Stem cell bioengineering for regenerative medicine

Expert Opinion on Biological Therapy

Volumn 4, Issue 5, 2004, Pages 631-644

  Audet, Julie ^a  

^a UNIVERSITY OF TORONTO   (Canada)

Author keywords

Bioengineering; Cellular therapy; Gene therapy; Regenerative medicine; Stem cell culture; Stem cells; Tissue engineering; Transplantation

Indexed keywords

BIOENGINEERING; CELL CULTURE; CELL DIFFERENTIATION; CELL FUNCTION; CELL PROLIFERATION; CELL REGENERATION; EMBRYO CELL; HUMAN; MULTIVARIATE ANALYSIS; REVIEW; STEM CELL; TISSUE ENGINEERING;

ANIMALS; CELLS, CULTURED; ENVIRONMENT; HUMANS; PROTEIN ENGINEERING; REGENERATION; STEM CELLS; TISSUE ENGINEERING;

ANIMALIA;

EID: 2442692762     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.4.5.631     Document Type: Review

References (173)

1. BLAU HM, BRAZELTON TR, WEIMANN JM: The evolving concept of a stem cell: entity or function? Cell (2001) 105(7):829-841.
2. ZANDSTRA PW, NAGY A: Stem cell bioengineering. Ann. Rev. Biomed. Eng. (2001) 3:275-305.
3. PETIT-ZEMAN S: Regenerative medicine. Nat. Biotechnol. (2001) 19(3):201-206.
4. LOEBEL DAF, WATSON CM, DE YOUNG RA, TAM PPL: Lineage choice and differentiation in mouse embryos and embryonic stem cells. Dev. Biol. (2003) 264(1):1-14.
5. ZON LI: Self-renewal versus differentiation, a job for the mighty morphogens. Nat. Immunol. (2001) 2:142-143.
6. SMITH AG, HEATH JK, DONALDSON DD et al.: Inhibition of pluripotential embryonic stem cell differentiation by purified polypeptides. Nature (1988) 336(6200):688-690.
7. LUMELSKY N, BLONDEL O, LAENG P, VELASCO I, RAVIN R, MCKAY R: Differentiation of embryonic stem cells to insulin-secreting structures similar to pancreatic islets. Science (2001) 292(5520):1389-1394. Suggests feasibility of ES cell-based therapy for diabetes.
8. KENNEDY M, FIRPO M, CHOI K et al.: A common precursor for primitive erythropoiesis and definitive haematopoiesis. Nature (1997) 386(6624):488-493.
9. HORI Y, RULIFSON IC, TSAI BC, HEIT JJ, CAHOY JD, KIM SK: Growth inhibitors promote differentiation of insulin-producing tissue from embryonic stem cells. Proc. Natl. Acad Sci. USA (2002) 99(25):16105-16110.
10. WICHTERLE H, LIEBERAM I, PORTER JA, JESSELL TM: Directed differentiation of embryonic stem cells into motor neurons. Cell (2002) 110(3):385-397. In vitro manipulation of instructive signals to drive differentiation.
11. SHAMBLOTT MJ, AXELMAN J, WANG S et al.: Derivation of pluripotent stem cells from cultured human primordial germ cells. Proc. Natl. Acad. Sci. USA (1998) 95:13726-13731.
12. THOMSON JA, ITSKOVITZ-ELDOR J, SHAPIRO SS et al.: Embryonic stem cell lines derived from human blastocysts. Science (1998) 282:1145-1147.
13. XU C, INOKUMA MS, DENHAM J et al.: Feeder-free growth of undifferentiated human embryonic stem cells. Nat. Biotechnol. (2001) 19(10):971-974.
14. ROSLER ES, FISK GJ, ARES X et al.: Long-term culture of human embryonic stem cells in feeder-free conditions. Dev. Dyn. (2004) 229(2):259-274.
15. LIU S, QU Y, STEWART TJ et al.: Embryonic stem cells differentiate into oligodendrocytes and myelinate in culture and after spinal cord transplantation. Proc. Natl. Acad. Sci. USA (2000) 97(11):6126-6131.
16. BARBERI T, KLIVENYI P, CALINGASAN NY et al.: Neural subtype specification of fertilization and nuclear transfer embryonic stem cells and application in parkinsonian mice. Nat. Biotechnol. (2003) 21(10):1200-1207.
17. KIM JH, AUERBACH JM, RODRIGUEZ-GOMEZ JA et al.: Dopamine neurons derived from embryonic stem cells function in an animal model of Parkinson's disease. Nature (2002) 418(6893):50-56. Functional evaluation of cultured NSCs.
18. NISHIMURA F, YOSHIKAWA M, KANDA S et al.: Potential use of embryonic stem cells for the treatment of mouse parkinsonian models: Improved behavior by transplantation of in vitro differentiated dopaminergic neurons from embryonic stem cells. Stem Cells (2003) 21(2):171-180.
