메뉴 건너뛰기




Volumn 8, Issue 4, 2011, Pages 659-667

Neural Stem Cell Transplantation as a Therapeutic Approach for Treating Lysosomal Storage Diseases

Author keywords

Cell transplantation; Lysosomal storage diseases; Neural progenitor cells; Neural stem cells; Niemann Pick A disease

Indexed keywords

LYSOSOME ENZYME; SPHINGOMYELIN PHOSPHODIESTERASE;

EID: 80955157861     PISSN: 19337213     EISSN: 18787479     Source Type: Journal    
DOI: 10.1007/s13311-011-0067-8     Document Type: Review
Times cited : (20)

References (77)
  • 1
    • 62949184826 scopus 로고    scopus 로고
    • Treating lysosomal storage disorders: current practice and future prospects
    • Platt F, Lachmann, RH. Treating lysosomal storage disorders: current practice and future prospects. Biochim Biophys Acta 2009; 1793: 737-345.
    • (2009) Biochim Biophys Acta , vol.1793 , pp. 345-737
    • Platt, F.1    Lachmann, R.H.2
  • 2
    • 0036895451 scopus 로고    scopus 로고
    • Enzyme replacement and enhancement therapies: lessons from lysosomal disorders
    • Desnick RJ, Schuchman EH. Enzyme replacement and enhancement therapies: lessons from lysosomal disorders. Nat Rev Genet 2002; 3: 954-966.
    • (2002) Nat Rev Genet , vol.3 , pp. 954-966
    • Desnick, R.J.1    Schuchman, E.H.2
  • 3
    • 0035499887 scopus 로고    scopus 로고
    • The development of neural stem cells
    • Temple S. The development of neural stem cells. Nature 2001; 414: 112-117.
    • (2001) Nature , vol.414 , pp. 112-117
    • Temple, S.1
  • 4
    • 0034712047 scopus 로고    scopus 로고
    • Mammalian neural stem cells
    • Gage FH. Mammalian neural stem cells. Science 2000; 287: 1433-1438.
    • (2000) Science , vol.287 , pp. 1433-1438
    • Gage, F.H.1
  • 5
    • 0028908299 scopus 로고
    • Isolation, characterization, and use of stem cells from the CNS
    • Gage FH, Ray J, Fisher LJ. Isolation, characterization, and use of stem cells from the CNS. Annu Rev Neurosci 1995; 18: 159-192.
    • (1995) Annu Rev Neurosci , vol.18 , pp. 159-192
    • Gage, F.H.1    Ray, J.2    Fisher, L.J.3
  • 6
    • 0030894092 scopus 로고    scopus 로고
    • Stem cells in the central nervous system
    • McKay R. Stem cells in the central nervous system. Science 1997; 276: 66-71.
    • (1997) Science , vol.276 , pp. 66-71
    • McKay, R.1
  • 8
    • 17844394405 scopus 로고    scopus 로고
    • Neural stem cells and their use as therapeutic tool in neurological disorders
    • Pluchino S, Zanotti L, Deleidi M, Martino G. Neural stem cells and their use as therapeutic tool in neurological disorders. Brain Res Brain Res Rev 2005; 48: 211-219.
    • (2005) Brain Res Brain Res Rev , vol.48 , pp. 211-219
    • Pluchino, S.1    Zanotti, L.2    Deleidi, M.3    Martino, G.4
  • 10
    • 0033044937 scopus 로고    scopus 로고
    • Establishment and properties of neural stem cell clones: plasticity in vitro and in vivo
    • Vescovi A, Snyder EY. Establishment and properties of neural stem cell clones: plasticity in vitro and in vivo. Brain Pathol 1999; 9: 569-598.
    • (1999) Brain Pathol , vol.9 , pp. 569-598
    • Vescovi, A.1    Snyder, E.Y.2
  • 11
    • 0033044936 scopus 로고    scopus 로고
    • Human neural stem cells: isolation, expansion and transplantation
    • Svendsen C, Caldwell MA, Ostenfeld T. Human neural stem cells: isolation, expansion and transplantation. Brain Pathol 1999; 9: 499-513.
