Stem cell-mediated gene delivering for the treatment of cerebral ischemia: Progress and prospectives

Current Drug Targets

Volumn 14, Issue 1, 2013, Pages 81-89

  Chen, Chao ^a,b   Wang, Yongting ^a,b   Yang, Guo Yuan ^a,b  

^a SHANGHAI JIAO TONG UNIVERSITY   (China)

^b SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE   (China)

Author keywords

Embryonic stem cell; Gene therapy; Ischemic stroke; Mesenchymal stem cell; Neural stem cell; Stem cell

Indexed keywords

BRAIN DERIVED NEUROTROPHIC FACTOR; CHEMOKINE RECEPTOR CXCR4; CILIARY NEUROTROPHIC FACTOR; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; NETRIN 1; NEUROTROPHIN 3; PLACENTAL GROWTH FACTOR; PROTEIN BCL 2; VASCULOTROPIN;

ANGIOGENESIS; APOPTOSIS; BRAIN ISCHEMIA; CELL SURVIVAL; EMBRYONIC STEM CELL; GENE EXPRESSION; GENE TRANSFER; HUMAN; MESENCHYMAL STEM CELL; NERVOUS SYSTEM DEVELOPMENT; NEURAL STEM CELL; NEUROPROTECTION; NONHUMAN; PLURIPOTENT STEM CELL; REVIEW; STEM CELL GENE THERAPY; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

EID: 84876719872     PISSN: 13894501     EISSN: 18735592     Source Type: Journal    
DOI: 10.2174/138945013804806497     Document Type: Review

References (77)