19. SASAI Y: Generation of dopaminergic neurons from embryonic stem cells. J. Neurol. (2002) 249(Suppl. 2):II41-II44.
20. ZHANG SC, WERNIG M, DUNCAN ID, BRUSTLE O, THOMSON JA: In vitro differentiation of transplantable neural precursors from human embryonic stem cells. Nat. Biotechnol. (2001) 19(12):1129-1133.
21. REUBINOFF BE, ITSYKSON P, TURETSKY T et al.: Neural progenitors from human embryonic stem cells. Nat. Biotechnol. (2001) 19(12):1134-1140.
22. BJORKLUND LM, SANCHEZ-PERNAUTE R, CHUNG S et al.: Embryonic stem cells develop into functional dopaminergic neurons after transplantation in a Parkinson rat model. Proc. Natl. Acad. Sci. USA (2002) 99(4):2344-2349.
23. DYER M, FARRINGTON S, MOHN D, MUNDAY J, BARON M: Indian hedgehog activates hematopoiesis and vasculogenesis and can respecify prospective neurectodermal cell fate in the mouse embryo. Development (2001) 128(10):1717-1730.
24. RIDEOUT WM III, HOCHEDLINGER K, KYBA M, DALEY GQ, JAENISCH R: Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy. Cell (2002) 109(1):17-27. Suggests feasibility of therapeutic cloning.
25. DALEY GQ: From embryos to embryoid bodies: Generating blood from embryonic stem cells. Ann. NY Acad. Sci. (2003) 996(1):122-131.
26. KAUFMAN DS, HANSON ET, LEWIS RL, AUERBACH R, THOMSON JA: Hematopoietic colony-forming cells derived from human embryonic stem cells. Proc. Natl. Acad. Sci. USA (2001) 98(19):10716-10721.
27. DOEVENDANS PA, KUBALAK SW, AN RH, BECKER DK, CHIEN KR, KASS RS: Differentiation of cardiomyocytes in floating embryoid bodies is comparable to fetal cardiomyocytes. J. Mol. Cell. Cardiol. (2000) 32(5):839-851.
28. KEHAT I, KENYAGIN-KARSENTI D, SNIR M et al.: Human embryonic stem cells can differentiate into myocytes with structural and functional properties of cardiomyocytes. J. Clin. Invest. (2001) 108(3):407-414.
29. KACZOROWSKI DJ, PATTERSON ES, JASTROMB WE, SHAMBLOTT MJ: Glucose-responsive insulin-producing cells from stem cells. Diabetes Metab. Res. Rev. (2002) 18(6):442-450.
30. ASSADY S, MAOR G, AMIT M, ITSKOVITZ-ELDOR J, SKORECKI KL, TZUKERMAN M: Insulin production by human embryonic stem cells. Diabetes (2001) 50(8):1691-1697.
31. HÜBNER K, FUHRMANN G, CHRISTENSON LK et al.: Derivation of oocytes from mouse embryonic stem cells. Science (2003) 300(5623):1251-1256.
32. GEIJSEN N, HOROSCHAK M, KIM K, GRIBNAU J, EGGAN K, DALEY GQ: Derivation of embryonic germ cells and male gametes from embryonic stem cells. Nature (2004) 427(6970):148-154.
33. MATSUDA T, NAKAMURA T, NAKAO K et al.: STAT3 activation is sufficient to maintain an undifferentiated state of mouse embryonic stem cells. EMBO J. (1999) 18(15):4261-4269.
34. ZANDSTRA PW, LE H-V, DALEY GQ, GRIFFITH LG, LAUFFENBURGER DA: Leukemia inhibitory factor (LIF) concentration modulates embryonic stem cell self-renewal and differentiation independently of proliferation. Biotechnol. Bioeng. (2000) 69:607-617.
35. VISWANATHAN S, BENATAR T, MILEIKOVSKY M, LAUFFENBURGER DA, NAGY A, ZANDSTRA PW: Supplementation-dependent differences in the rates of embryonic stem cell self-renewal, differentiation, and apoptosis. Biotechnol. Bioeng. (2003) 84(5):505-517.
36. VISWANATHAN S, BENATAR T, ROSE-JOHN S, LAUFFENBURGER DA, ZANDSTRA PW: Ligand/receptor signaling threshold (LIST) model accounts for gp130-mediated embryonic stem cell self-renewal responses to LIF and HIL-6. Stem Cells (2002) 20(2):119-138. Insights into the action mechanisms of cytokines that signal through gp130.
37. YING QL, NICHOLS J, CHAMBERS I, SMITH A: BMP induction of Id proteins suppresses differentiation and sustains embryonic stem cell self-renewal in collaboration with STAT3. Cell (2003) 115 (3):281-292. Identification of molecular events controlling stem cell self-renewal.
