Cell therapy for Parkinson's disease: New hope from reprogramming technologies

Aging and Disease

Volumn 6, Issue 6, 2015, Pages 499-503

  Chen, Zhiguo ^a,b  

^a CAPITAL MEDICAL UNIVERSITY   (China)

^b BEIJING INSTITUTE FOR BRAIN DISORDERS   (China)

Author keywords

Cell therapy; Clinical trials; Dopaminergic neurons; Parkinson's disease; Reprogramming

Indexed keywords

BRN2 PROTEIN; CYCLOSPORIN; LEVODOPA; MYT1L PROTEIN; PEPTIDES AND PROTEINS; TRANSCRIPTION FACTOR MASH1; UNCLASSIFIED DRUG; VIRUS VECTOR; ZIC1 PROTEIN;

ADAPTIVE IMMUNITY; CELL PROLIFERATION; CELL SELECTION; CELL THERAPY; CONTROLLED CLINICAL TRIAL (TOPIC); DOPAMINE RELEASE; DOPAMINERGIC NERVE CELL; DYSKINESIA; FETAL TISSUE TRANSPLANTATION; FIBROBLAST; FLOW CYTOMETRY; HUMAN; MESENCEPHALIC TISSUE; NEURAL STEM CELL; NONHUMAN; NUCLEAR REPROGRAMMING; PARKINSON DISEASE; PLURIPOTENT STEM CELL; POSITRON EMISSION TOMOGRAPHY; REVIEW;

EID: 84975688051     PISSN: None     EISSN: 21525250     Source Type: Journal    
DOI: 10.14336/AD.2014.1201     Document Type: Review

References (56)

