Stem cell-based strategies for treating pediatric disorders of myelin

Human Molecular Genetics

Volumn 17, Issue R1, 2008, Pages

  Goldman, Steven A ^a,b   Schanz, Steven ^b   Windrem, Martha S ^b  

^a Center for Translational Neuromedicine   (United States)

^b UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CELL ISOLATION; DEMYELINATING DISEASE; EMBRYONIC STEM CELL; ENGRAFTMENT; GLIA CELL; GRAFT REJECTION; HUMAN; LEUKODYSTROPHY; LYSOSOME STORAGE DISEASE; MYELIN DEFICIENCY; NEURAL STEM CELL; NONHUMAN; OLIGODENDROGLIA; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; REVIEW; STEM CELL TRANSPLANTATION;

CHILD; DEMYELINATING DISEASES; HUMANS; LEUKODYSTROPHY, GLOBOID CELL; LYSOSOMAL STORAGE DISEASES; MYELIN SHEATH; NEUROGLIA; STEM CELL TRANSPLANTATION; STEM CELLS; TISSUE THERAPY;

EID: 48049084706     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddn052     Document Type: Review

References (48)

1. Garbern, J., Cambi, F., Shy, M. and Kamholz, J. (1999) The molecular pathogenesis of Pelizaeus-Merzbacher disease. Arch. Neurol., 56 1210-1214.
2. Kaye, E. (2001) Update on genetic disorders affecting white matter. Pediatr. Neurol., 24, 11-24.
3. Kumar, S., Mattan, N. and de Vellis, J. (2006) Canavan disease: A white matter disorder. Ment. Retard. Dev. Disabil. Res. Rev., 12, 157-165.
4. van der Knaap, M., Pronk, J. and Scheper, G. (2006) Vanishing white matter disease. Lancet Neurol., 5, 413-423.
5. Powers, J. (2004) The leukodystrophies: Overview and classification. In Lazzarini, R.A. (eds), Myelin Biology and Disorders. Elsevier Academic Press, San Diego, pp. 663-690.
6. Back, S. and Rivkees, S. (2004) Emerging concepts in periventricular white matter injury. Semin. Perinatol., 6, 405-414.
7. Follett, P., Deng, W., Dai, W., Talos, D., Massilon, L., Rosenberg, P., Volpe, J. and Jensen, F. (2004) Glutamate receptor-mediated oligodendrocyte toxicity in periventricular leukomalacia: A protective role for topiramate. J. Neurosci. Res., 24, 4412-4420.
8. Levison, S.W., Rothstein, R.P., Romanko, M.J., Snyder, M.J., Meyers, R.L. and Vannucci, S.J. (2001) Hypoxia/ischemia depletes the rat perinatal subventricular zone of oligodendrocyte progenitors and neural stem cells. Dev. Neurosci., 23, 234-247.
9. Robinson, S., Petelenz, K., Li, Q., Cohen, M., Dechant A., Tabrizi, N., Bucek, M., Lust D. and Miller, R. (2005) Developmental changes induced by graded prenatal systemic hypoxic-ischemic insults in rats. Neurobiol. Dis., 18, 568-581.
10. Gage, F.H. (2000) Mammalian neural stem cells. Science, 287, 1433-1438.
11. Goldman, S.A. (1998) Adult neurogenesis: From canaries to the clinic. J. Neurobiol., 36, 267-286.
12. Keyoung, H.M., Roy, N.S., Benraiss, A., Louissaint, A., Jr, Suzuki, A., Hashimoto, M., Rashbaum, W.K., Okano, H. and Goldman, S.A. (2001) High-yield selection and extraction of two promoter-defined phenotypes of neural stem cells from the fetal human brain. Nat. Biotechnol., 19, 843-850.
13. Uchida, N., Buck, D.W., He, D., Reitsma, M.J., Masek, M., Phan, T.V., Tsukamoto, A.S., Gage, F.H. and Weissman, I.L. (2000) Direct isolation of human central nervous system stem cells. Proc. Natl Acad. Sci. USA 97, 14720-14725.
14. Arsenijevic, Y., Villemure, J.G., Brunet, J.F., Bloch, J.J., Deglon, N., Kostic, C., Zum, A. and Aebischer, P. (2001) Isolation of multipotent neural precursors residing in the cortex of the adult human brain. Exp. Neurol., 170, 48-62.
15. Pincus, D.W., Harrison-Restelli, C., Barry, J., Goodman, R.R., Fraser, R.A., Nedergaard, M. and Goldman, S.A. (1997) In vitro neurogenesis by adult human epileptic temporal neocortex. Clin. Neurosurg., 44, 17-25.