38. MOORE KA, EMA H, LEMISCHKA IR. In vitro maintenance of highly purified, transplantable hematopoietic stem cells. Blood (1997) 89:4337-4347.
39. VARNUM-FINNEY B, PURTON LE, YU M et al.: The Notch ligand, Jagged-1, influences the development of primitive hematopoietic precursor cells. Blood (1998) 91:4084-4091.
40. KARANU FN, MURDOCH B, MIYABAYASHI T et al.: Human homologues of Delta-1 and Delta-4 function as mitogenic regulators of primitive human hematopoietic cells. Blood (2001) 97:1960-1967.
41. VAN DEN BERG DJ, SHARMA AK, BRUNO E, HOFFMAN R: Role of members of the Wnt gene family in human hematopoiesis. Blood (1998) 92:3189-3202.
42. EAVES CJ: Manipulating hematopoietic stem cell amplification with Wnt. Nat. Immunol. (2003) 4:511-512.
43. WILLERT K, BROWN JD, DANENBERG E et al.: Wnt proteins are lipid-modified and can act as stem cell growth factors. Nature (2003) 423(6938):448-452. Demonstrates the usefulness of wnt ligands for HSC expansion in vitro.
44. DE HAAN G, WEERSING E, DONTJE B et al.: In vitro generation of long-term repopulating hematopoietic stem cells by fibroblast growth factor-1. Dev. Cell (2003) 4(2):241-251.
45. UEDA T, TSUJI K, YOSHINO H et al.: Expansion of human NOD/SCID-repopulating cells by stem cell factor, Flk2/ Flt3 ligand, thrombopoietin, IL-6 and soluble IL-6 receptor. J. Clin. Invest. (2000) 105:1013-1021.
46. AUDET J, MILLER CL, ROSE-JOHN S, PIRET JM, EAVES CJ: Distinct role of gp130 activation in promoting self-renewal divisions by mitogenically stimulated murine hematopoietic stem cells. Proc. Natl. Acad. Sci. USA (2001) 98:1757-1762.
47. EMA H, TAKANO H, SUDO K, NAKAUCHI H: In vitro self-renewal division of hematopoietic stem cells. J. Exp. Med. (2000) 192(9):1281-1288.
48. BHATIA M, BONNET D, WU D et al.: Bone morphogenic proteins regulate the developmental program of human hematopoietic stem cells. J. Exp. Med. (1999) 189:1139-1148.
49. BHARDWAJ G, MURDOCH B, WU D et al.: Sonic hedgehog induces the proliferation of primitive human hematopoietic cells via BMP regulation. Nat. Immunol. (2001) 2:172-180.
50. MOSTAFA SS, PAPOUTSAKIS ET, MILLER WM: Oxygen tension modulates the expression of cytokine receptors, transcription factors, and lineage-specific markers in cultured human megakaryocytes. Exp. Hematol. (2001) 29(7):873-883.
51. MOSTAFA SS, MILLER WM, PAPOUTSAKIS ET: Oxygen tension influences the differentiation, maturation and apoptosis of human megakaryocytes. Br. J. Haematol. (2000) 111(3):879-889. Demonstrates the influence of physicochemical parameters in HSC culture.
52. YANG H, MILLER WM, PAPOUTSAKIS ET: Higher pH promotes megakaryocytic maturation and apoptosis. Stem Cells (2002) 20(4):320-328.
53. MCADAMS TA, MILLER WM, PAPOUTSAKIS ET: pH is a potent modulator of erythroid differentiation. Br. J. Haematol. (1998) 103:317-325.
54. HEVEHAN DL, PAPOUTSAKIS ET, MILLER WM: Physiologically significant effects of pH and oxygen tension on granulopoiesis. Exp. Hematol. (2000) 28:267-275.
55. HEVEHAN DL, WENNING LA, MILLER WM, PAPOUTSAKIS ET: Dynamic model of ex vivo granulocytic kinetics to examine the effects of oxygen tension, pH, and interleukin-3. Exp. Hematol. (2000) 28(9):1016-1028.
56. REYNOLDS BA, WEISS S: Generation of neurons and astrocytes from isolated cells of the adult mammalian central nervous system. Science (1992) 255(5052):1707-1710.
57. MORSHEAD CM, REYNOLDS BA, CRAIG CG et al.: Neural stem cells in the adult mammalian forebrain: a relatively quiescent subpopulation of subependymal cells. Neuron (1994) 13(5):1071-1082.
58. DOETSCH F, PETREANU L, CAILLE I, GARCIA-VERDUGO JM, ALVAREZ-BUYLLA A: EGF converts transit-amplifying neurogenic precursors in the adult brain into multipotent stem cells. Neuron (2002) 36(6):1021-1034.
59. PEVNY L, RAO MS: The stem-cell menagerie. Trends Neurosci. (2003) 26(7):351-359.
60. SHIMAZAKI T, SHINGO T, WEISS S: The ciliary neurotrophic factor/leukemia inhibitory factor/gp130 receptor complex operates in the maintenance of mammalian forebrain neural stem cells. J. Neurosci. (2001) 21(19):7642-7653.
