Protein-based human iPS cells efficiently generate functional dopamine neurons and can treat a rat model of Parkinson disease

Journal of Clinical Investigation

Volumn 121, Issue 6, 2011, Pages 2326-2335

  Rhee, Yong Hee ^a   Ko, Ji Yun ^a   Chang, Mi Yoon ^b   Yi, Sang Hoon ^a   Kim, Dohoon ^b   Kim, Chun Hyung ^a   Shim, Jae Won ^a   Jo, A Young ^a   Kim, Byung Woo ^a   Lee, Hyunsu ^c   Lee, Suk Ho ^c   Suh, Wonhee ^d,f   Park, Chang Hwan ^a   Koh, Hyun Chul ^a   Lee, Yong Sung ^a   Lanza, Robert ^e   Kim, Kwang Soo ^b,d   Lee, Sang Hun ^a  

^a Hanyang University   (South Korea)

^b HARVARD MEDICAL SCHOOL   (United States)

^c Seoul National University College of Medicine   (South Korea)

^d CHA University   (South Korea)

^e Stem Cell and Regenerative Medicine International   (United States)

^f Ajou University   (South Korea)

Author keywords

[No Author keywords available]

Indexed keywords

CYCLOSPORIN A; DOPAMINE; KRUPPEL LIKE FACTOR 4; OCTAMER TRANSCRIPTION FACTOR 4; PROTEIN; PROTEIN P53; TRANSCRIPTION FACTOR; TRANSCRIPTION FACTOR LIN28; TRANSCRIPTION FACTOR NANOG; TRANSCRIPTION FACTOR SOX2; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL MODEL; APOPTOSIS; ARTICLE; CELL AGING; CELL DEATH; CELL DIFFERENTIATION; CELL FUNCTION; CELL LINE; CELL PROLIFERATION; CONTROLLED STUDY; DOPAMINERGIC NERVE CELL; GENE EXPRESSION; GRAFT REJECTION; HUMAN; HUMAN CELL; LENTIVIRINAE; NEURAL STEM CELL; NEURONAL PRECURSOR CELL; NONHUMAN; NUCLEAR REPROGRAMMING; PARKINSON DISEASE; PLURIPOTENT STEM CELL; PLURIPOTENT STEM CELL TRANSPLANTATION; PRIORITY JOURNAL; PROTEIN INDUCTION; RAT; RETROVIRUS; SIGNAL TRANSDUCTION; STEM CELL EXPANSION; STEM CELL TRANSPLANTATION; STROMA CELL;

ANIMALS; APOPTOSIS; ARGININE; CELL AGING; CELL DIFFERENTIATION; CELL LINE; CELL LINEAGE; DOPAMINE; GENE EXPRESSION REGULATION, DEVELOPMENTAL; GENES, P53; GENETIC VECTORS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; KRUPPEL-LIKE TRANSCRIPTION FACTORS; LENTIVIRUS; NEURONS; NUCLEAR REPROGRAMMING; OCTAMER TRANSCRIPTION FACTOR-3; PARKINSONIAN DISORDERS; PROTO-ONCOGENE PROTEINS C-MYC; RATS; RETROVIRIDAE; SOXB1 TRANSCRIPTION FACTORS; TUMOR SUPPRESSOR PROTEIN P53;

EID: 79957913513     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI45794     Document Type: Article

References (49)