16. Pincus, D.W., Keyoung, H.M., Harrison-Restelli, C., Goodman, R.R., Fraser, R.A., Edgar, M., Sakakibara, S., Okano, H., Nedergaard, M. and Goldman, S.A. (1998) Fibroblast growth factor-2/brain-derived neurotrophic factor-associated maturation of new neurons generated from adult human subependymal cells. Ann. Neurol., 43, 576-585.
17. Roy, N.S., Benraiss, A., Wang, S., Fraser, R.A., Goodman, R., Couldwell, W.T., Nedergaard, M., Kawaguchi, A., Okano, H. and Goldman, S.A. (2000) Promoter-targeted selection and isolation of neural progenitor cells from the adult human ventricular zone. J. Neurosci. Res., 59 321-331.
18. Goldman, S.A. (2005) Stem and progenitor cell-based therapy of the human cental nervous system. Nat. Biotechnol., 23, 862-871.
19. Nunes, M.C., Roy, N.S., Keyoung, H.M., Goodman, R.R., McKhann, G., Jiang, L., Kang, J., Nedergaard, M. and Goldman, S.A. (2003) Identification and isolation of multipotential neural progenitor cells from the subcortical white matter of the adult human brain. Nat. Med. 9, 439-447.
20. Roy, N.S., Wang, S., Harrison-Restelli, C., Benraiss, A., Fraser, R.A., Gravel, M., Braun, P.E. and Goldman, S.A. (1999) Identification, isolation, and promoter-defined separation of mitotic oligodendrocyte progenitor cells from the adult human subcortical white matter. J. Neurosci., 19, 9986-9995.
21. Windrem, M.S., Nunes, M.C., Rashbaum, W.K., Schwartz, T.H., Goodman, R.A., McKhann, G., Roy, N.S. and Goldman, S.A. (2004) Fetal and adult human oligodendrocyte progenitor cell isolates myelinate the congenitally dysmyelinated brain. Nat. Med., 10, 93-97.
22. Belachew, S., Chittajallu, R., Aguirre, A.A., Yuan, X., Kirby, M., Anderson, S. and Gallo, V. (2003) Postnatal NG2 proteoglycan-expressing progenitor cells are intrinsically multipotent and generate functional neurons. J. Cell Biol., 161, 169-186.
23. Noble, M., Wren, D. and Wolswijk, G. (1992) The O-2A(adult) progenitor cell: A glial stem cell of the adult central nervous system. Semin. Cell Biol., 3, 413-422.
24. Archer, D., Cuddon, P., Lipsitz, D. and Duncan, I. (1997) Myelination of the canine central nervous system by glial cell transplantation: A model for repair of human myetin disease. Nat. Med., 3, 54-59.
25. Duncan, I.D., Grever, W.E. and Zhang, S.C. (1997) Repair of myelin disease: Strategies and progress in animal models. Mol. Med. Today 3, 554-561.
26. Windrem, M.S., Roy, N.S., Wang, J., Nunes, M., Benraiss, A., Goodman, R., McKhann, G.M. and Goldman, S.A. (2002) Progenitor cells derived from the adult human subcortical white matter disperse and differentiate as oligodendrocytes within demyelinated lesions of the rat brain. J. Neurosci. Res., 69, 966-975.
27. Lee, J.-P., Jeyakumar, M., Gonzalez, R., Takahashi, H., Lee, P., Park, K.-I., Butters, T., Dwek, R., Schwartz, P., Tong, G. et al. (2007) Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease. Nat. Med., 13, 439-447.
28. Snyder, E.Y., Taylor, R.M. and Wolfe, J.H. (1995) Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature, 374, 367-370.
29. Lachapelle, F., Gumpel, M., Baulac, M., Jacque, C., Duc, P. and Baumann, N. (1983) Transplantation of CNS fragments into the brain of shiverer mutant mice: Extensive myelination by implanted oligodendrocytes. I. Immunohistochemical studies. Dev. Neurosci., 6, 325-334.
30. Lachapelle, F., Gumpel, M. and Baumann, N. (1994) Contribution of transplantations to the understanding of the role of the PLP gene. Neurochem. Res., 19, 1083-1090.
31. Yandava, B.D., Billinghurst, L.L. and Snyder, E.Y. (1999) 'Global' cell replacement is feasible via neural stem cell hunsplantation: Evidence from the dysmyelinated shiverer mouse brain. Proc. Natl Acad. Sci. USA, 96, 7029-7034.