    • (1999) Brain Pathol , vol.9 , pp. 499-513
    • Svendsen, C.1    Caldwell, M.A.2    Ostenfeld, T.3
  • 12
    • 0034687840 scopus 로고    scopus 로고
    • Direct isolation of human central nervous system stem cells
    • Uchida N, Buck DW, He D, et al. Direct isolation of human central nervous system stem cells. Proc Natl Acad Sci U S A 2000; 97: 14720-14725.
    • (2000) Proc Natl Acad Sci U S A , vol.97 , pp. 14720-14725
    • Uchida, N.1    Buck, D.W.2    He, D.3
  • 14
    • 0344549791 scopus 로고    scopus 로고
    • Isolation and characterization of neural progenitor cells from post-mortem human cortex
    • Schwartz P, Bryant PJ, Fuja TJ, Su H, O'Dowd DK, Klassen H. Isolation and characterization of neural progenitor cells from post-mortem human cortex. J Neurosci Res 2003; 74: 838-851.
    • (2003) J Neurosci Res , vol.74 , pp. 838-851
    • Schwartz, P.1    Bryant, P.J.2    Fuja, T.J.3    Su, H.4    O'Dowd, D.K.5    Klassen, H.6
  • 15
    • 0029860591 scopus 로고    scopus 로고
    • Multipotent CNS stem cells are present in the adult mammalian spinal cord and ventricular neuroaxis
    • Weiss S, Dunne C, Hewson J, et al. Multipotent CNS stem cells are present in the adult mammalian spinal cord and ventricular neuroaxis. J Neurosci 1996; 16: 7599-7609.
    • (1996) J Neurosci , vol.16 , pp. 7599-7609
    • Weiss, S.1    Dunne, C.2    Hewson, J.3
  • 16
    • 0029610078 scopus 로고
    • Survival and differentiation of adult neuronal progenitor cells transplanted to the adult brain
    • Gage FH, Coates PW, Palmer TD, et al. Survival and differentiation of adult neuronal progenitor cells transplanted to the adult brain. Proc Natl Acad Sci U S A 1995; 92: 11879-11883.
    • (1995) Proc Natl Acad Sci U S A , vol.92 , pp. 11879-11883
    • Gage, F.H.1    Coates, P.W.2    Palmer, T.D.3
  • 17
    • 0029018239 scopus 로고
    • Cloned multipotential precursors from the mouse cerebrum require FGF-2, whereas glial restricted precursors are stimulated with either FGF-2 or EGF
    • Kilpatrick T, Bartlett PF. Cloned multipotential precursors from the mouse cerebrum require FGF-2, whereas glial restricted precursors are stimulated with either FGF-2 or EGF. J Neurosci 1995; 15: 3653-3661.
    • (1995) J Neurosci , vol.15 , pp. 3653-3661
    • Kilpatrick, T.1    Bartlett, P.F.2
  • 18
    • 0029971286 scopus 로고    scopus 로고
    • Multipotential stem cells from the adult mouse brain proliferate and self-renew in response to basic fibroblast growth factor
    • Gritti A, Parati EA, Cova L, et al. Multipotential stem cells from the adult mouse brain proliferate and self-renew in response to basic fibroblast growth factor. J Neurosci 1996; 16: 1091-1100.
    • (1996) J Neurosci , vol.16 , pp. 1091-1100
    • Gritti, A.1    Parati, E.A.2    Cova, L.3
  • 19
    • 0031771599 scopus 로고    scopus 로고
    • Engraftable human neural stem cells respond to developmental cues, replace neurons, and express foreign genes
    • Flax JD, Aurora S, Yang C, et al. Engraftable human neural stem cells respond to developmental cues, replace neurons, and express foreign genes. Nature Biotech 1998; 16: 1033-1039.
