Translational strategies and challenges in regenerative medicine

Nature Medicine

Volumn 20, Issue 8, 2014, Pages 814-821

  Dimmeler, Stefanie ^a   Ding, Sheng ^b   Rando, Thomas A ^c,d   Trounson, Alan ^e  

^a GOETHE UNIVERSITY   (Germany)

^b GLADSTONE INSTITUTE OF CARDIOVASCULAR DISEASE   (United States)

^c STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d STANFORD UNIVERSITY   (United States)

^e California Institute for Regenerative Medicine (CIRM)   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADULT STEM CELLS; CELL- AND TISSUE-BASED THERAPY; GUIDED TISSUE REGENERATION; HEMATOPOIETIC STEM CELLS; HUMANS; MESENCHYMAL STROMAL CELLS; REGENERATIVE MEDICINE; STEM CELL NICHE; STEM CELL RESEARCH; STEM CELL TRANSPLANTATION; TISSUE ENGINEERING; WOUND HEALING;

EID: 84905739577     PISSN: 10788956     EISSN: 1546170X     Source Type: Journal    
DOI: 10.1038/nm.3627     Document Type: Review

References (78)

1. Schofield, R. The relationship between the spleen colony-forming cell and the haemopoietic stem cell. Blood Cells 4, 7-25 (1978). (Pubitemid 8376317)
2. Daley, G.Q. The promise and perils of stem cell therapeutics. Cell Stem Cell 10, 740-749 (2012).
3. Conboy, I.M., Conboy, M.J., Smythe, G.M. & Rando, T.A. Notch-mediated restoration of regenerative potential to aged muscle. Science 302, 1575-1577 (2003). (Pubitemid 37475703)
4. van Praag, H., Kempermann, G. & Gage, F.H. Running increases cell proliferation and neurogenesis in the adult mouse dentate gyrus. Nat. Neurosci. 2, 266-270 (1999). (Pubitemid 30491298)
5. Trejo, J.L., Carro, E. & Torres-Aleman, I. Circulating insulin-like growth factor I mediates exercise-induced increases in the number of new neurons in the adult hippocampus. J. Neurosci. 21, 1628-1634 (2001). (Pubitemid 32182203)
6. Fabel, K. et al. VEGF is necessary for exercise-induced adult hippocampal neurogenesis. Eur. J. Neurosci. 18, 2803-2812 (2003). (Pubitemid 37542133)
7. Li, W., Li, K., Wei, W. & Ding, S. Chemical approaches to stem cell biology and therapeutics. Cell Stem Cell 13, 270-283 (2013).
8. Sato, T. & Clevers, H. Growing self-organizing mini-guts from a single intestinal stem cell: Mechanism and applications. Science 340, 1190-1194 (2013).
9. Watt, F.M. & Hogan, B.L. Out of Eden: Stem cells and their niches. Science 287, 1427-1430 (2000). (Pubitemid 30127126)
10. Watt, F.M. & Huck, W.T. Role of the extracellular matrix in regulating stem cell fate. Nat. Rev. Mol. Cell Biol. 14, 467-473 (2013).
11. Calvi, L.M. et al. Osteoblastic cells regulate the haematopoietic stem cell niche. Nature 425, 841-846 (2003). (Pubitemid 37351468)
12. Friedman, S.L., Sheppard, D., Duffield, J.S. & Violette, S. Therapy for fibrotic diseases: Nearing the starting line. Sci. Transl. Med. 5, 167-1 (2013).
13. Engler, A.J., Sen, S., Sweeney, H.L. & Discher, D.E. Matrix elasticity directs stem cell lineage specification. Cell 126, 677-689 (2006). (Pubitemid 44233625)
14. Huang, N.F. & Li, S. Regulation of the matrix microenvironment for stem cell engineering and regenerative medicine. Ann. Biomed. Eng. 39, 1201-1214 (2011).
15. Turner, N.J. & Badylak, S.F. Biologic scaffolds for musculotendinous tissue repair. Eur. Cell. Mater. 25, 130-143 (2013).
