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Prospects for Cell Replacement Therapies for Neurodegenerative Diseases

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Induced Pluripotent Stem Cells in Brain Diseases

Part of the book series: SpringerBriefs in Neuroscience ((BRIEFSNEUROSCI))

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Abstract

Our extensive knowledge of pluripotent SCs has been used by many researchers to develop cell replacement therapies for different diseases, including those affecting the brain. Different studies with animal models of neurological disease showed that cell grafts are able to improve clinical symptoms. The recent advances in the iPSC field encourage more neuroscientists to improve transplantation strategies for clinical purposes. Here we review preclinical studies involving cell-based therapies for Parkinson’s disease (PD), and discuss the future prospects for cell replacement therapies for PD and the childhood white matter disorder, Vanishing White Matter (VWM), which disorders are our research focus.

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References

  • Albin RL, Young AB, Penney JB (1995) The functional anatomy of disorders of the basal ganglia. Trends Neurosci 18:63–64

    Article  PubMed  CAS  Google Scholar 

  • Bankiewicz KS, Plunkett RJ, Jacobowitz DM, Porrino L, di PU, London WT, Kopin IJ, Oldfield EH (1990) The effect of fetal mesencephalon implants on primate MPTP-induced parkinsonism. Histochemical and behavioral studies. J Neurosurg 72:231–244

    Article  PubMed  CAS  Google Scholar 

  • Braak H, Braak E (2000) Pathoanatomy of Parkinson’s disease. J Neurol 247(Suppl2):II3–10

    Google Scholar 

  • Brundin P, Nilsson OG, Strecker RE, Lindvall O, Astedt B, Bjorklund A (1986) Behavioural effects of human fetal dopamine neurons grafted in a rat model of Parkinson’s disease. Exp Brain Res 65:235–240

    Article  PubMed  CAS  Google Scholar 

  • Bugiani M, Boor I, van Kollenburg B, Postma N, Polder E, van Berkel C, van Kesteren RE, Windrem MS, Hol EM, Scheper GC, Goldman SA, van der Knaap MS (2011) Defective glial maturation in vanishing white matter disease. J Neuropathol Exp Neurol 70:69–82

    Article  PubMed  Google Scholar 

  • Chaudhuri KR, Healy DG, Schapira AH (2006) Non-motor symptoms of Parkinson’s disease: diagnosis and management. Lancet Neurol 5:235–245

    Article  PubMed  Google Scholar 

  • Cooper O, Hargus G, Deleidi M, Blak A, Osborn T, Marlow E, Lee K, Levy A, Perez-Torres E, Yow A, Isacson O (2010) Differentiation of human ES and Parkinson’s disease iPS cells into ventral midbrain dopaminergic neurons requires a high activity form of SHH, FGF8a and specific regionalization by retinoic acid. Mol Cell Neurosci 45:258–266

    Article  PubMed  CAS  Google Scholar 

  • de Rijk MC, Tzourio C, Breteler MM, Dartigues JF, Amaducci L, Lopez-Pousa S, Manubens-Bertran JM, Alperovitch A, Rocca WA (1997) Prevalence of parkinsonism and Parkinson’s disease in Europe: the EUROPARKINSON collaborative study. European Community concerted action on the epidemiology of Parkinson’s disease. J Neurol Neurosurg Psychiatry 62:10–15

    Article  PubMed  Google Scholar 

  • Eichler F, Van Haren K (2007) Immune response in leukodystrophies. Pediatr Neurol 37:235–244

    Article  PubMed  Google Scholar 

  • Freed CR (2002) Will embryonic stem cells be a useful source of dopamine neurons for transplant into patients with Parkinson’s disease? Proc Natl Acad Sci USA 99:1755–1757

    Article  PubMed  CAS  Google Scholar 

  • Gelb DJ, Oliver E, Gilman S (1999) Diagnostic criteria for Parkinson disease. Arch Neurol 56:33–39

    Article  PubMed  CAS  Google Scholar 

  • Geva M, Cabilly Y, Assaf Y, Mindroul N, Marom L, Raini G, Pinchasi D, Elroy-Stein O (2010) A mouse model for eukaryotic translation initiation factor 2B-leucodystrophy reveals abnormal development of brain white matter. Brain 133:2448–2461

    Article  PubMed  Google Scholar 

  • Gibb WR, Lees AJ (1989) The significance of the Lewy body in the diagnosis of idiopathic Parkinson’s disease. Neuropathol Appl Neurobiol 15:27–44

    Article  PubMed  CAS  Google Scholar 

  • Hargus G, Cooper O, Deleidi M, Levy A, Lee K, Marlow E, Yow A, Soldner F, Hockemeyer D, Hallett PJ, Osborn T, Jaenisch R, Isacson O (2010) 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 USA 107:15921–15926

    Article  PubMed  CAS  Google Scholar 

  • Hedlund E, Perlmann T (2009) Neuronal cell replacement in Parkinson’s disease. J Intern Med 266:358–371