    • (1998) Nature Biotech , vol.16 , pp. 1033-1039
    • Flax, J.D.1    Aurora, S.2    Yang, C.3
  • 20
    • 0030079438 scopus 로고    scopus 로고
    • Physiological relevance and functional potential of central nervous system-derived cell lines
    • Whittemore S, Snyder EY. Physiological relevance and functional potential of central nervous system-derived cell lines. Mol Neurobiol 1996; 12: 13-38.
    • (1996) Mol Neurobiol , vol.12 , pp. 13-38
    • Whittemore, S.1    Snyder, E.Y.2
  • 21
    • 0033929434 scopus 로고    scopus 로고
    • Human neural precursor cells express low levels of telomerase in vitro and show diminishing cell proliferation with extensive axonal outgrowth following transplantation
    • Ostenfeld T, Caldwell MA, Prowse KR, Linskens MH, Jauniaux E, Svendsen CN. Human neural precursor cells express low levels of telomerase in vitro and show diminishing cell proliferation with extensive axonal outgrowth following transplantation. Exp Neurol 2000; 164: 215-226.
    • (2000) Exp Neurol , vol.164 , pp. 215-226
    • Ostenfeld, T.1    Caldwell, M.A.2    Prowse, K.R.3    Linskens, M.H.4    Jauniaux, E.5    Svendsen, C.N.6
  • 22
    • 33644823573 scopus 로고    scopus 로고
    • Differential properties of adult rat and mouse brain-derived neural stem/progenitor cells
    • Ray J, Gage FH. Differential properties of adult rat and mouse brain-derived neural stem/progenitor cells. Mol Cell Neurosci 2006; 31: 560-573.
    • (2006) Mol Cell Neurosci , vol.31 , pp. 560-573
    • Ray, J.1    Gage, F.H.2
  • 23
    • 0035453310 scopus 로고    scopus 로고
    • Human neural stem and progenitor cells: in vitro and in vivo properties, and potential for gene therapy and cell replacement in the CNS
    • Martinez-Serrano A, Rubio FJ, Navarro B, Bueno C, Villa A. Human neural stem and progenitor cells: in vitro and in vivo properties, and potential for gene therapy and cell replacement in the CNS. Curr Gene Ther 2001; 1: 279-299.
    • (2001) Curr Gene Ther , vol.1 , pp. 279-299
    • Martinez-Serrano, A.1    Rubio, F.J.2    Navarro, B.3    Bueno, C.4    Villa, A.5
  • 24
    • 0037106091 scopus 로고    scopus 로고
    • Engraftment of sorted/expanded human central nervous system stem cells from fetal brain
    • Tamaki S, Eckert K, He D, et al. Engraftment of sorted/expanded human central nervous system stem cells from fetal brain. J Neurosci Res 2002; 69: 976-986.
    • (2002) J Neurosci Res , vol.69 , pp. 976-986
    • Tamaki, S.1    Eckert, K.2    He, D.3
  • 25
    • 0032491416 scopus 로고    scopus 로고
    • Embryonic stem cell lines derived from human blastocysts
    • Thomson JA, Itskovitz-Eldor J, Shapiro SS, et al. Embryonic stem cell lines derived from human blastocysts. Science 1998; 282: 1145-1147.
    • (1998) Science , vol.282 , pp. 1145-1147
    • Thomson, J.A.1    Itskovitz-Eldor, J.2    Shapiro, S.S.3
  • 27
    • 32344438401 scopus 로고    scopus 로고
    • Derivation of human embryonic stem cells in defined conditions
    • Ludwig TE, Levenstein ME, Jones JM, et al. Derivation of human embryonic stem cells in defined conditions. Nat Biotechnol 2006; 24: 185-187.
    • (2006) Nat Biotechnol , vol.24 , pp. 185-187
    • Ludwig, T.E.1    Levenstein, M.E.2    Jones, J.M.3
  • 28
    • 24944484633 scopus 로고    scopus 로고
    • Niche-independent symmetrical self-renewal of a mammalian tissue stem cell
    • Conti L, Pollard SM, Gorba T, et al. Niche-independent symmetrical self-renewal of a mammalian tissue stem cell. PLoS Biol 2005; 3: 1594-1606.