1. Buttery PC, Barker RA (2014). Treating Parkinson's disease in the 21st century: can stem cell transplantation compete? J Comp Neurol, 522: 2802-2816.
2. Williams-Gray CH, Evans JR, Goris A, Foltynie T, Ban M, Robbins TW, et al. (2009). The distinct cognitive syndromes of Parkinson's disease: 5 year follow-up of the CamPaIGN cohort. Brain, 132: 2958-2969.
3. Chaudhuri KR, Healy DG, Schapira AH (2006). Non-motor symptoms of Parkinson's disease: diagnosis and management. Lancet Neurol, 5: 235-245.
4. Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, Lawlor PA, et al. (2007). Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet, 369: 2097-2105.
5. Marks WJ, Jr., Ostrem JL, Verhagen L, Starr PA, Larson PS, Bakay RA, et al. (2008). Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol, 7: 400-408.
6. Marks WJ, Jr., Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, et al. (2010). Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol, 9: 1164-1172.
7. Bankiewicz KS, Forsayeth J, Eberling JL, Sanchez-Pernaute R, Pivirotto P, Bringas J, et al. (2006). Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol Ther, 14: 564-570.
8. Azzouz M, Martin-Rendon E, Barber RD, Mitrophanous KA, Carter EE, Rohll JB, et al. (2002). Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease. J Neurosci, 22: 10302-10312.
9. Jarraya B, Boulet S, Ralph GS, Jan C, Bonvento G, Azzouz M, et al. (2009). Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia. Sci Transl Med, 1: 2ra4.
10. Arjona V, Minguez-Castellanos A, Montoro RJ, Ortega A, Escamilla F, Toledo-Aral JJ, et al. (2003). Autotransplantation of human carotid body cell aggregates for treatment of Parkinson's disease. Neurosurgery, 53: 321-328; discussion 328-330.
11. Backlund EO, Granberg PO, Hamberger B, Knutsson E, Martensson A, Sedvall G, et al. (1985). Transplantation of adrenal medullary tissue to striatum in parkinsonism. First clinical trials. J Neurosurg, 62: 169-173.
12. Itakura T, Uematsu Y, Nakao N, Nakai E, Nakai K (1997). Transplantation of autologous sympathetic ganglion into the brain with Parkinson's disease. Long-term follow-up of 35 cases. Stereotact Funct Neurosurg, 69: 112-115.
13. Schumacher JM, Ellias SA, Palmer EP, Kott HS, Dinsmore J, Dempsey PK, et al. (2000). Transplantation of embryonic porcine mesencephalic tissue in patients with PD. Neurology, 54: 1042-1050.
14. Lindvall O, Brundin P, Widner H, Rehncrona S, Gustavii B, Frackowiak R, et al. (1990). Grafts of fetal dopamine neurons survive and improve motor function in Parkinson's disease. Science, 247: 574-577.
15. Hagell P, Schrag A, Piccini P, Jahanshahi M, Brown R, Rehncrona S, et al. (1999). Sequential bilateral transplantation in Parkinson's disease: effects of the second graft. Brain, 122 (Pt 6): 1121-1132.
16. Mendez I, Vinuela A, Astradsson A, Mukhida K, Hallett P, Robertson H, et al. (2008). Dopamine neurons implanted into people with Parkinson's disease survive without pathology for 14 years. Nat Med, 14: 507-509.
17. Piccini P, Brooks DJ, Bjorklund A, Gunn RN, Grasby PM, Rimoldi O, et al. (1999). Dopamine release from nigral transplants visualized in vivo in a Parkinson's patient. Nat Neurosci, 2: 1137-1140.
18. Wenning GK, Odin P, Morrish P, Rehncrona S, Widner H, Brundin P, et al. (1997). Short-and long-term survival and function of unilateral intrastriatal dopaminergic grafts in Parkinson's disease. Ann Neurol, 42: 95-107.
19. Remy P, Samson Y, Hantraye P, Fontaine A, Defer G, Mangin JF, et al. (1995). Clinical correlates of [18F]fluorodopa uptake in five grafted parkinsonian patients. Ann Neurol, 38: 580-588.
20. Widner H, Tetrud J, Rehncrona S, Snow B, Brundin P, Gustavii B, et al. (1992). Bilateral fetal mesencephalic grafting in two patients with parkinsonism induced by 1-methyl-4-phenyl-1, 2, 3, 6-tetrahydropyridine (MPTP). N Engl J Med, 327: 1556-1563.
21. Olanow CW, Goetz CG, Kordower JH, Stoessl AJ, Sossi V, Brin MF, et al. (2003). A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann Neurol, 54: 403-414.
22. Freed CR, Greene PE, Breeze RE, Tsai WY, DuMouchel W, Kao R, et al. (2001). Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N Engl J Med, 344: 710-719.
23. Ma Y, Tang C, Chaly T, Greene P, Breeze R, Fahn S, et al. (2010). Dopamine cell implantation in Parkinson's disease: long-term clinical and (18)F-FDOPA PET outcomes. J Nucl Med, 51: 7-15.
24. Politis M, Lindvall O (2012). Clinical application of stem cell therapy in Parkinson's disease. BMC Med, 10: 1.
25. Chen Z, Palmer TD (2008). Cellular repair of CNS disorders: an immunological perspective. Hum Mol Genet, 17: R84-92.
26. Chen Z, Phillips LK, Gould E, Campisi J, Lee SW, Ormerod BK, et al. (2011). MHC mismatch inhibits neurogenesis and neuron maturation in stem cell allografts. PLoS One, 6: e14787.
27. Chen Z, Palmer TD (2013). Differential roles of TNFR1 and TNFR2 signaling in adult hippocampal neurogenesis. Brain Behav Immun, 30: 45-53.
28. Shin E, Tronci E, Carta M (2012). Role of Serotonin Neurons in L-DOPA-and Graft-Induced Dyskinesia in a Rat Model of Parkinson's Disease. Parkinsons Dis, 2012: 370190.