    Article  PubMed  CAS  Google Scholar 

  • Hwang DY, Kim DS, Kim DW (2010) Human ES and iPS cells as cell sources for the treatment of Parkinson’s disease: current state and problems. J Cell Biochem 109:292–301

    PubMed  CAS  Google Scholar 

  • Kawasaki H, Suemori H, Mizuseki K, Watanabe K, Urano F, Ichinose H, Haruta M, Takahashi M, Yoshikawa K, Nishikawa S, Nakatsuji N, Sasai Y (2002) Generation of dopaminergic neurons and pigmented epithelia from primate ES cells by stromal cell-derived inducing activity. Proc Natl Acad Sci USA 99:1580–1585

    Article  PubMed  CAS  Google Scholar 

  • Kim SU, de Vellis VJ (2009) Stem cell-based cell therapy in neurological diseases: a review. J Neurosci Res 87:2183–2200

    Article  PubMed  CAS  Google Scholar 

  • Koch P, Kokaia Z, Lindvall O, Brustle O (2009) Emerging concepts in neural stem cell research: autologous repair and cell-based disease modelling. Lancet Neurol 8:819–829

    Article  PubMed  Google Scholar 

  • Kordower JH, Freeman TB, Snow BJ, Vingerhoets FJ, Mufson EJ, Sanberg PR, Hauser RA, Smith DA, Nauert GM, Perl DP (1995) Neuropathological evidence of graft survival and striatal reinnervation after the transplantation of fetal mesencephalic tissue in a patient with Parkinson’s disease. N Engl J Med 332:1118–1124

    Article  PubMed  CAS  Google Scholar 

  • Labauge P, Horzinski L, Ayrignac X, Blanc P, Vukusic S, Rodriguez D, Mauguiere F, Peter L, Goizet C, Bouhour F, Denier C, Confavreux C, Obadia M, Blanc F, de SJ, Fogli A, Boespflug-Tanguy O (2009) Natural history of adult-onset eIF2B-related disorders: a multi-centric survey of 16 cases. Brain 132:2161–2169

    Article  PubMed  Google Scholar 

  • Lee SH, Lumelsky N, Studer L, Auerbach JM, McKay RD (2000) Efficient generation of midbrain and hindbrain neurons from mouse embryonic stem cells. Nat Biotechnol 18:675–679

    Article  PubMed  CAS  Google Scholar 

  • Lindvall O, Widner H, Rehncrona S, Brundin P, Odin P, Gustavii B, Frackowiak R, Leenders KL, Sawle G, Rothwell JC (1992) Transplantation of fetal dopamine neurons in Parkinson’s disease: one-year clinical and neurophysiological observations in two patients with putaminal implants. Ann Neurol 31:155–165

    Article  PubMed  CAS  Google Scholar 

  • Luquin MR, Scipioni O, Vaamonde J, Gershanik O, Obeso JA (1992) Levodopa-induced dyskinesias in Parkinson’s disease: clinical and pharmacological classification. Mov Disord 7:117–124

    Article  PubMed  CAS  Google Scholar 

  • Maries E, Dass B, Collier TJ, Kordower JH, Steece-Collier K (2003) The role of alpha-synuclein in Parkinson’s disease: insights from animal models. Nat Rev Neurosci 4:727–738

    Article  PubMed  CAS  Google Scholar 

  • Montastruc JL, Llau-Bousquet ME, Senard JM, Rascol O (1997) Movement disorders of drug origin. Rev Prat 47:1109–1116

    PubMed  CAS  Google Scholar 

  • Morizane A, Li JY, Brundin P (2008) From bench to bed: the potential of stem cells for the treatment of Parkinson’s disease. Cell Tissue Res 331:323–336

    Article  PubMed  Google Scholar 

  • Olanow CW, Goetz CG, Kordower JH, Stoessl AJ, Sossi V, Brin MF, Shannon KM, Nauert GM, Perl DP, Godbold J, Freeman TB (2003) A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson’s disease. Ann Neurol 54:403–414

    Article  PubMed  Google Scholar 

  • Papadeas ST, Maragakis NJ (2009) Advances in stem cell research for Amyotrophic Lateral Sclerosis. Curr Opin Biotechnol 20:545–551

    Article  PubMed  CAS  Google Scholar 

  • Park CH, Minn YK, Lee JY, Choi DH, Chang MY, Shim JW, Ko JY, Koh HC, Kang MJ, Kang JS, Rhie DJ, Lee YS, Son H, Moon SY, Kim KS, Lee SH (2005) In vitro and in vivo analyses of human embryonic stem cell-derived dopamine neurons. J Neurochem 92:1265–1276

    Article  PubMed  CAS  Google Scholar 

  • Preynat-Seauve O, Burkhard PR, Villard J, Zingg W, Ginovart N, Feki A, Dubois-Dauphin M, Hurst SA, Mauron A, Jaconi M, Krause KH (2009) Pluripotent stem cells as new drugs? The example of Parkinson’s disease. Int J Pharm 381:113–121