    • (2005) PLoS Biol , vol.3
    • Conti, L.1    Pollard, S.M.2    Gorba, T.3
  • 29
    • 0036454328 scopus 로고    scopus 로고
    • Repairing the damaged spinal cord: a summary of our early success with embryonic stem cell transplantation and remyelination
    • McDonald JW, Howard MJ. Repairing the damaged spinal cord: a summary of our early success with embryonic stem cell transplantation and remyelination. Prog Brain Res 2002; 137: 299-309.
    • (2002) Prog Brain Res , vol.137 , pp. 299-309
    • McDonald, J.W.1    Howard, M.J.2
  • 30
    • 13544264789 scopus 로고    scopus 로고
    • Human embryonic stem cells differentiate into oligodendrocytes in high purity and myelinate after spinal cord transplantation
    • Nistor GI, Totoiu MO, Haque N, Carpenter MK, Keirstead HS. Human embryonic stem cells differentiate into oligodendrocytes in high purity and myelinate after spinal cord transplantation. Glia 2005; 49: 385-396.
    • (2005) Glia , vol.49 , pp. 385-396
    • Nistor, G.I.1    Totoiu, M.O.2    Haque, N.3    Carpenter, M.K.4    Keirstead, H.S.5
  • 31
    • 20644447816 scopus 로고    scopus 로고
    • Cell biology. Ready or not? Human ES cells head toward the clinic
    • Vogel G. Cell biology. Ready or not? Human ES cells head toward the clinic. Science 2005; 308: 1534-1538.
    • (2005) Science , vol.308 , pp. 1534-1538
    • Vogel, G.1
  • 32
    • 62149089434 scopus 로고    scopus 로고
    • Geron gets green light for human trial of ES cell-derived product
    • Alper J. Geron gets green light for human trial of ES cell-derived product. Nat Biotechnol 2009; 27: 213-214.
    • (2009) Nat Biotechnol , vol.27 , pp. 213-214
    • Alper, J.1
  • 33
    • 36248966518 scopus 로고    scopus 로고
    • Induction of pluripotent stem cells from adult human fibroblasts by defined factors
    • Takahashi K, Tanabe K, Ohnuki M, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007; 131: 861-872.
    • (2007) Cell , vol.131 , pp. 861-872
    • Takahashi, K.1    Tanabe, K.2    Ohnuki, M.3
  • 34
    • 36749043230 scopus 로고    scopus 로고
    • Induced pluripotent stem cell lines derived from human somatic cells
    • Yu J, Vodyanik MA, Smuga-Otto K, et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007; 318: 1917-1920.
    • (2007) Science , vol.318 , pp. 1917-1920
    • Yu, J.1    Vodyanik, M.A.2    Smuga-Otto, K.3
  • 35
    • 38049187707 scopus 로고    scopus 로고
    • Reprogramming of human somatic cells to pluripotency with defined factors
    • Park IH, Zhao R, West JA, et al. Reprogramming of human somatic cells to pluripotency with defined factors. Nature 2008; 451: 141-146.
    • (2008) Nature , vol.451 , pp. 141-146
    • Park, I.H.1    Zhao, R.2    West, J.A.3
  • 36
    • 77954784173 scopus 로고    scopus 로고
    • Back to immortality: the restoration of embryonic telomere length during induced pluripotency
    • West MD, Vaziri H. Back to immortality: the restoration of embryonic telomere length during induced pluripotency. Regen Med 2010; 5: 485-488.
    • (2010) Regen Med , vol.5 , pp. 485-488
    • West, M.D.1    Vaziri, H.2
  • 37
    • 79956357347 scopus 로고    scopus 로고
    • Direct reprogramming of mouse fibroblasts to neural progenitors
    • Kim J, Efe JA, Zhu S, et al. Direct reprogramming of mouse fibroblasts to neural progenitors. Proc Natl Acad Sci U S A 2011; 108: 7838-7843.