1. Savitt JM, Dawson VL, Dawson TM. Diagnosis and treatment of Parkinson disease: molecules to medicine. J Clin Invest. 2006;116(7):1744-1754. (Pubitemid 44033293)
2. Piccini P, et al. Dopamine release from nigral transplants visualized in vivo in a Parkinson's patient. Nat Neurosci. 1999;2(12):1137-1140. (Pubitemid 30126374)
3. Freed CR, et al. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N Engl J Med. 2001;344(10):710-719. (Pubitemid 32204988)
4. Dunnett SB, Bjorklund A, Lindvall O. Cell therapy in Parkinson's disease - stop or go? Nat Rev Neurosci. 2001;2(5):365-369. (Pubitemid 33680578)
5. Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell. 2006; 126(4):663-676. (Pubitemid 44233629)
6. Takahashi K, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell. 2007;131(5):861-872. (Pubitemid 350138099)
7. Yu J, et al. Induced pluripotent stem cell lines derived from human somatic cells. Science. 2007; 318(5858):1917-1920.
8. Park IH, et al. Reprogramming of human somatic cells to pluripotency with defined factors. Nature. 2008;451(7175):141-146.
9. Gearhart J, Pashos EE, Prasad MK. Pluripotency redux - advances in stem-cell research. N Engl J Med. 2007;357(15):1469-1472. (Pubitemid 47572631)
10. Kim D, et al. Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins. Cell Stem Cell. 2009;4(6):472-476.
11. Osafune K, et al. Marked differences in differentiation propensity among human embryonic stem cell lines. Nat Biotechnol. 2008;26(3):313-315. (Pubitemid 351355125)
12. Miura K, et al. Variation in the safety of induced pluripotent stem cell lines. Nat Biotechnol. 2009; 27(8):743-745.
13. Hu BY, et al. Neural differentiation of human induced pluripotent stem cells follows developmental principles but with variable potency. Proc Natl Acad Sci U S A. 2010;107(9):4335-4340.
14. Kawasaki H, et al. Induction of midbrain dopaminergic neurons from ES cells by stromal cell-derived inducing activity. Neuron. 2000;28(1):31-40.
15. Perrier AL, et al. Derivation of midbrain dopamine neurons from human embryonic stem cells. Proc Natl Acad Sci U S A. 2004;101(34):12543-12548. (Pubitemid 39122060)
16. Kim DW, et al. Stromal cell-derived inducing activity, Nurr1, and signaling molecules synergistically induce dopaminergic neurons from mouse embryonic stem cells. Stem Cells. 2006;24(3):557-567.
17. Ko JY, et al. Human embryonic stem cell-derived neural precursors as a continuous, stable, and on-demand source for human dopamine neurons. J Neurochem. 2007;103(4):1417-1429.
18. Shen Q, et al. Endothelial cells stimulate self-renewal and expand neurogenesis of neural stem cells. Science. 2004;304(5675):1338-1340. (Pubitemid 38697433)
19. Roy NS, Cleren C, Singh SK, Yang L, Beal MF, Goldman SA. Functional engraftment of human ES cell-derived dopaminergic neurons enriched by coculture with telomerase-immortalized midbrain astrocytes. Nat Med. 2006;12(11):1259- 1268. (Pubitemid 44706928)
20. Pankratz MT, Li XJ, Lavaute TM, Lyons EA, Chen X, Zhang SC. Directed neural differentiation of human embryonic stem cells via an obligated primitive anterior stage. Stem Cells. 2007;25(6):1511-1520. (Pubitemid 47106020)
21. Elkabetz Y, Panagiotakos G, Al Shamy G, Socci ND, Tabar V, Studer L. Human ES cell-derived neural rosettes reveal a functionally distinct early neural stem cell stage. Genes Dev. 2008;22(2):152-165.
22. LaVaute TM, Yoo YD, Pankratz MT, Weick JP, Gerstner JR, Zhang SC. Regulation of neural specification from human embryonic stem cells by BMP and FGF. Stem Cells. 2009;27(8):1741-1749.
23. Okita K, Nakagawa M, Hyenjong H, Ichisaka T, Yamanaka S. Generation of mouse induced pluripotent stem cells without viral vectors. Science. 2008;322(5903):949-953.
24. Graham V, Khudyakov J, Ellis P, Pevny L. SOX2 functions to maintain neural progenitor identity. Neuron. 2003;39(5):749-765. (Pubitemid 37088088)
25. Kim JB, et al. Pluripotent stem cells induced from adult neural stem cells by reprogramming with two factors. Nature. 2008;454(7204):646-650.