32. Mitome, M., Low, H.P., van Den Pol, A., Nunnari, J.J., Wolf, M.K., Billings-Gagliardi, S. and Schwartz, W.J. (2001) Towards the reconstruction of central nervous system white matter using neural precursor cells. Brain, 124, 2147-2161.
33. Roy, N., Windrem, M. and Goldman, S.A. (2004) Progenitor cells of the adult white matter. In Lazzarini, R. (ed.). Myelin Biology and Disorders, Elsevier, Amsterdam, pp. 259-287.
34. Windreni, M.S., Roy, N., Nunes, M. and Goldman, S.A. (2003) Identification, selection and use of adult human oligodendrocyte progenitor cells. In Zigova, T. and Snyder, E. (eds), Neural Stem Cells for Brain Repair, Humana, New York pp. 69-88.
35. Keyoung, H.M. and Goldman, S.A. (2007) Glial progenitor-based repair of demyelinating neurological diseases. Neurosurg. Clin. N. Am., 18, 93-104.
36. Suzuki, K. (2003) Globoid cell leukodystrophy (Krabbe's disease): update. J Child. Neurol., 18, 595-603.
37. Urayama, A., Grubb, J., Sly, W. and Banks, W. (2004) Developmentally regulated mannose 6-phophate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier. Proc. Natl Acad. Sci. USA, 101, 12658-12663.
38. Jeyakumar, M., Dwek, R., Butters, T. and Platt, F. (2005) Storage solutions: Treating lysosomal disorders of the brain. Nat. Rev. Neurosci., 6, 1-12.
39. Lacorazza, H.D., Flax, J.D., Snyder, E.Y. and Jendoubi, M. (1996) Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells. Nat. Med., 2, 424-429.
40. Pellegatta, S., Tunici, P., Poliani, P., Dolcetta, D., Cajola, L., Colombelli, C., Ciusani, E., DiDonato, S. and Finocchiaro, G. (2006) The therapeutic potential of neural stem/progenitor cells in murine globoid cell leukodystrophy is conditioned by macrophage/microglial activation. Neurobiol. Dis., 21, 314-323.
41. Pluchino, S., Furlan, R. and Martino, G. (2004) Cell-based remyelinating therapies in multiple sclerosis: Evidence from experimental studies. Curr. Opin. Neurol., 17, 247-255.
42. Pluchino, S., Quattrini, A., Brambilla, E., Gritti, A., Salani, G., Dina, G., Galli, R., Del Carro, U., Amadio, S., Bergami, A. et al. (2003) Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis. 422, 688-694.
43. Escolar, M., Poe, M., Provenzale, J., Richards, K., Allison, J., Wood, S., Wenger, D. and Kurtzberg, J. (2005) Transplantation of umbilical cord blood in babies with infantile Krabbe's disease. N. Engl. J. Med., 352, 2069-2081.
44. Koc, O., Day, J., Nieder, M., Gerson, S., Lazarus, H. and Krivit, W. (2002) Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy and Hurler syndrome (MPS-1H). Bone Marrow Transplant., 30, 215-222.
45. Givogri, M.I., Galbiati, F., Fasano, S., Amadio, S., Perani, L., Superchi, D., Morana, P., Del Carro, U., Marchesini, S., Brarabilla, R. et al. (2006) Oligodendroglial progenitor cell therapy limits central neurological deficits in mice with metachromatic leukodystrophy. J. Neurosci., 26, 3109-3119.
46. Nistor, G., Totoiu, M., Haque, N.S., Carpenter, M. and Kieirstead, H. (2005) Human embryonic stem cells differentiate into oligodendrocytes in high purity and myelinate after spinal cord contusion. Glia, 49 385-396.
47. Brustle, O., Jones, K.N., Learish, R.D., Karram, K., Choudhary, K., Wiestler, O.D., Duncan, I.D. and McKay, R.D. (1999) Embryonic stem cell-derived glial precursors: A source of myelinating transplants [Comments]. Science, 285, 754-756.
48. Roy, N.S., Cleren, C., Singh, S., Yang, L., Beal, M.F. and Goldman, S.A. (2006) Functional engraftment of human ES cell-derived dopaminergic neurons enriched by co-culture with telomerase-immortalized midbrain astrocytes. Nat. Med., 12, 1259-1268.