    • (2011) Proc Natl Acad Sci U S A , vol.108 , pp. 7838-7843
    • Kim, J.1    Efe, J.A.2    Zhu, S.3
  • 38
    • 66749175714 scopus 로고    scopus 로고
    • Transdetermination: a new trend in cellular reprogramming
    • Manohar R, Lagasse E. Transdetermination: a new trend in cellular reprogramming. Mol Ther 2009; 17: 936-938.
    • (2009) Mol Ther , vol.17 , pp. 936-938
    • Manohar, R.1    Lagasse, E.2
  • 40
    • 4444363727 scopus 로고    scopus 로고
    • Neurospheres induced from bone marrow stromal cells are multipotent for differentiation into neuron, astrocyte, and oligodendrocyte phenotypes
    • Suzuki H, Taguchi T, Tanaka H, et al. Neurospheres induced from bone marrow stromal cells are multipotent for differentiation into neuron, astrocyte, and oligodendrocyte phenotypes. Biochem Biophys Res Commun 2004; 322: 918-1022.
    • (2004) Biochem Biophys Res Commun , vol.322 , pp. 918-1022
    • Suzuki, H.1    Taguchi, T.2    Tanaka, H.3
  • 41
    • 33645462716 scopus 로고    scopus 로고
    • Neural transdifferentiation of mesenchymal stem cells - a critical review
    • Krabbe C, Zimmer J, Meyer M. Neural transdifferentiation of mesenchymal stem cells - a critical review. APMIS 2005; 113: 831-844.
    • (2005) Apmis , vol.113 , pp. 831-844
    • Krabbe, C.1    Zimmer, J.2    Meyer, M.3
  • 42
    • 3242755834 scopus 로고    scopus 로고
    • A new human somatic stem cell from placental cord blood with intrinsic pluripotent differentiation potential
    • Kögler G, Sensken S, Airey JA, et al. A new human somatic stem cell from placental cord blood with intrinsic pluripotent differentiation potential. J Exp Med 2004; 200: 123-135.
    • (2004) J Exp Med , vol.200 , pp. 123-135
    • Kögler, G.1    Sensken, S.2    Airey, J.A.3
  • 43
    • 33846406448 scopus 로고    scopus 로고
    • Stem cells in the umbilical cord
    • Weiss ML, Troyer DL. Stem cells in the umbilical cord. Stem Cell Rev 2006; 2: 155-162.
    • (2006) Stem Cell Rev , vol.2 , pp. 155-162
    • Weiss, M.L.1    Troyer, D.L.2
  • 44
    • 33144474684 scopus 로고    scopus 로고
    • Clonal amniotic fluid-derived stem cells express characteristics of both mesenchymal and neural stem cells
    • Tsai MS, Hwang SM, Tsai YL, Cheng FC, Lee, JL, Chang YJ. Clonal amniotic fluid-derived stem cells express characteristics of both mesenchymal and neural stem cells. Biol Reprod 2006; 74: 545-551.
    • (2006) Biol Reprod , vol.74 , pp. 545-551
    • Tsai, M.S.1    Hwang, S.M.2    Tsai, Y.L.3    Cheng, F.C.4    Lee, J.L.5    Chang, Y.J.6
  • 45
    • 3042753986 scopus 로고    scopus 로고
    • Efficient generation of neural precursors from adult human skin: astrocytes promote neurogenesis from skin-derived stem cells
    • Joannides A, Gaughwin P, Schwiening C, et al. Efficient generation of neural precursors from adult human skin: astrocytes promote neurogenesis from skin-derived stem cells. Lancet 2004; 364: 172-178.
    • (2004) Lancet , vol.364 , pp. 172-178
    • Joannides, A.1    Gaughwin, P.2    Schwiening, C.3
  • 46
    • 2342491483 scopus 로고    scopus 로고
    • Characterization of neuronal/glial differentiation of murine adipose-derived adult stromal cells
    • Safford KM, Safford SD, Gimble JM, Shetty, AK, Rice HE. Characterization of neuronal/glial differentiation of murine adipose-derived adult stromal cells. Exp Neurol 2004; 187: 319-328.