16. Wong, V.W., Levi, B., Rajadas, J., Longaker, M.T. & Gurtner, G.C. Stem cell niches for skin regeneration. Int. J. Biomater. 2012, 926059 (2012).
17. Peerani, R. & Zandstra, P.W. Enabling stem cell therapies through synthetic stem cell-niche engineering. J. Clin. Invest. 120, 60-70 (2010).
18. Gómez-Gaviro, M.V., Lovell-Badge, R., Fernandez-Aviles, F. & Lara-Pezzi, E. The vascular stem cell niche. J. Cardiovasc. Transl. Res. 5, 618-630 (2012).
19. Palmer, T.D., Willhoite, A.R. & Gage, F.H. Vascular niche for adult hippocampal neurogenesis. J. Comp. Neurol. 425, 479-494 (2000).
20. Ma, J. et al. Concise review: Cell-based strategies in bone tissue engineering and regenerative medicine. Stem Cells Transl. Med. 3, 98-107 (2014).
21. Suda, T., Takubo, K. & Semenza, G.L. Metabolic regulation of hematopoietic stem cells in the hypoxic niche. Cell Stem Cell 9, 298-310 (2011).
22. Titmarsh, D.M., Chen, H., Glass, N.R. & Cooper-White, J.J. Concise review: Microfluidic technology platforms: Poised to accelerate development and translation of stem cell-derived therapies. Stem Cells Transl. Med. 3, 81-90 (2014).
23. Conboy, I.M. et al. Rejuvenation of aged progenitor cells by exposure to a young systemic environment. Nature 433, 760-764 (2005). (Pubitemid 40292986)
24. Villeda, S.A. et al. The ageing systemic milieu negatively regulates neurogenesis and cognitive function. Nature 477, 90-94 (2011).
25. Villeda, S.A. et al. Young blood reverses age-related impairments in cognitive function and synaptic plasticity in mice. Nat. Med. 20, 659-663 (2014).
26. Katsimpardi, L. et al. Vascular and neurogenic rejuvenation of the aging mouse brain by young systemic factors. Science 344, 630-634 (2014).
27. Elabd, C. et al. Oxytocin is an age-specific circulating hormone that is necessary for muscle maintenance and regeneration. Nat. Commun. 5, 4082 (2014).
28. Maus, M.V. et al. Adoptive immunotherapy for cancer or viruses. Annu. Rev. Immunol. 32, 189-225 (2014).
29. Tebas, P. et al. N. Engl. J. Med. 370, 901-910 (2014).
30. Cutler, C. et al. Prostaglandin-modulated umbilical cord blood hematopoietic stem cell transplantation. Blood 122, 3074-3081 (2013).
31. Christopherson, K.W., Hangoc, G., Mantel, C.R. & Broxmeyer, H.E. Modulation of hematopoietic stem cell homing and engraftment by CD26. Science 305, 1000-1003 (2004). (Pubitemid 39071774)
32. Chinnadurai, R., Copland, I.B., Patel, S.R. & Galipeau, J. IDO-independent suppression of T cell effector function by IFN-g-licensed human mesenchymal stromal cells. J. Immunol. 192, 1491-1501 (2014).
33. Seeger, F.H., Zeiher, A.M. & Dimmeler, S. MicroRNAs in stem cell function and regenerative therapy of the heart. Arterioscler. Thromb. Vasc. Biol. 33, 1739-1746 (2013).
34. Liles, W.C. et al. Mobilization of hematopoietic progenitor cells in healthy volunteers by AMD3100, a CXCR4 antagonist. Blood 102, 2728-2730 (2003). (Pubitemid 37248839)
35. Zaruba, M.-M. et al. Synergy between CD26/DPP-IV inhibition and G-CSF improves cardiac function after acute myocardial infarction. Cell Stem Cell 4, 313-323 (2009).
36. Broxmeyer, H.E. et al. Dipeptidylpeptidase 4 negatively regulates colony-stimulating factor activity and stress hematopoiesis. Nat. Med. 18, 1786-1796 (2012).