61. LAI K, KASPAR BK, GAGE FH, SCHAFFER DV: Sonic hedgehog regulates adult neural progenitor proliferation in vitro and in vivo. Nat. Neurosci. (2003) 6(1):21-27. Insight into the signalling factors regulating NSC proliferation.
62. RAFF M: Adult stem cell plasticity: factor artifact? Ann. Rev. Cell Dev. Biol. (2003) 19(1):1-22.
63. PLUCHINO S, QUATTRINI A, BRAMBILLA E et al.: Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis. Nature (2003) 422(6933):688-694. Functional evaluation of cultured neurospheres.
64. BEN-HUR T, EINSTEIN O, MIZRACHI-KOL R et al.: Transplanted multipotential neural precursor cells migrate into the inflamed white matter in response to experimental autoimmune encephalomyelitis. Glia (2003) 41(1):73-80.
65. GERMAIN L, GOULET F, MOULIN V, BERTHOD F, AUGER FA: Engineering human tissues for in vivo applications. Ann. NY Acad. Sci. (2002) 961(1):268-270.
66. ATALA A: Tissue engineering, stem cells, and cloning for the regeneration of urologic organs. Clin. Plast. Surg. (2003) 30(4):649-667.
67. BJORNSON CR, RIETZE RL, REYNOLDS BA, MAGLI MC, VESCOVI AL: Turning brain into blood: a hematopoietic fare adopted by adult neural stem cells in vivo. Science (1999) 283:534-537.
68. CLARKE DL, JOHANSSON CB, WILBERTZ J et al.: Generalized potential of adult neural stem cells. Science (2000) 288:1660-1663.
69. TANIGUCHI H, TOYOSHIMA T, FUKAO K, NAKAUCHI H: Presence of hematopoietic stem cells in the adult liver. Nat. Med. (1996) 2:198-203.
70. GUSSONI E, SONEOKA Y, STRICKLAND CD et al.: Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature (1999) 401:390-394.
71. JACKSON KA, MI T, GOODELL MA: Hematopoietic potential of stem cells isolated from murine skeletal muscle. Proc. Natl. Acad. Sci. USA (1999) 96:14482-14486.
72. SEALE P, SABOURIN LA, GIRGIS-GABARDO A, MANSOURI A, GRUSS P, RUDNICKI MA: Pax7 is required for the specification of myogenic satellite cells. Cell (2000) 102(6):777-786.
73. PEREIRA RF, O'HARA MD, LAPTEV AV et al.: Marrow stromal cells as a source of progenitor cells for nonhematopoietic tissues in transgenic mice with a phenotype of osteogenesis imperfecta. Proc. Natl. Acad. Sci. USA (1998) 95:1142-1147.
74. HORWITZ EM, PROCKOP DJ, FITZPATRICK LA et al.: Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta. Nat. Med. (1999) 5:309-313.
75. FERRARI G, CUSELLA-DE ANGELIS G, COLETTA M et al.: Muscle regeneration by bone marrow-derived myogenic progenitors. Science (1998) 279:1528-1530.
76. PETERSEN BE, BOWEN WC, PATRENE KD et al.: Bone marrow as a potential source of hepatic oval cells. Science (1999) 284:1168-1170.
77. LAGASSE E, CONNORS H, AL-DHALIMY M et al.: Purified hematopoietic stem cells can differentiate into hepatocytes in vivo. Nat. Med. (2000) 6:1229-1234.
78. EGLITIS MA, MEZEY E: Hematopoietic cells differentiate into both microglia and macroglia in the brains of adult mice. Proc. Natl. Acad. Sci. USA (1997) 94:4080-4085.
79. KOPEN GC, PROCKOP DJ, PHINNEY DG: Marrow stromal cells migrate throughout forebrain and cerebellum, and they differentiate into astrocytes after injection into neonatal mice brains. Proc. Natl. Acad. Sci. USA (1999) 96:10711-10716.
80. BRAZELTON TR, ROSSI FM, KESHET GI, BLAU HM: From marrow to brain: expression of neuronal phenotypes in adult mice. Science (2000) 290(5497):1775-1779.
81. SHI Q, RAFII S, WU MH et al.: Evidence for circulating bone marrow-derived endothelial cells. Blood (1998) 92:362-367.
82. WANG X, WILLENBRING H, AKKARI Y et al.: Cell fusion is the principal source of bone-marrow-derived hepatocytes. Nature (2003) 422(6934):897-901.
83. MCKINNEY-FREEMAN SL, MAJKA SM, JACKSON KA, NORWOOD K, HIRSCHI KK, GOODELL MA: Altered phenotype and reduced function of muscle-derived hematopoietic stem cells. Exp. Hematol. (2003) 31(9):806-814.
