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Mini Reviews

Drug discovery toward successful cell transplantation therapy for Parkinson's disease using human pluripotent stem cells

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Article: 31772 | Received 20 Mar 2016, Accepted 18 Jul 2016, Published online: 09 Aug 2016

References

  • Brundin P, Strecker RE, Lindvall O, Isacson O, Nilsson OG, Barbin G et al. Intracerebral grafting of dopamine neurons. Experimental basis for clinical trials in patients with Parkinson's disease. Ann N Y Acad Sci. 1987; 495: 473-96. [ PubMed Abstract]
  • Lindvall O, Rehncrona S, Brundin P, Gustavii B, Astedt B, Widner H et al. Human fetal dopamine neurons grafted into the striatum in two patients with severe Parkinson's disease. A detailed account of methodology and a 6-month follow-up. Arch Neurol. 1989; 46: 615-31. [ PubMed Abstract]
  • Hauser RA, Freeman TB, Snow BJ, Nauert M, Gauger L, Kordower JH et al. Long-term evaluation of bilateral fetal nigral transplantation in Parkinson disease. Arch Neurol. 1999; 56: 179-87. [ PubMed Abstract]
  • Widner H, Tetrud J, Rehncrona S, Snow B, Brundin P, Gustavii B et al. 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. 1992; 327: 1556-63. [ PubMed Abstract]
  • Freed CR, Greene PE, Breeze RE, Tsai WY, DuMouchel W, Kao R et al. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N Engl J Med. 2001; 344: 710-19. [ PubMed Abstract]
  • Olanow CW, Goetz CG, Kordower JH, Stoessl AJ, Sossi V, Brin MF et al. A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann Neurol. 2003; 54: 403-14. [ PubMed Abstract]
  • Mendez I, Viñuela A, Astradsson A, Mukhida K, Hallett P, Robertson H et al. Dopamine neurons implanted into people with Parkinson's disease survive without pathology for 14 years. Nat Med. 2008; 14: 507-9. [ PubMed Abstract] [PubMed CentralFull Text]
  • Hallett PJ, Cooper O, Sadi D, Robertson H, Mendez I, Isacson O. Long-term health of dopaminergic neuron transplants in Parkinson's disease patients. Cell Rep. 2014; 7: 1755-61. [ PubMed Abstract] [PubMed CentralFull Text]
  • Kefalopoulou Z, Politis M, Piccini P, Mencacci N, Bhatia K, Jahanshahi M et al. Long-term clinical outcome of fetal cell transplantation for Parkinson disease: two case reports. JAMA Neurol. 2014; 71: 83-7. [ PubMed Abstract] [PubMed CentralFull Text]
  • Lindvall O, Kokaia Z. Prospects of stem cell therapy for replacing dopamine neurons in Parkinson's disease. Trends Pharmacol Sci. 2009; 30: 260-7. [ PubMed Abstract]
  • Grealish S, Diguet E, Kirkeby A, Mattsson B, Heuer A, Bramoulle Y et al. Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease. Cell Stem Cell. 2014; 15: 653-65. [ PubMed Abstract] [PubMed CentralFull Text]
  • Lindvall O. Clinical translation of stem cell transplantation in Parkinson's disease. J Intern Med. 2016; 279: 30-40. [ PubMed Abstract]
  • Kriks S, Shim JW, Piao J, Ganat YM, Wakeman DR, Xie Z et al. Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinson's disease. Nature. 2011; 480: 547-51. [ PubMed Abstract] [PubMed CentralFull Text]
  • Kirkeby A, Grealish S, Wolf DA, Nelander J, Wood J, Lundblad M et al. Generation of regionally specified neural progenitors and functional neurons from human embryonic stem cells under defined conditions. Cell Rep. 2012; 1: 703-14. [ PubMed Abstract]
  • Sundberg M, Bogetofte H, Lawson T, Jansson J, Smith G, Astradsson A et al. Improved cell therapy protocols for Parkinson's disease based on differentiation efficiency and safety of hESC-, hiPSC-, and non-human primate iPSC-derived dopaminergic neurons. Stem Cells. 2013; 31: 1548-62. [ PubMed Abstract]
  • Doi D, Samata B, Katsukawa M, Kikuchi T, Morizane A, Ono Y et al. Isolation of human induced pluripotent stem cell-derived dopaminergic progenitors by cell sorting for successful transplantation. Stem Cell Rep. 2014; 2: 337-50.