37. Kragl, M. et al. Cells keep a memory of their tissue origin during axolotl limb regeneration. Nature 460, 60-65 (2009).
38. Efe, J.A. et al. Conversion of mouse fibroblasts into cardiomyocytes using a direct reprogramming strategy. Nat. Cell Biol. 13, 215-222 (2011).
39. Wang H. et al. Small molecules enable cardiac reprogramming of mouse fibroblasts with a single factor, Oct4. Cell Rep. 6, 951-960 (2014).
40. Wang, F. et al. Targeted inhibition of mutant IDH2 in leukemia cells induces cellular differentiation. Science 340, 622-626 (2013).
41. Baker, D.J. et al. Clearance of p16Ink4a-positive senescent cells delays ageing-associated disorders. Nature 479, 232-236 (2011).
42. Zhang, Q. et al. Small-molecule synergist of the Wnt/b-catenin signaling pathway. Proc. Natl. Acad. Sci. USA 104, 7444-7448 (2007). (Pubitemid 47185925)
43. Gu, E., Chen, W.Y., Gu, J., Burridge, P. & Wu, J.C. Molecular imaging of stem cells: Tracking survival, biodistribution, tumorigenicity, and immunogenicity. Theranostics 2, 335-345 (2012).
44. van Laake, L.W., Passier, R., Doevendans, P.A. & Mummery, C.L. Human embryonic stem cell-derived cardiomyocytes and cardiac repair in rodents. Circ. Res. 102, 1008-1010 (2008). (Pubitemid 351667854)
45. Rosado-de-Castro, P.H. et al. Biodistribution of bone marrow mononuclear cells after intra-arterial or intravenous transplantation in subacute stroke patients. Regen. Med. 8, 145-155 (2013).
46. Mohsin, S. et al. Human cardiac progenitor cells engineered with Pim-I kinase enhance myocardial repair. J. Am. Coll. Cardiol. 60, 1278-1287 (2012).
47. Ghadge, S.K., Mühlstedt, S., Ozcelik, C. & Bader, M. SDF-1αas a therapeutic stem cell homing factor in myocardial infarction. Pharmacol. Ther. 129, 97-108 (2011).
48. Frey, N. & Olson, E.N. Cardiac hypertrophy: The good, the bad, and the ugly. Annu. Rev. Physiol. 65, 45-79 (2003). (Pubitemid 38249147)
49. Lee, A.S., Tang, C., Rao, M.S., Weissman, I.L. & Wu, J.C. Tumorigenicity as a clinical hurdle for pluripotent stem cell therapies. Nat. Med. 19, 998-1004 (2013).
50. Severs, N.J., Bruce, A.F., Dupont, E. & Rothery, S. Remodelling of gap junctions and connexin expression in diseased myocardium. Cardiovasc. Res. 80, 9-19 (2008).
51. Li, F. et al. Neuroinflammation and cell therapy for Parkinson's disease. Front. Biosci. (schol. ed.) 3, 1407-1420 (2011).
52. Hu, J. et al. Endothelial cell-derived angiopoietin-2 controls liver regeneration as a spatiotemporal rheostat. Science 343, 416-419 (2014).
53. Ding, B.S. et al. Divergent angiocrine signals from vascular niche balance liver regeneration and fibrosis. Nature 505, 97-102 (2014).
54. Kang, T.W. et al. Senescence surveillance of pre-malignant hepatocytes limits liver cancer development. Nature 479, 547-551 (2011).
55. Katsimpardi, L. et al. Vascular and neurogenic rejuvenation of the aging mouse brain by young systemic factors. Science 344, 630-634 (2014).
56. Segers, V.F. & Lee, R.T. Biomaterials to enhance stem cell function in the heart. Circ. Res. 109, 910-922 (2011).
57. Dimmeler, S. & Leri, A. Aging and disease as modifiers of efficacy of cell therapy. Circ. Res. 102, 1319-1330 (2008).