84. WAGERS AJ, WEISSMAN IL: Plasticity of adult stem cells. Cell (2004) 116(5):639-648.
85. KRAUSE DS, THEISE ND, COLLECTOR MI et al.: Multi-organ, multi-lineage engraftment by a single bone marrow-derived stem cell. Cell (2001) 105:369-377. Evaluation of bone marrow stem cell plasticity at the single-cell level.
86. CORBEL SY, LEE A, YI L et al.: Contribution of hematopoietic stem cells to skeletal muscle. Nat. Med. (2003) 9(12):1528-1532.
87. BAKSH D, DAVIES JE, ZANDSTRA PW: Adult human bone marrow-derived mesenchymal progenitor cells are capable of adhesion-independent survival and expansion. Exp. Hematol. (2003) 31(8):723-732.
88. JIANG Y, JAHAGIRDAR BN, REINHARDT RL et al.: Pluripotency of mesenchymal stem cells derived from adult marrow. Nature (2002) 418(6893):41-49. Characterisation of MAPCs.
89. VERFAILLIE CM, SCHWARTZ R, REYES M, JIANG Y: Unexpected potential of adult stem cells. Ann. NY Acad. Sci. (2003) 996(1):231-234.
90. JIANG Y, HENDERSON D, BLACKSTAD M, CHEN A, MILLER RF, VERFAILLIE CM: Neuroectodermal differentiation from mouse multipotent adult progenitor cells. Proc. Natl. Acad. Sci. USA (2003) 100(Suppl. 1):11854-11860.
91. SCHWARTZ RE, REYES M, KOODIE L et al.: Multipotent adult progenitor cells from bone marrow differentiate into functional hepatocyte-like cells. J. Clin. Invest. (2002) 109(10):1291-1302.
92. JIANG Y, VAESSEN B, LENVIK T, BLACKSTAD M, REYES M, VERFAILLIE CM: Multipotent progenitor cells can be isolated from postnatal murine bone marrow, muscle, and brain. Exp. Hematol. (2002) 30(8):896-904.
93. TOMA JG, AKHAVAN M, FERNANDES KJ et al.: Isolation of multipotent adult stem cells from the dermis of mammalian skin. Nat. Cell Biol. (2001) 3:778-784. Discovery of a new source of multipotent progenitors in the adult.
94. SZILVASSY SJ, NICOLINI FE, EAVES CJ, MILLER CL: Quantitation of murine and human hematopoietic stem cells by limiting-dilution analysis in competitively repopulated hosts. In: Hematopoietic Stem Cell Protocols. Jordon CT, Klug CA (Eds), Humana Press, Totowa, NJ, USA (2001).
95. BHATIA M, BONNET D, MURDOCH B, GAN OI, DICK JE: A newly discovered class of human hematopoietic cells with SCID-repopulating activity. Nat. Med. (1998) 4:1038-1045.
96. + cells. Exp. Hematol. (1998) 26:353-360.
97. PELED A, PETIT I, KOLLET O et al.: Dependence of human stem cell engraftment and repopulation of NOD/SCID mice on CXCR4. Science (1999) 283:845-848.
98. + cells in non-obese diabetic/severe combined immune-deficient mice. Blood (1998) 92:2641-2649.
99. HABIBIAN HK, PETERS SO, HSIEH CC et al.: The fluctuating phenotype of the lymphohematopoietic stem cell with cell cycle transit. J. Exp. Med. (1998) 188:393-398.
100. GLIMM H, OH I-H, EAVES C: Human hematopoietic stem cells stimulated to proliferate in vitro lose engraftment potential during their S/G2/M transit and do not reenter G0. Blood (2000) 96:4185-4193.
101. LAMBERT J-F, LIU M, COLVIN GA et al.: Marrow stem cells shift gene expression and engraftment phenotype with cell cycle transit. J. Exp. Med. (2003) 197(11):1563-1572.
102. GLIMM H, TANG P, CLARK-LEWIS I, VON KALLE C, EAVES C: Ex vivo treatment of proliferating human cord blood stem cells with stroma-derived factor-1 enhances their ability to engraft NOD/SCID mice. Blood (2002) 99(9):3454-3457.
103. MADLAMBAYAN GJ, BAKSH D, ZANDSTRA PW: A systematic approach to the development of stem cell expansion cultures. In: Adult & Fetal Stem Cells. Lanza R (Ed.), Elsevier Science, San Diego, USA (In Press).
104. BOX GEP, DRAPER NR: Empirical Model-Building and Response Surfaces. Box GEP, Draper NR (Eds), Wiley & Sons, New York, USA (1987):669.
105. AUDET J, MILLER CL, EAVES CJ, PIRET JM: Common and distinct features of cytokine effects on hematopoietic stem and progenitor cells revealed by dose-response surface analysis. Biotechnol. Bioeng. (2002) 80:393-404.