1. Strong K, Mathers C, Bonita R. Preventing stroke: saving lives around the world. Lancet Neurol 2007; 6(2): 182-7.
2. Lees KR, Bluhmki E, von Kummer R, et al. Time to treatment with intravenous alteplase and outcome in stroke: an updated pooled analysis of ECASS, ATLANTIS, NINDS, and EPITHET trials. Lancet 2010; 375(9727): 1695-703.
3. Tissue plasminogen activator for acute ischemic stroke. The National Institute of Neurological Disorders and Stroke rt-PA Stroke Study Group. N Engl J Med 1995; 333(24): 1581-7.
4. Both G, Alexander I, Fletcher S, et al. Gene therapy: therapeutic applications and relevance to pathology. Pathology 2011; 43(6): 642-56.
5. Aiuti A, Cattaneo F, Galimberti S, et al. Gene therapy for immuno deficiency due to adenosine deaminase deficiency. N Engl J Med 2009; 360(5): 447-58.
6. Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. 20 years of gene therapy for SCID. Nat Immunol 2010; 11(6): 457-60.
7. Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in Xlinked adrenoleukodystrophy. Science 2009; 326(5954): 818-23.
8. Fanning G, Amado R, Symonds G. Gene therapy for HIV/AIDS: the potential for a new therapeutic regimen. J Gene Med 2003; 5(8): 645-53.
9. Macpherson JL, Boyd MP, Arndt AJ, et al. Long-term survival and concomitant gene expression of ribozyme-transduced CD4+ Tlymphocytes in HIV-infected patients. J Gene Med 2005; 7(5): 552-64.
10. Freytag SO, Stricker H, Peabody J, et al. Five-year follow-up of trial of replication-competent adenovirus-mediated suicide gene therapy for treatment of prostate cancer. Mol Ther 2007; 15(3): 636-42.
11. Zhao H, Yenari MA, Cheng D, Sapolsky RM, Steinberg GK. Bcl-2 overexpression protects against neuron loss within the ischemic margin following experimental stroke and inhibits cytochrome c translocation and caspase-3 activity. J Neurochem 2003; 85(4): 1026-36.
12. Yamashita T, Deguchi K, Nagotani S, Kamiya T, Abe K. Gene and stem cell therapy in ischemic stroke. Cell Transplant 2009; 18(9): 999-1002.
13. Shi Q, Zhang P, Zhang J, et al. Adenovirus-mediated brain-derived neurotrophic factor expression regulated by hypoxia response element protects brain from injury of transient middle cerebral artery occlusion in mice. Neurosci Lett 2009; 465(3): 220-5.
14. Jin G, Inoue M, Hayashi T, et al. Sendai virus-mediated gene transfer of GDNF reduces AIF translocation and ameliorates ischemic cerebral injury. Neurol Res 2008; 30(7): 731-9.
15. Lim KI. Retroviral integration profiles: their determinants and implications for gene therapy. BMB Rep 2012; 45(4): 207-12.
16. Wu X, Burgess SM. Integration target site selection for retroviruses and transposable elements. Cell Mol Life Sci 2004; 61(19-20): 2588-96.
17. Zhu JM, Zhao YY, Chen SD, et al. Functional recovery after transplantation of neural stem cells modified by brain-derived neurotrophic factor in rats with cerebral ischaemia. J Int Med Res 2011; 39(2): 488-98.
18. Kurozumi K, Nakamura K, Tamiya T, et al. BDNF gene-modified mesenchymal stem cells promote functional recovery and reduce infarct size in the rat middle cerebral artery occlusion model. Mol Ther 2004; 9(2): 189-97.
19. Zhang ZH, Wang RZ, Li GL, et al. Transplantation of neural stem cells modified by human neurotrophin-3 promotes functional recovery after transient focal cerebral ischemia in rats. Neurosci Lett 2008; 444(3): 227-30.
20. Kurozumi K, Nakamura K, Tamiya T, et al. Mesenchymal stem cells that produce neurotrophic factors reduce ischemic damage in the rat middle cerebral artery occlusion model. Mol Ther 2005; 11(1): 96-104.
21. Borlongan CV, Glover LE, Tajiri N, Kaneko Y, Freeman TB. The great migration of bone marrow-derived stem cells toward the ischemic brain: therapeutic implications for stroke and other neurological disorders. Prog Neurobiol 2011; 95(2): 213-28.
22. Fan Y, Shen F, Frenzel T, et al. Endothelial progenitor cell transplantation improves long-term stroke outcome in mice. Ann Neurol 2010; 67(4): 488-97.
23. Chen C, Xiao SF. Induced pluripotent stem cells and neurodegenerative diseases. Neurosci Bull 2011; 27(2): 107-14.
24. Chen J, Li Y, Wang L, et al. Therapeutic benefit of intracerebral transplantation of bone marrow stromal cells after cerebral ischemia in rats. J Neurol Sci 2001; 189(1-2): 49-57.
25. Burns TC, Steinberg GK. Stem cells and stroke: opportunities, challenges and strategies. Expert Opin Biol Ther 2011; 11(4): 447-61.
26. Honmou O, Houkin K, Matsunaga T, et al. Intravenous adminis tration of auto serum-expanded autologous mesenchymal stem cells in stroke. Brain 2011; 134(Pt 6): 1790-807.