  • Sortwell CE, Pitzer MR, Collier TJ. Time course of apoptotic cell death within mesencephalic cell suspension grafts: implications for improving grafted dopamine neuron survival. Exp Neurol. 2000; 165: 268-77. [ PubMed Abstract]
  • Rosenblad C, Martinez-Serrano A, Björklund A. Glial cell line-derived neurotrophic factor increases survival, growth and function of intrastriatal fetal nigral dopaminergic grafts. Neuroscience. 1996; 75: 979-85. [ PubMed Abstract]
  • Koyanagi M, Takahashi J, Arakawa Y, Doi D, Fukuda H, Hayashi H et al. Inhibition of the rho/ROCK pathway reduces apoptosis during transplantation of embryonic stem cell-derived neural precursors. J Neurosci Res. 2008; 86: 270-80. [ PubMed Abstract]
  • Parish CL, Castelo-Branco G, Rawal N, Tonnesen J, Sorensen AT, Salto C et al. Wnt5a-treated midbrain neural stem cells improve dopamine cell replacement therapy in parkinsonian mice. J Clin Invest. 2008; 118: 149-60. [ PubMed Abstract]
  • Kauhausen JA, Thompson LH, Parish CL. Chondroitinase improves midbrain pathway reconstruction by transplanted dopamine progenitors in parkinsonian mice. Mol Cell Neurosci. 2015; 69: 22-9. [ PubMed Abstract]
  • Peng SP, Schachner M, Boddeke E, Copray S. Effect of cell adhesion molecules on the neurite outgrowth of induced pluripotent stem cell-derived dopaminergic neurons. Cell Reprogram. 2016; 18: 55-66. [ PubMed Abstract]
  • Nishimura K, Doi D, Samata B, Murayama S, Tahara T, Onoe H et al. Estradiol facilitates functional integration of iPSC-derived dopaminergic neurons into striatal neuronal circuits via activation of integrin α5β1. Stem Cell Rep. 2016; 6: 932-44.
  • Thomson JA, Itskovitz-Eldor J, Shapiro SS, Waknitz MA, Swiergiel JJ, Marshall VS et al. Embryonic stem cell lines derived from human blastocysts. Science. 1998; 282: 1145-7. [ PubMed Abstract]
  • Rodin S, Antonsson L, Niaudet C, Simonson OE, Salmela E, Hansson EM et al. Clonal culturing of human embryonic stem cells on laminin-521/E-cadherin matrix in defined and xeno-free environment. Nat Commun. 2014; 5: 3195 [ PubMed Abstract]
  • Canham MA, Van Deusen A, Brison DR, De Sousa PA, Downie J, Devito L et al. The molecular karyotype of 25 clinical-grade human embryonic stem cell lines. Sci Rep. 2015; 5: 17258 [ PubMed Abstract] [PubMed CentralFull Text]
  • Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell. 2007; 131: 861—72
  • Yu J, Vodyanik MA, Smuga-Otto K, Antosiewicz-Bourget J, Frane JL, Tian S et al. Induced pluripotent stem cell lines derived from human somatic cells. Science. 2007; 318: 1917-20. [ PubMed Abstract]
  • Okita K, Matsumura Y, Sato Y, Okada A, Morizane A, Okamoto S et al. A more efficient method to generate integration-free human iPS cells. Nat Methods. 2011; 8: 409-12. [ PubMed Abstract]
  • Nakagawa M, Taniguchi Y, Senda S, Takizawa N, Ichisaka T, Asano K et al. A novel efficient feeder-free culture system for the derivation of human induced pluripotent stem cells. Sci Rep. 2014; 4: 3594 [ PubMed Abstract] [PubMed CentralFull Text]
  • Morizane A, Doi D, Kikuchi T, Okita K, Hotta A, Kawasaki T et al. Direct comparison of autologous and allogeneic transplantation of iPSC-derived neural cells in the brain of a nonhuman primate. Stem Cell Rep. 2013; 1: 283-92.