26. Favaro R, et al. Hippocampal development and neural stem cell maintenance require Sox2-dependent regulation of Shh. Nat Neurosci. 2009;12(10):1248-1256.
27. Chung S, et al. Neural precursors derived from embryonic stem cells, but not those from fetal ventral mesencephalon, maintain the potential to differentiate into dopaminergic neurons after expansion in vitro. Stem Cells. 2006;24(6):1583-1593. (Pubitemid 44464676)
28. Hong S, Kang UJ, Isacson O, Kim KS. Neural precursors derived from human embryonic stem cells maintain long-term proliferation without losing the potential to differentiate into all three neural lineages, including dopaminergic neurons. J Neurochem. 2008;104(2):316-324. (Pubitemid 350293864)
29. Sharpless NE, DePinho RA. How stem cells age and why this makes us grow old. Nat Rev Mol Cell Biol. 2007;8(9):703-713. (Pubitemid 47312828)
30. Green DR, Kroemer G. Cytoplasmic functions of the tumour suppressor p53. Nature. 2009; 458(7242):1127-1130.
31. Lee SH, Ho WK. Characterization of somatic Ca2+ clearance mechanisms in young and mature hippocampal granule cells. Cell Calcium. 2009; 45(5):465-473.
32. Hamaguchi I, et al. Lentivirus vector gene expression during ES cell-derived hematopoietic development in vitro. J Virol. 2000;74(22):10778- 10784.
33. Laker C, et al. Host cis-mediated extinction of a retrovirus permissive for expression in embryonal stem cells during differentiation. J Virol. 1998;72(1):339-348. (Pubitemid 28048845)
34. Feng Q, et al. Hemangioblastic derivatives from human induced pluripotent stem cells exhibit limited expansion and early senescence. Stem Cells. 2010;28(4):704-712.
35. Kim JB, et al. Direct reprogramming of human neural stem cells by OCT4. Nature. 2009; 461(7264):649-643.
36. Borowiak M, et al. Small molecules efficiently direct endodermal differentiation of mouse and human embryonic stem cells. Cell Stem Cell. 2009; 4(4):348-358.
37. Lin T, et al. A chemical platform for improved induction of human iPSCs. Nat Methods. 2009; 6(11):805-808.
38. Soldner F, et al. Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell. 2009;136(5):964-977.
39. Woltjen K, et al. piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells. Nature. 2009;458(7239):766-770.
40. Kaji K, Norrby K, Paca A, Mileikovsky M, Mohseni P, Woltjen K. Virus-free induction of pluripotency and subsequent excision of reprogramming factors. Nature. 2009;458(7239):771-775.
41. Yu J, et al. Human induced pluripotent stem cells free of vector and transgene sequences. Science. 2009;324(5928):797-801.
42. Jia F, et al. A nonviral minicircle vector for deriving human iPS cells. Nat Methods. 2010;7(3):197-199.
43. Hargus G, et al. Differentiated Parkinson patient-derived induced pluripotent stem cells grow in the adult rodent brain and reduce motor asymmetry in Parkinsonian rats. Proc Natl Acad Sci U S A. 2010;107(36):15921-15926.
44. Swistowski A, et al. Efficient generation of functional dopaminergic neurons from human induced pluripotent stem cells under defined conditions. Stem Cells. 2010;28(10):1893-1904.
45. Park CH, et al. In vitro and in vivo analyses of human embryonic stem cell-derived dopamine neurons. J Neurochem. 2005;92(5):1265-1276.
46. Jo AY, et al. Generation of dopamine neurons with improved cell survival and phenotype maintenance using a degradation-resistant nurr1 mutant. Stem Cells. 2009;27(9):2238-2246.
47. Lee TH, et al. Functional recapitulation of smooth muscle cells via induced pluripotent stem cells from human aortic smooth muscle cells. Circ Res. 2010;106(1):120-128.
48. Carpenter MK, Rosler E, Rao M. Characterization and differentiation of human embryonic stem cells. Cloning Stem Cells. 2003;5(1):79-88. (Pubitemid 36418429)
49. Thomson JA, et al. Embryonic stem cell lines derived from human blastocysts. Science. 1998; 282(5391):1145-1147. (Pubitemid 28516247)