106. KITANO H: Systems biology: a brief overview. Science (2002) 295(5560):1662-1664.
107. UNWIN RD, GASKELL SJ, EVANS CA, WHETTON AD: The potential for proteomic definition of stem cell populations. Exp. Hematol. (2003) 31(12):1147-1159.
108. PARK IK, HE Y, LIN F et al.: Differential gene expression profiling of adult murine hematopoietic stem cells. Blood (2002) 99(2):488-498. Demonstrates the value of a systems-level approach in stem cell biology.
109. PRUDHOMME W, DALEY GQ, ZANDSTRA PW, LAUFFENBURGER DA: Multivariate proteomic analysis of murine embryonic stem cell self-renewal versus differentiation behavior. Proc. Natl. Acad. Sci. USA (2004) 101(9):2900-2905. Innovative multivariate and systems-level approaches applied to the study of stem cell differentiation.
110. SCHAFFER DV: Genetic approaches to tissue repair. Ann. NY Acad. Sci. (2002) 961:68-70.
111. PARK KI, OUREDNIK J, OUREDNIK V et al.: Global gene and cell replacement strategies via stem cells. Gene Ther. (2002) 9(10):613-624.
112. SNYDER EY, TAYLOR RM, WOLFE JH: Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature (1995) 374(6520):367-370.
113. FLAX JD, AURORA S, YANG C et al.: Engraftable human neural stem cells respond to developmental cues, replace neurons, and express foreign genes. Nat. Biotechnol. (1998) 16(11):1033-1039.
114. AKERUD P, CANALS JM, SNYDER EY, ARENAS E: Neuroprotection through delivery of glial cell line-derived neurotrophic factor by neural stem cells in a mouse model of Parkinson's disease. J. Neurosci. (2001) 21(20):8108-8118.
115. BURT RK, TRAYNOR A: Hematopoietic stem cell therapy of autoimmune diseases. Curr. Opin. Hematol. (1998) 5:472-477.
116. NICOLINI FE, IMREN S, OH I-H et al.: Expression of a human beta-globin transgene in erythroid cells derived from retrovirally transduced transplantable human fetal liver and cord blood cells. Blood (2002) 100(4):1257-1264.
117. CAVAZZANA-CALVO M, HACEIN-BEY S, DE SAINT BASILE G et al.: Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science (2000) 288(5466):669-672. Human gene therapy trial.
118. ABONOUR R, WILLIAMS DA, EINHORN L et al.: Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells. Nat. Med. (2000) 6(6):652-658.
119. CHALANDON Y, JIANG X, HAZLEWOOD G et al.: Modulation of p210(BCR-ABL) activity in transduced primary human hematopoietic cells controls lineage programming. Blood (2002) 99(9):3197-3204.
120. IMREN S, PAYEN E, WESTERMAN KA et al.: Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc. Natl. Acad. Sci. USA (2002) 99(22):14380-14385.
121. PAWLIUK R, WESTERMAN KA, FABRY ME et al.: Correction of sickle cell disease in transgenic mouse models by gene therapy. Science (2001) 294(5550):2368-2371.
122. MAY C, RIVELLA S, CALLEGARI J et al.: Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature (2000) 406(6791):82-86.
123. GALIMI F, NOLL M, KANAZAWA Y et al.: Gene therapy of Fanconi anemia: preclinical efficacy using lentiviral vectors. Blood (2002) 100(8):2732-2736.
124. BANK A: Hematopoietic stem cell gene therapy: selecting only the best. J. Clin. Invest. (2003) 112(10):1478-1480.
125. VOLLWEILER JL, ZIELSKE SP, REESE JS, GERSON SL: Hematopoietic stem cell gene therapy: progress toward therapeutic targets. Bone Marrow Transpl. (2003) 32(1):1-7.
126. JIN L, SIRITANARATKUL N, EMERY DW et al.: Targeted expansion of genetically modified bone marrow cells. Proc. Natl. Acad. Sci. USA (1998) 95(14):8093-8097.
127. RICHARD RE, BLAU CA: Small-molecule-directed Mpl signaling can complement growth factors to selectively expand genetically modified cotd blood cells. Stem Cells (2003) 21(1):71-78.
128. WHITNEY ML, OTTO KG, BLAU CA, REINECKE H, MURRY CE: Control of myoblast proliferation with a synthetic ligand. J. Biol. Chem. (2001) 276(44):41191-41196.
129. HACEIN-BEY-ABINA S, LE DEIST F, CARLIER F et al.: Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N. Engl. J. Med. (2002) 346(16):1185-1193.
130. HACEIN-BEY-ABINA S, VON KALLE C, SCHMIDT M et al.: LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science (2003) 302(5644):415-419. Unfortunate outcome of a human gene therapy trial.
131. LOU J, XU F, MERKEL K, MANSKE P: Gene therapy: adenovirus-mediated human bone morphogenetic protein-2 gene transfer induces mesenchymal progenitor cell proliferation and differentiation in vitro and bone formation in vivo. J. Orthop. Res. (1999) 17(1):43-50.