  • Hallett PJ, Deleidi M, Astradsson A, Smith GA, Cooper O, Osborn TM et al. Successful function of autologous iPSC-derived dopamine neurons following transplantation in a non-human primate model of Parkinson's disease. Cell Stem Cell. 2015; 16: 269-74. [ PubMed Abstract] [PubMed CentralFull Text]
  • Arenas E, Denham M, Villaescusa JC. How to make a midbrain dopaminergic neuron. Development. 2015; 142: 1918-36. [ PubMed Abstract]
  • Chambers SM, Fasano CA, Papapetrou EP, Tomishima M, Sadelain M, Studer L. Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling. Nat Biotechnol. 2009; 27: 275-80. [ PubMed Abstract] [PubMed CentralFull Text]
  • Morizane A, Doi D, Kikuchi T, Nishimura K, Takahashi J. Small-molecule inhibitors of bone morphogenic protein and activin/nodal signals promote highly efficient neural induction from human pluripotent stem cells. J Neurosci Res. 2011; 89: 117-26. [ PubMed Abstract]
  • Denham M, Bye C, Leung J, Conley BJ, Thompson LH, Dottori M. Glycogen synthase kinase 3β and activin/nodal inhibition in human embryonic stem cells induces a pre-neuroepithelial state that is required for specification to a floor plate cell lineage. Stem Cells. 2012; 30: 2400-11. [ PubMed Abstract] [PubMed CentralFull Text]
  • Sundberg M, Isacson O. Advances in stem-cell-generated transplantation therapy for Parkinson's disease. Expert Opin Biol Ther. 2014; 14: 437-53. [ PubMed Abstract]
  • Fukuda H, Takahashi J, Watanabe K, Hayashi H, Morizane A, Koyanagi M et al. Fluorescence-activated cell sorting-based purification of embryonic stem cell-derived neural precursors averts tumor formation after transplantation. Stem Cells. 2006; 24: 763-71. [ PubMed Abstract]
  • Pruszak J, Ludwig W, Blak A, Alavian K, Isacson O. CD15, CD24, and CD29 define a surface biomarker code for neural lineage differentiation of stem cells. Stem Cells. 2009; 27: 2928-40. [ PubMed Abstract] [PubMed CentralFull Text]
  • Chung S, Moon JI, Leung A, Aldrich D, Lukianov S, Kitayama Y et al. ES cell-derived renewable and functional midbrain dopaminergic progenitors. Proc Natl Acad Sci USA. 2011; 108: 9703-8. [ PubMed Abstract] [PubMed CentralFull Text]
  • Ganat YM, Calder EL, Kriks S, Nelander J, Tu EY, Jia F et al. Identification of embryonic stem cell-derived midbrain dopaminergic neurons for engraftment. J Clin Invest. 2012; 122: 2928-39. [ PubMed Abstract] [PubMed CentralFull Text]
  • Bye CR, Jönsson ME, Björklund A, Parish CL, Thompson LH. Transcriptome analysis reveals transmembrane targets on transplantable midbrain dopamine progenitors. Proc Natl Acad Sci USA. 2015; 112: E1946-55. [ PubMed Abstract] [PubMed CentralFull Text]
  • Peng J, Liu Q, Rao MS, Zeng X. Survival and engraftment of dopaminergic neurons manufactured by a good manufacturing practice-compatible process. Cytotherapy. 2014; 16: 1305-12. [ PubMed Abstract]
  • Carpenter MK, Rao MS. Concise review: making and using clinically compliant pluripotent stem cell lines. Stem Cells Transl Med. 2015; 4: 381-8. [ PubMed Abstract] [PubMed CentralFull Text]
  • Morizane A, Takahashi J. Cell therapy for Parkinson's disease. Neurol Med Chir (Tokyo). 2016; 15: 102-9.
  • Daley G, Hyun I, Apperley J, Barker R, Benvenisty N, Bredenoord A et al. Setting global standards for stem cell research and clinical translation: the ISSCR guidelines. Stem Cell Rep. 2016; 6: 787-97.
  • Yoshikawa T, Samata B, Ogura A, Miyamoto S, Takahashi J. Systemic administration of valproic acid and zonisamide promotes differentiation of induced pluripotent stem cell-derived dopaminergic neurons. Front Cell Neurosci. 2013; 7: 11 [ PubMed Abstract] [PubMed CentralFull Text]
  • Nishimura K, Murayama S, Takahashi J. Identification of neurexophilin 3 as a novel supportive factor for survival of induced pluripotent stem cell-derived dopaminergic progenitors. Stem Cells Transl Med. 2015; 4: 932-44. [ PubMed Abstract] [PubMed CentralFull Text]
  • Sørensen AT, Thompson L, Kirik D, Björklund A, Lindvall O, Kokaia M. Functional properties and synaptic integration of genetically labelled dopaminergic neurons in intrastriatal grafts. Eur J Neurosci. 2005; 21: 2793-9.
  • Grealish S, Heuer A, Cardoso T, Kirkeby A, Jönsson M, Johansson J et al. Monosynaptic tracing using modified rabies virus reveals early and extensive circuit integration of human embryonic stem cell-derived neurons. Stem Cell Rep. 2015; 4: 975-83.
  • Steinbeck JA, Choi SJ, Mrejeru A, Ganat Y, Deisseroth K, Sulzer D et al. Optogenetics enables functional analysis of human embryonic stem cell-derived grafts in a Parkinson's disease model. Nat Biotechnol. 2015; 33: 204-9. [ PubMed Abstract]
  • Chen Y, Xiong M, Dong Y, Haberman A, Cao J, Liu H et al. Chemical control of grafted human PSC-derived neurons in a mouse model of Parkinson's disease. Cell Stem Cell. 2016; 18: 817-26. [ PubMed Abstract] [PubMed CentralFull Text]