allogeneic porcine cells into pigs with inactivated RAG2 and accompanying severe combined immunodeficiency" 111 : 7260-7265, 2014

52 Schwartz SD, "Embryonic stem cell trials for macular degeneration : a preliminary report" 379 : 713-720, 2012

53 Ledran MH, "Efficient hematopoietic differentiation of human embryonic stem cells on stromal cells derived from hematopoietic niches" 3 : 85-98, 2008

54 Gori JL, "Efficient generation, purification, and expansion of CD34(+) hematopoietic progenitor cells from nonhuman primate-induced pluripotent stem cells" 120 : 35-44, 2012

55 Scaradavou A, "Double unit grafts successfully extend the application of umbilical cord blood transplantation in adults with acute leukemia" 121 : 752-758, 2013

56 Chen U, "Do we have a workable clinical protocol for differentiating lympho-hematopoietic stem cells from the source of embryonic stem cells and induced pluripotent stem cells in culture?" 80 : 247-249, 2014

57 김창성, "Disease modeling and cell based therapy with iPSC: future therapeutic option with fast and safe application" 대한혈액학회 49 (49): 7-14, 2014

58 Batta K, "Direct reprogramming of murine fibroblasts to hematopoietic progenitor cells" 9 : 1871-1884, 2014

59 Szabo E, "Direct conversion of human fibroblasts to multilineage blood progenitors" 468 : 521-526, 2010

60 Ishii M, "Development of a highresolution purification method for precise functional characterization of primitive human cord blood-derived CD34-negative SCID-repopulating cells" 39 : 203-213.e1, 2011

61 Pasquini MC, "Current uses and outcomes of hematopoietic stem cell transplantation: 2014 CIBMTR summary slides"

62 de Lima M, "Cord-blood engraftment with ex vivo mesenchymal-cell coculture" 367 : 2305-2315, 2012

63 Knorr DA, "Clinical-scale derivation of natural killer cells from human pluripotent stem cells for cancer therapy" 2 : 274-283, 2013

64 Araki H, "Chromatin-modifying agents permit human hematopoietic stem cells to undergo multiple cell divisions while retaining their repopulating potential" 109 : 3570-3578, 2007

65 Maeder ML, "CRISPR RNA-guided activation of endogenous human genes" 10 : 977-979, 2013

66 Hansen JA, "Bone marrow transplants from unrelated donors for patients with chronic myeloid leukemia" 338 : 962-968, 1998

67 Silva LC, "Anti-TNF-α agents in the treatment of immune-mediated inflammatory diseases : mechanisms of action and pitfalls" 2 : 817-833, 2010

68 Pineault N, "Advances in umbilical cord blood stem cell expansion and clinical translation" 43 : 498-513, 2015

69 Wagner JE, "Acceleration of umbilical cord blood (UCB) stem engraftment: Results of a phase I clinical trial with stemregenin-1 (SR1) expansion culture" 21 : S48-S49, 2015

70 Kim H, "A guide to genome engineering with programmable nucleases" 15 : 321-334, 2014

71 Schlaeger TM, "A comparison of non-integrating reprogramming methods" 33 : 58-63, 2015

72 Elman JS, "A comparison of adipose and bone marrow-derived mesenchymal stromal cell secreted factors in the treatment of systemic inflammation" 11 : 1-, 2014

73 Ding Q, "A TALEN genome-editing system for generating human stem cell-based disease models" 12 : 238-251, 2013