132. CHANG SC, CHUANG HL, CHEN YR et al.: Ex vivo gene therapy in autologous bone marrow stromal stem cells for tissue-engineered maxillofacial bone regeneration. Gene Ther. (2003) 10(24):2013-2019.
133. TSUCHIDA H, HASHIMOTO J, CRAWFORD E, MANSKE P, LOU J: Engineered allogeneic mesenchymal stem cells repair femoral segmental defect in rats. J. Orthop. Res. (2003) 21(1):44-53.
134. MOUTSATSOS IK, TURGEMAN G, ZHOU S et al.: Exogenously regulated stem cell-mediated gene therapy for bone regeneration. Mol. Ther. (2001) 3(4):449-461.
135. BAXTER MA, WYNN RF, DEAKIN JA et al.: Retrovirally mediated correction of bone marrow-derived mesenchymal stem cells from patients with mucopolysaccharidosis type I. Blood (2002) 99(5):1857-1859.
136. SHERLEY JL: Asymmetric cell kinetics genes: the key to expansion of adult stem cells in culture. Stem Cells (2002) 20(6):561-572.
137. FAWELL S, SEERY J, DARCH Y et al.: Tat-mediated delivery of heterologous proteins into cells. Proc. Natl. Acad. Sci. USA (1994) 91(2):664-668.
138. WADIA JS, DOWDY SF: Protein transduction technology. Curr. Opin. Biotech. (2002) 13(1):52-56.
139. KROSL J, AUSTIN P, BESLU N, KROON E, HUMPHRIES RK, SAUVAGEAU G: In vitro expansion of hematopoietic stem cells by recombinant TAT-HOXB4 protein. Nat. Med. (2003) 9(11):1428-1432. Innovative and useful approach to increase HSC expansion in vitro.
140. ANTONCHUK J, SAUVAGEAU G, HUMPHRIES RK: HOXB4-induced expansion of adult hematopoietic stem cells ex vivo. Cell (2002) 109(1):39-45.
141. COLLINS PC, MILLER WM, PAPOUTSAKIS ET: Stirred culture of peripheral and cord blood hematopoietic cells offers advantages over traditional static systems for clinically relevant applications. Biotechnol. Bioeng (1998) 59:532-543.
142. KWON J, KIM BS, KIM MJ, PARK HW: Suspension culture of hematopoietic stem cells in stirred bioreactors. Biotechnol. Lett. (2003) 25(2):179-182.
143. ZANDSTRA P, PETZER A, EAVES C, PIRET M: Cellular determinants affecting the rate of cytokine depletion in cultures of human hematopoietic cells. Biotechnol. Bioeng. (1997) 54:58-66.
144. KALLOS MS, SEN A, BEHIE LA: Large-scale expansion of mammalian neural stem cells: a review. Med. Biol. Eng. Comput. (2003) 41(3):271-282.
145. KALLOS MS, BEHIE LA: Inoculation and growth conditions for high-cell-density expansion of mammalian neural stem cells in suspension bioreactors. Biotechnol. Bioeng. (1999) 63(4):473-483. Elegant application of engineering for the development of a scaled-up NSC culture system.
146. SEN A, KALLOS MS, BEHIE LA: Expansion of mammalian neural stem cells in bioreactors: effect of power input and medium viscosity. Dev. Brain Res. (2002) 134(1-2):103-113.
147. SEN A, KALLOS MS, BEHIE LA: Passaging protocols for mammalian neural stem cells in suspension bioteactors. Biotechnol. Prog. (2002) 18(2):337-345.
148. BOUDREAULT P, TREMBLAY JP, PEPIN MF, GARNIER A: Scale-up of a myoblast culture process. J. Biotechnol. (2001) 91(1):63-74.
149. ZANDSTRA PW, BAUWENS C, YIN T et al.: Scalable production of embryonic stem cell-derived cardiomyocytes. Tissue Eng. (2003) 9(4):767-778.
150. DANG SM, KYBA M, PERLINGEIRO R, DALEY GQ, ZANDSTRA PW: Efficiency of embryoid body formation and hematopoietic development from embryonic stem cells in different culture systems. Biotechnol. Bioeng. (2002) 78(4):442-453. Demonstrates the impact of culture system design on stem cell differentiation and lineage selection.
151. CABRITA GJ, FERREIRA BS, DA SILVA CL, GONCALVES R, ALMEIDA-PORADA G, CABRAL JM: Hematopoietic stem cells: from the bone to the bioreactor. Trends Biotechnol. (2003) 21(5):233-240.
152. NIELSEN LK: Bioreactors for hematopoietic cell culture. Ann. Pev. Biomed. Eng. (1999) 1:129-152.
153. LUNDELL BI, VREDENBURGH JJ, TYER C, DESOMBRE K, SMITH AK: Ex vivo expansion of bone marrow from breast cancer patients: reduction in tumor cell content through passive purging. Bone Marrow Transpl. (1998) 22:153-159.