    • (2004) Exp Neurol , vol.187 , pp. 319-328
    • Safford, K.M.1    Safford, S.D.2    Gimble, J.M.3    Shetty, A.K.4    Rice, H.E.5
  • 48
    • 0033565709 scopus 로고    scopus 로고
    • Site-specific migration and neuronal differentiation of human neural progenitor cells after transplantation in the adult rat brain
    • Fricker RA, Carpenter MK, Winkler C, Greco C, Gates MA, Bjorklund A. Site-specific migration and neuronal differentiation of human neural progenitor cells after transplantation in the adult rat brain. J Neurosci 1999; 19: 5990-6005.
    • (1999) J Neurosci , vol.19 , pp. 5990-6005
    • Fricker, R.A.1    Carpenter, M.K.2    Winkler, C.3    Greco, C.4    Gates, M.A.5    Bjorklund, A.6
  • 50
    • 0037197831 scopus 로고    scopus 로고
    • Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier
    • Sly WS, Vogler C. Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier. Proc Natl Acad Sci U S A 2002; 99: 5760-5762.
    • (2002) Proc Natl Acad Sci U S A , vol.99 , pp. 5760-5762
    • Sly, W.S.1    Vogler, C.2
  • 51
    • 0015231036 scopus 로고
    • Correction of abnormal cerebroside sulfate metabolism in cultured metachromatic leukodystrophy fibroblasts
    • Porter MT, Fluharty AL, Kihara H. Correction of abnormal cerebroside sulfate metabolism in cultured metachromatic leukodystrophy fibroblasts. Science 1971; 172: 1263-1265.
    • (1971) Science , vol.172 , pp. 1263-1265
    • Porter, M.T.1    Fluharty, A.L.2    Kihara, H.3
  • 52
    • 0015794917 scopus 로고
    • Sanfilippo disease type B: enzyme replacement and metabolic correction in cultured fibroblasts
    • O'Brien JS, Miller AL, Loverde AW, Veath ML. Sanfilippo disease type B: enzyme replacement and metabolic correction in cultured fibroblasts. Science 1973; 181: 753-755.
    • (1973) Science , vol.181 , pp. 753-755
    • O'Brien, J.S.1    Miller, A.L.2    Loverde, A.W.3    Veath, M.L.4
  • 53
    • 5744228699 scopus 로고    scopus 로고
    • Enzyme replacement therapy
    • F. M. Platt and S. U. Walkely (Eds.), Oxford: Oxford University Press
    • Neufeld EF. Enzyme replacement therapy. In: Platt FM, Walkely SU, ed. Lysosomal disorders of the brain. Oxford: Oxford University Press, 2004: 327-338.
    • (2004) Lysosomal Disorders of the Brain , pp. 327-338
    • Neufeld, E.F.1
  • 54
    • 0030854815 scopus 로고    scopus 로고
    • Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase
    • Taylor RM, Wolfe JH. Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase. Nat Med 1997; 3: 771-774.
    • (1997) Nat Med , vol.3 , pp. 771-774
    • Taylor, R.M.1    Wolfe, J.H.2
  • 55
    • 0036256645 scopus 로고    scopus 로고
    • Intracerebral transplantation of mesenchymal stem cells into acid sphingomyelinase-deficient mice delays the onset of neurological abnormalities and extends their life span
    • Jin HK, Carter JE, Huntley GW, Schuchman EH. Intracerebral transplantation of mesenchymal stem cells into acid sphingomyelinase-deficient mice delays the onset of neurological abnormalities and extends their life span. J Clin Invest 2002; 109: 1183-1191.