29. Petit GH, Olsson TT, Brundin P (2014). The future of cell therapies and brain repair: Parkinson's disease leads the way. Neuropathol Appl Neurobiol, 40: 60-70.
30. Takahashi K, Yamanaka S (2006). Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell, 126: 663-676.
31. Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, et al. (2007). Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell, 131: 861-872.
32. Kaji K, Norrby K, Paca A, Mileikovsky M, Mohseni P, Woltjen K (2009). Virus-free induction of pluripotency and subsequent excision of reprogramming factors. Nature, 458: 771-775.
33. Okita K, Hong H, Takahashi K, Yamanaka S (2010). Generation of mouse-induced pluripotent stem cells with plasmid vectors. Nat Protoc, 5: 418-428.
34. Soldner F, Hockemeyer D, Beard C, Gao Q, Bell GW, Cook EG, et al. (2009). Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell, 136: 964-977.
35. Stadtfeld M, Nagaya M, Utikal J, Weir G, Hochedlinger K (2008). Induced pluripotent stem cells generated without viral integration. Science, 322: 945-949.
36. Woltjen K, Michael IP, Mohseni P, Desai R, Mileikovsky M, Hamalainen R, et al. (2009). piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells. Nature, 458: 766-770.
37. Hou P, Li Y, Zhang X, Liu C, Guan J, Li H, et al. (2013). Pluripotent stem cells induced from mouse somatic cells by small-molecule compounds. Science, 341: 651-654.
38. Dowey SN, Huang X, Chou BK, Ye Z, Cheng L (2012). Generation of integration-free human induced pluripotent stem cells from postnatal blood mononuclear cells by plasmid vector expression. Nat Protoc, 7: 2013-2021.
39. Pruszak J, Sonntag KC, Aung MH, Sanchez-Pernaute R, Isacson O (2007). Markers and methods for cell sorting of human embryonic stem cell-derived neural cell populations. Stem Cells, 25: 2257-2268.
40. Jonsson ME, Ono Y, Bjorklund A, Thompson LH (2009). Identification of transplantable dopamine neuron precursors at different stages of midbrain neurogenesis. Exp Neurol, 219: 341-354.
41. Xi J, Liu Y, Liu H, Chen H, Emborg ME, Zhang SC (2012). Specification of midbrain dopamine neurons from primate pluripotent stem cells. Stem Cells, 30: 1655-1663.
42. Chung S, Moon JI, Leung A, Aldrich D, Lukianov S, Kitayama Y, et al. (2011). ES cell-derived renewable and functional midbrain dopaminergic progenitors. Proc Natl Acad Sci U S A, 108: 9703-9708.
43. Vierbuchen T, Ostermeier A, Pang ZP, Kokubu Y, Sudhof TC, Wernig M (2010). Direct conversion of fibroblasts to functional neurons by defined factors. Nature, 463: 1035-1041.
44. Sheng C, Zheng Q, Wu J, Xu Z, Sang L, Wang L, et al. (2012). Generation of dopaminergic neurons directly from mouse fibroblasts and fibroblast-derived neural progenitors. Cell Res, 22: 769-772.
45. Caiazzo M, Dell'Anno MT, Dvoretskova E, Lazarevic D, Taverna S, Leo D, et al. (2011). Direct generation of functional dopaminergic neurons from mouse and human fibroblasts. Nature, 476: 224-227.
46. Liu X, Li F, Stubblefield EA, Blanchard B, Richards TL, Larson GA, et al. (2012). Direct reprogramming of human fibroblasts into dopaminergic neuron-like cells. Cell Res, 22: 321-332.
47. Liu X, Huang Q, Li F, Li CY (2014). Enhancing the efficiency of direct reprogramming of human primary fibroblasts into dopaminergic neuron-like cells through p53 suppression. Sci China Life Sci, 57: 867-875.
48. Pfisterer U, Kirkeby A, Torper O, Wood J, Nelander J, Dufour A, et al. (2011). Direct conversion of human fibroblasts to dopaminergic neurons. Proc Natl Acad Sci U S A, 108: 10343-10348.
49. Sheng C, Zheng Q, Wu J, Xu Z, Wang L, Li W, et al. (2012). Direct reprogramming of Sertoli cells into multipotent neural stem cells by defined factors. Cell Res, 22: 208-218.
50. Wu J, Sheng C, Liu Z, Jia W, Wang B, Li M, et al. (2015). Lmx1a enhances the effect of iNSCs in a PD model. Stem Cell Res, 14: 1-9.
51. Lujan E, Chanda S, Ahlenius H, Sudhof TC, Wernig M (2012). Direct conversion of mouse fibroblasts to self-renewing, tripotent neural precursor cells. Proc Natl Acad Sci U S A, 109: 2527-2532.
52. Kim SM, Flasskamp H, Hermann A, Arauzo-Bravo MJ, Lee SC, Lee SH, et al. (2014). Direct conversion of mouse fibroblasts into induced neural stem cells. Nat Protoc, 9: 871-881.
53. Kim J, Efe JA, Zhu S, Talantova M, Yuan X, Wang S, et al. (2011). Direct reprogramming of mouse fibroblasts to neural progenitors. Proc Natl Acad Sci U S A, 108: 7838-7843.
54. Wang L, Huang W, Su H, Xue Y, Su Z, Liao B, et al. (2013). Generation of integration-free neural progenitor cells from cells in human urine. Nat Methods, 10: 84-89.
55. Thier M, Worsdorfer P, Lakes YB, Gorris R, Herms S, Opitz T, et al. (2012). Direct conversion of fibroblasts into stably expandable neural stem cells. Cell Stem Cell, 10: 473-479.
56. Han DW, Tapia N, Hermann A, Hemmer K, Hoing S, Arauzo-Bravo MJ, et al. (2012). Direct reprogramming of fibroblasts into neural stem cells by defined factors. Cell Stem Cell, 10: 465-472.