154. UDOMSAKDI C, EAVES CJ, SWOLIN B, REID DS, BARNETT MJ, EAVES AC: Rapid decline of chronic myeloid leukemic cells in long-term culture due to a defect at the leukemic stem cell level. Proc. Natl. Acad. Sci. USA (1992) 89:6192-6196.
155. EAVES AC, EAVES CJ, PHILLIPS GL, BARNETT MJ: Culture purging in leukemia: past, present, and future. Leuk. Lymphoma. (1993) 11(Suppl. 1):259-263.
156. CHRIST B, EAVES C, FORREST D et al.: Autografting with cultured marrow in chronic myeloid leukemia (CML): Long-term follow-up of the Vancouver experience. Blood (2003) 102:740a.
157. STIFF P, CHEN B, FRANKLIN W et al.: Autologous transplantation of ex vivo expanded bone marrow cells grown from small aliquots after high-dose chemotherapy for breast cancer. Blood (2000) 95:2169-2174.
158. JAROSCAK J, GOLTRY K, SMITH A et al.: Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a Phase I trial using the Aastrom Replicell System. Blood (2003) 101(12):5061-5067. Results of a clinical trial on the transplantation of haematopoietic cells cultured in a perfusion bioreactor.
159. STEWART AK, SUTHERLAND DR, NANJI S et al.: Engraftment of gene-marked hematopoietic progenitors in myeloma patients after transplant of autologous long-term marrow cultures. Hum. Gene Ther. (1999) 10:1953-1964. One of the rare human studies in which the progeny of cultured haematopoietic cells was tracked in vivo following transplantation.
160. VISWANATHAN S, ZANDSTRA PW: Towards predictive models of stem cell fate. Cytotechnology (2003) 41:75-92.
161. DEASY BM, JANKOWSKI RJ, PAYNE TR et al.: Modeling stem cell population growth: Incorporating terms for proliferative heterogeneity. Stem Cells (2003) 21(5):536-545.
162. DEASY BM, QU-PETERSON Z, GREENBERGER JS, HUARD J: Mechanisms of muscle stem cell expansion with cytokines. Stem Cells (2002) 20(1):50-60.
163. YANG H, PAPOUTSAKIS ET, MILLER MW: Model-based estimation of myeloid hematopoietic progenitor cells in ex vivo cultures for cell and gene therapies. Biotechnol. Bioeng. (2001) 72:144-155.
164. TERADA N, HAMAZAKI T, OKA M et al.: Bone marrow cells adopt the phenotype of other cells by spontaneous cell fusion. Nature (2002) 416(6880):542-545.
165. ALVAREZ-DOLADO M, PARDAL R, GARCIA-VERDUGO JM et al.: Fusion of bone-marrow-derived cells with Purkinje neurons, cardiomyocytes and hepatocytes. Nature (2003) 425(6961):968-973.
166. NAGAI Y, MIYAZAKI M, AOKI R et al.: A fluorescent indicator for visualizing cAMP-induced phosphorylation in vivo. Nat. Biotechnol. (2000) 18:313-316.
167. ZACCOLO M, DE GIORGI F, CHO CY et al.: A genetically encoded, fluorescent indicator for cyclic AMP in living cells. Nat. Cell Biol. (2000) 2:25-29.
168. MEREDITH GD, SIMS CE, SOUGHAYER JS, ALLBRITTON NL: Measutement of kinase activation in single mammalian cells. Nat. Biotechnol. (2000) 18:309-312. Novel technology to measure the activity of multiple kinases in single cells.
169. ZARRINE-AFSAR A, KRYLOV SN: Use of capillary electrophoresis and endogenous fluorescent substrate to monitor intracellular activation of protein kinase A. Anal. Chem. (2003) 75(15):3720-3724.
170. TERUEL MN, MEYER T: Parallel single-cell monitoring of receptor-triggered membrane translocation of a calcium-sensing protein module. Science (2002) 295(5561):1910-1912. High-throughput, temporal and spatial analysis of signal transduction in single cells.
171. LI H, SIMS CE, KALUZOVA M, STANBRIDGE EJ, ALLBRITTON NL: A quantitative single-cell assay for protein kinase B reveals important insights into the biochemical behavior of an intracellular substrate peptide. Biochemistry (2004) 43(6):1599-1608.
172. AUDET J, SOUGHAYER JS, SIMS CE, ONG ST, ALLBRITTON NL: Quantitative measurements of growth-promoting signals in single chronic myeloid leukemia cells (abstract). Blood (2003) 102:420a.
173. PEREZ OD, NOLAN GP: Simultaneous measurement of multiple active kinase states using polychromatic flow cytometry. Nat. Biotechnol. (2002) 20:155-162. Demonstrates the value of flow cytometry to simultaneously measure the activation state of multiple kinases.