27. Lee JS, Hong JM, Moon GJ, et al. A long-term follow-up study of intravenous autologous mesenchymal stem cell transplantation in patients with ischemic stroke. Stem Cells 2010; 28(6): 1099-106.
28. Naldini L. Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet 2011; 12(5): 301-15.
29. Czabotar PE, Lessene G. Bcl-2 family proteins as therapeutic targets. Curr Pharm Des 2010; 16(28): 3132-48.
30. Chu K, Kim M, Park KI, et al. Human neural stem cells improve sensorimotor deficits in the adult rat brain with experimental focal ischemia. Brain Res 2004; 1016(2): 145-53.
31. Gopurappilly R, Pal R, Mamidi MK, et al. Stem cells in stroke repair: current success & future prospects. CNS Neurol Disord Drug Targets 2011; 10(6): 741-56.
32. Nakaguchi K, Masuda H, Kaneko N, Sawamoto K. Strategies for regenerating striatal neurons in the adult brain by using endogenous neural stem cells. Neurol Res Int 2011; 2011: 898012.
33. Hwang DH, Jeong SR, Kim BG. Gene transfer mediated by stem cell grafts to treat CNS injury. Expert Opin Biol Ther 2011; 11(12): 1599-610.
34. Lee HJ, Kim KS, Park IH, Kim SU. Human neural stem cells overexpressing VEGF provide neuroprotection, angiogenesis and functional recovery in mouse stroke model. PLoS One 2007; 2(1): e156.
35. Lian Jin H, Pennant WA, Hyung Lee M, et al. Neural stem cells modified by a hypoxia-inducible VEGF gene expression system improve cell viability under hypoxic conditions and spinal cord injury. Spine (Phila Pa 1976) 2011; 36(11): 857-64.
36. Choi YJ, Li WY, Moon GJ, et al. Enhancing trophic support of mesenchymal stem cells by ex vivo treatment with trophic factors. J Neurol Sci 2010; 298(1-2): 28-34.
37. Fumagalli F, Molteni R, Calabrese F, et al. Neurotrophic factors in neurodegenerative disorders: potential for therapy. CNS Drugs 2008; 22(12): 1005-19.
38. Blesch A. Neurotrophic factors in neurodegeneration. Brain Pathol 2006; 16(4): 295-303.
39. Miki Y, Nonoguchi N, Ikeda N, et al. Vascular endothelial growth factor gene-transferred bone marrow stromal cells engineered with a herpes simplex virus type 1 vector can improve neurological deficits and reduce infarction volume in rat brain ischemia. Neurosurgery 2007; 61(3): 586-94; discussion 594-5.
40. Zhao H, Bao XJ, Wang RZ, et al. Postacute ischemia vascular endothelial growth factor transfer by transferrin-targeted liposomes attenuates ischemic brain injury after experimental stroke in rats. Hum Gene Ther 2011; 22(2): 207-15.
41. Toyama K, Honmou O, Harada K, et al. Therapeutic benefits of angiogenetic gene-modified human mesenchymal stem cells after cerebral ischemia. Exp Neurol 2009; 216(1): 47-55.
42. Shin HY, Lee YJ, Kim HJ, et al. Protective role of COMP-Ang1 in ischemic rat brain. J Neurosci Res 2010; 88(5): 1052-63.
43. Onda T, Honmou O, Harada K, et al. Therapeutic benefits by human mesenchymal stem cells (hMSCs) and Ang-1 genemodified hMSCs after cerebral ischemia. J Cereb Blood Flow Metab 2008; 28(2): 329-40.
44. Zhu W, Fan Y, Hao Q, et al. Postischemic IGF-1 gene transfer promotes neurovascular regeneration after experimental stroke. J Cereb Blood Flow Metab 2009; 29(9): 1528-37.
45. Liu H, Honmou O, Harada K, et al. Neuroprotection by PlGF genemodified human mesenchymal stem cells after cerebral ischaemia. Brain 2006; 129(Pt 10): 2734-45.
46. Shimamura M, Sato N, Oshima K, et al. Novel therapeutic strategy to treat brain ischemia: overexpression of hepatocyte growth factor gene reduced ischemic injury without cerebral edema in rat model. Circulation 2004; 109(3): 424-31.
47. Zhao MZ, Nonoguchi N, Ikeda N, et al. Novel therapeutic strategy for stroke in rats by bone marrow stromal cells and ex vivo HGF gene transfer with HSV-1 vector. J Cereb Blood Flow Metab 2006; 26(9): 1176-88.
48. Wang YZ, Plane JM, Jiang P, Zhou CJ, Deng W. Concise review: Quiescent and active states of endogenous adult neural stem cells: identification and characterization. Stem Cells 2011; 29(6): 907-12.
49. Barkho BZ, Zhao X. Adult neural stem cells: response to stroke injury and potential for therapeutic applications. Curr Stem Cell Res Ther 2011; 6(4): 327-38.
50. Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006; 126(4): 663-76.
51. Takahashi K, Tanabe K, Ohnuki M, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007; 131(5): 861-72.
52. Oki K, Tatarishvili J, Wood J, et al. Human-induced pluripotent stem cells form functional neurons and improve recovery after grafting in stroke-damaged brain. Stem Cells 2012; 30(6): 1120-33.