    Article  PubMed  CAS  Google Scholar 

  • Rhee YH, Ko JY, Chang MY, Yi SH, Kim D, Kim CH, Shim JW, Jo AY, Kim BW, Lee H, Lee SH, Suh W, Park CH, Koh HC, Lee YS, Lanza R, Kim KS, Lee SH (2011) Protein-based human iPS cells efficiently generate functional dopamine neurons and can treat a rat model of Parkinson disease. J Clin Invest 121:2326–2335

    Article  PubMed  CAS  Google Scholar 

  • Soldner F, Hockemeyer D, Beard C, Gao Q, Bell GW, Cook EG, Hargus G, Blak A, Cooper O, Mitalipova M, Isacson O, Jaenisch R (2009) Parkinson’s disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell 136:964–977

    Article  PubMed  CAS  Google Scholar 

  • Spillantini MG, Schmidt ML, Lee VM, Trojanowski JQ, Jakes R, Goedert M (1997) Alpha-synuclein in Lewy bodies. Nature 388:839–840

    Article  PubMed  CAS  Google Scholar 

  • Takagi Y, Takahashi J, Saiki H, Morizane A, Hayashi T, Kishi Y, Fukuda H, Okamoto Y, Koyanagi M, Ideguchi M, Hayashi H, Imazato T, Kawasaki H, Suemori H, Omachi S, Iida H, Itoh N, Nakatsuji N, Sasai Y, Hashimoto N (2005) Dopaminergic neurons generated from monkey embryonic stem cells function in a Parkinson primate model. J Clin Invest 115:102–109

    PubMed  CAS  Google Scholar 

  • van der Knaap MS, Pronk JC, Scheper GC (2006) Vanishing white matter disease. Lancet Neurol 5:413–423

    Article  PubMed  Google Scholar 

  • van der Knaap MS, van Berkel CG, Herms J, Van CR, Baethmann M, Naidu S, Boltshauser E, Willemsen MA, Plecko B, Hoffmann GF, Proud CG, Scheper GC, Pronk JC (2003) eIF2B-related disorders: antenatal onset and involvement of multiple organs. Am J Hum Genet 73:1199–1207

    Article  PubMed  Google Scholar 

  • Wernig M, Zhao JP, Pruszak J, Hedlund E, Fu D, Soldner F, Broccoli V, Constantine-Paton M, Isacson O, Jaenisch R (2008) 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 105:5856–5861

    Article  PubMed  CAS  Google Scholar 

  • Wijesekera LC, Leigh PN (2009) Amyotrophic lateral sclerosis. Orphanet J Rare Dis 4:3

    Google Scholar 

  • Wijeyekoon R, Barker RA (2009) Cell replacement therapy for Parkinson’s disease. Biochim Biophys Acta 1792:688–702

    PubMed  CAS  Google Scholar 

  • Winkler C, Kirik D, Bjorklund A (2005) Cell transplantation in Parkinson’s disease: how can we make it work? Trends Neurosci 28:86–92

    Article  PubMed  CAS  Google Scholar 

  • Witt J, Marks WJ Jr (2011) An update on gene therapy in Parkinson’s disease. Curr Neurol Neurosci Rep 11:362–370

    Article  PubMed  CAS  Google Scholar 

  • Yasuhara T, Matsukawa N, Hara K, Yu G, Xu L, Maki M, Kim SU, Borlongan CV (2006) Transplantation of human neural stem cells exerts neuroprotection in a rat model of Parkinson’s disease. J Neurosci 26:12497–12511

    Article  PubMed  CAS  Google Scholar 

  • Yoshizaki T, Inaji M, Kouike H, Shimazaki T, Sawamoto K, Ando K, Date I, Kobayashi K, Suhara T, Uchiyama Y, Okano H (2004) Isolation and transplantation of dopaminergic neurons generated from mouse embryonic stem cells. Neurosci Lett 363:33–37

    Article  PubMed  CAS  Google Scholar 

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Author notes

  1. Judith Wagner

    Present address: Adolf-Butenandt-Institute, Biochemistry, Ludwig-Maximilians-University, 80336, Munich, Germany

Authors and Affiliations

  1. Center for Children with White Matter Disorders, VU University Medical Center, De Boelelaan 1117, 1081 HV, Amsterdam, The Netherlands

    Vivi M. Heine, Stephanie Dooves & Dwayne Holmes

  2. Clinical Genetics, VU University Medical Center, Van der Boechorststraat 7, 1081 BT, Amsterdam, The Netherlands

    Judith Wagner

Authors
  1. Vivi M. Heine
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  2. Stephanie Dooves
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  3. Dwayne Holmes
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  4. Judith Wagner
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Correspondence to Vivi M. Heine .

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Heine, V.M., Dooves, S., Holmes, D., Wagner, J. (2012). Prospects for Cell Replacement Therapies for Neurodegenerative Diseases. In: Induced Pluripotent Stem Cells in Brain Diseases. SpringerBriefs in Neuroscience. Springer, Dordrecht. https://doi.org/10.1007/978-94-007-2816-5_3

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