58. Xu, Q. et al. Micro-RNA-34a contributes to the impaired function of bone marrow-derived mononuclear cells from patients with cardiovascular disease. J. Am. Coll. Cardiol. 59, 2107-2117 (2012).
59. Trounson, A., Baum, E., Gibbons, D. & Tekamp-Olson, P. Developing a model for successful translation of stem cell therapies. Cell Stem Cell 6, 513-516 (2010).
60. Trounson, A. in Stem Cells Handbook, 2nd edn. (ed. Sell, S.) 377-389 (Springer, 2013).
61. Nowbar, A.N. et al. Discrepancies in autologous bone marrow stem cell and enhancement of ejection fraction (DAMASCENE): Weighted regression and meta-analysis. Br. Med. J. 348, 2688 (2014).
62. Figueroa, F.E., Carrión, F., Villanueva, S. & Khoury, M. Mesenchymal stem cell treatment for autoimmune diseases: A critical review. Biol. Res. 45, 269-277 (2012).
63. Ren, G. et al. Concise review: Mesenchymal stem cells and translational medicine: Emerging issues. Stem Cells Transl. Med. 1, 51-58 (2012).
64. California Institute for Regenerative Medicine. Cell therapies for Parkinson's Disease from discovery to clinic. (CIRM, 2013). http://www.cirm.ca. gov/sites/default/files/files/about-cirm/PD-Workshop-WP-10-10-13.pdf.
65. Li, M., Suzuki, K., Kim, N.Y., Liu, G.H. & Izpisua Belmonte, J.C. A cut above the rest: Targeted genome editing technologies in human pluripotent stem cells. J. Biol. Chem. 289, 4594-4599 (2014).
66. Bellin, M., Marchetto, M.C., Gage, F.H. & Mummery, C.L. Induced pluripotent stem cells: The new patient? Nat. Rev. Mol. Cell Biol. 13, 713-726 (2012).
67. Chong, J.J.H. et al. Human embryonic-stem-cell-derived cardiomyocytes regenerate non-human primate hearts. Nature 510, 273-277 (2014).
68. Turner, M. et al. Towards the development of a global induced pluripotent stem cell library. Cell Stem Cell 13, 382-384 (2013).
69. Rong, Z. et al. An effective approach to prevent immune rejection of human ESC-derived allografts. Cell Stem Cell 14, 121-130 (2014).
70. Schulz, T.C. et al. A scalable system for production of functional pancreatic progenitors from human embryonic stem cells. PLoS ONE 7, e37004 (2012).
71. Bara, J.J., Richards, R.G., Alini, M. & Stoddard, M.J. Bone marrow-derived mesenchymal stem cells change phenotype following in vitro culture: Implications for basic research and the clinic. Stem Cells 32, 1713-1723 (2014).
72. Ginsberg, M. et al. Efficient direct reprogramming of mature amniotic cells into endothelial cells by ETS factors and TGFb suppression. Cell 151, 559-575 (2012).
73. Bharti, K. et al. Developing cellular therapies for retinal degenerative diseases. Invest. Ophthalmol. Vis. Sci. 55, 1191-1202 (2014).
74. Zhang, D. et al. Tissue-engineered cardiac patch for advanced functional maturation of human ESC-derived cardiomyocytes. Biomaterials 34, 5813-5820 (2013).
75. Fu, J.-D. et al. Direct reprogramming of human fibroblasts towards a cardiomyocyte-like state. Stem Cell Rep. 1, 235-247 (2013).
76. Ruckh, J.M. et al. Rejuvenation of regeneration in the aging central nervous system. Cell Stem Cell 10, 96-103 (2012).
77. van Deursen, J.M. The role of senescent cells in ageing. Nature 509, 439-446 (2014).
78. David, D.C. Aging and the aggregating proteome. Front. Genet. 3, 247 (2012).