53. Jensen MB, Yan H, Krishnaney-Davison R, Al Sawaf A, Zhang SC. Survival and Differentiation of Transplanted Neural Stem Cells Derived from Human Induced Pluripotent Stem Cells in A Rat Stroke Model. J Stroke Cerebrovasc Dis 2011.
54. Jiang M, Lv L, Ji H, et al. Induction of pluripotent stem cells transplantation therapy for ischemic stroke. Mol Cell Biochem 2011; 354(1-2): 67-75.
55. Kawai H, Yamashita T, Ohta Y, et al. Tridermal tumorigenesis of induced pluripotent stem cells transplanted in ischemic brain. J Cereb Blood Flow Metab 2010; 30(8): 1487-93.
56. Chen SJ, Chang CM, Tsai SK, et al. Functional improvement of focal cerebral ischemia injury by subdural transplantation of induced pluripotent stem cells with fibrin glue. Stem Cells Dev 2010; 19(11): 1757-67.
57. Lin YC, Ko TL, Shih YH, et al. Human umbilical mesenchymal stem cells promote recovery after ischemic stroke. Stroke 2011; 42(7): 2045-53.
58. Yu X, Chen D, Zhang Y, et al. Overexpression of CXCR4 in mesenchymal stem cells promotes migration, neuroprotection and angiogenesis in a rat model of stroke. J Neurol Sci 2012; 316(1-2): 141-9.
59. Tapuria N, Kumar Y, Habib MM, et al. Remote ischemic preconditioning: a novel protective method from ischemia reperfusion injury--a review. J Surg Res 2008; 150(2): 304-30.
60. Font MA, Arboix A, Krupinski J. Angiogenesis, neurogenesis and neuroplasticity in ischemic stroke. Curr Cardiol Rev 2010; 6(3): 238-44.
61. Shirakura M, Inoue M, Fujikawa S, et al. Postischemic adminis tration of Sendai virus vector carrying neurotrophic factor genes prevents delayed neuronal death in gerbils. Gene Ther 2004; 11(9): 784-90.
62. Gao P, Shen F, Gabriel RA, et al. Attenuation of brain response to vascular endothelial growth factor-mediated angiogenesis and neurogenesis in aged mice. Stroke 2009; 40(11): 3596-600.
63. Shen F, Fan Y, Su H, et al. Adeno-associated viral vector-mediated hypoxia-regulated VEGF gene transfer promotes angiogenesis following focal cerebral ischemia in mice. Gene Ther 2008; 15(1): 30-9.
64. Zhang ZG, Zhang L, Jiang Q, et al. VEGF enhances angiogenesis and promotes blood-brain barrier leakage in the ischemic brain. J Clin Invest 2000; 106(7): 829-38.
65. Li Z, Wang R, Li S, et al. Intraventricular pre-treatment with rAAV-VEGF induces intracranial hypertension and aggravates ischemic injury at the early stage of transient focal cerebral ischemia in rats. Neurol Res 2008; 30(8): 868-75.
66. Sugiura S, Kitagawa K, Tanaka S, et al. Adenovirus-mediated gene transfer of heparin-binding epidermal growth factor-like growth factor enhances neurogenesis and angiogenesis after focal cerebral ischemia in rats. Stroke 2005; 36(4): 859-64.
67. Leker RR, Soldner F, Velasco I, et al. Long-lasting regeneration after ischemia in the cerebral cortex. Stroke 2007; 38(1): 153-61.
68. Sun H, Le T, Chang TT, et al. AAV-mediated netrin-1 over expression increases peri-infarct blood vessel density and improves motor function recovery after experimental stroke. Neurobiol Dis 2011; 44(1): 73-83.
69. Zhu W, Fan Y, Frenzel T, et al. Insulin growth factor-1 gene transfer enhances neurovascular remodeling and improves longterm stroke outcome in mice. Stroke 2008; 39(4): 1254-61.
70. McColl BW, McGregor AL, Wong A, et al. APOE epsilon3 gene transfer attenuates brain damage after experimental stroke. J Cereb Blood Flow Metab 2007; 27(3): 477-87.
71. Zhang WR, Sato K, Iwai M, et al. Therapeutic time window of adenovirus-mediated GDNF gene transfer after transient middle cerebral artery occlusion in rat. Brain Res 2002; 947(1): 140-5.
72. Li N, Kong X, Ye R, et al. Age-related differences in experimental stroke: possible involvement of mitochondrial dysfunction and oxidative damage. Rejuvenation Res 2011; 14(3): 261-73.
73. Howe SJ, Mansour MR, Schwarzwaelder K, et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 2008; 118(9): 3143-50.
74. Hacein-Bey-Abina S, Hauer J, Lim A, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2010; 363(4): 355-64.
75. Bilic J, Izpisua Belmonte JC. Concise review: Induced pluripotent stem cells versus embryonic stem cells: close enough or yet too far apart? Stem Cells 2012; 30(1): 33-41.
76. Wernig M, Zhao JP, Pruszak J, et al. Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease. Proc Natl Acad Sci USA 2008; 105(15): 5856-61.
77. Bang OY, Lee JS, Lee PH, Lee G. Autologous mesenchymal stem cell transplantation in stroke patients. Ann Neurol 2005; 57(6): 874-82.