    • (2002) J Clin Invest , vol.109 , pp. 1183-1191
    • Jin, H.K.1    Carter, J.E.2    Huntley, G.W.3    Schuchman, E.H.4
  • 56
    • 0036212994 scopus 로고    scopus 로고
    • Long-term expression of beta-glucuronidase by genetically modified human neural progenitor cells grafted into the mouse central nervous system
    • Buchet D, Serguera C, Zennou V, Charneau P, Mallet J. Long-term expression of beta-glucuronidase by genetically modified human neural progenitor cells grafted into the mouse central nervous system. Mol Cell Neurosci 2002; 19: 389-401.
    • (2002) Mol Cell Neurosci , vol.19 , pp. 389-401
    • Buchet, D.1    Serguera, C.2    Zennou, V.3    Charneau, P.4    Mallet, J.5
  • 57
    • 0141705207 scopus 로고    scopus 로고
    • Brain transplantation of genetically engineered human neural stem cells globally corrects brain lesions in the mucopolysaccharidosis type VII mouse
    • Meng XL, Shen JS, Ohashi T, Maeda H, Kim SU, Eto Y. Brain transplantation of genetically engineered human neural stem cells globally corrects brain lesions in the mucopolysaccharidosis type VII mouse. J Neurosci Res 2003; 74: 266-277.
    • (2003) J Neurosci Res , vol.74 , pp. 266-277
    • Meng, X.L.1    Shen, J.S.2    Ohashi, T.3    Maeda, H.4    Kim, S.U.5    Eto, Y.6
  • 58
    • 9344260848 scopus 로고    scopus 로고
    • Intracerebral transplantation of adult mouse neural progenitor cells into the Niemann-Pick-A mouse leads to a marked decrease in lysosomal storage pathology
    • Shihabuddin LS, Numan S, Huff MR, et al. Intracerebral transplantation of adult mouse neural progenitor cells into the Niemann-Pick-A mouse leads to a marked decrease in lysosomal storage pathology. J Neurosci 2004; 24: 10642-10651.
    • (2004) J Neurosci , vol.24 , pp. 10642-10651
    • Shihabuddin, L.S.1    Numan, S.2    Huff, M.R.3
  • 59
    • 11144354757 scopus 로고    scopus 로고
    • Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells
    • Biffi A, De Palma M, Quattrini A, et al. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. J Clin Invest 2004; 113: 1118-1129.
    • (2004) J Clin Invest , vol.113 , pp. 1118-1129
    • Biffi, A.1    de Palma, M.2    Quattrini, A.3
  • 60
    • 0028951119 scopus 로고
    • Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain
    • Snyder EY, Taylor RM, Wolfe JH. Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature 1995; 374: 367-370.
    • (1995) Nature , vol.374 , pp. 367-370
    • Snyder, E.Y.1    Taylor, R.M.2    Wolfe, J.H.3
  • 61
    • 0029898457 scopus 로고    scopus 로고
    • Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells
    • Lacorazza HD, Flax JD, Snyder EY, Jendoubi M. 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 1996; 2: 424-429.
    • (1996) Nat Med , vol.2 , pp. 424-429
    • Lacorazza, H.D.1    Flax, J.D.2    Snyder, E.Y.3    Jendoubi, M.4
  • 62
    • 34147160607 scopus 로고    scopus 로고
    • Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease
    • Lee JP, Jeyakumar M, Gonzalez R, et al. Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease. Nat Med 2007; 13: 439-447.
    • (2007) Nat Med , vol.13 , pp. 439-447
    • Lee, J.P.1    Jeyakumar, M.2    Gonzalez, R.3
  • 63
    • 70349869180 scopus 로고    scopus 로고
    • Neural stem cell transplantation benefits a monogenic neurometabolic disorder during the symptomatic phase of disease
    • Jeyakumar M, Lee JP, Sibson NR, et al. Neural stem cell transplantation benefits a monogenic neurometabolic disorder during the symptomatic phase of disease. Stem Cells 2009; 27: 2362-2370.
    • (2009) Stem Cells , vol.27 , pp. 2362-2370
    • Jeyakumar, M.1    Lee, J.P.2    Sibson, N.R.3
  • 64
    • 69449089145 scopus 로고    scopus 로고
    • Neuroprotection of host cells by human central nervous system stem cells in a mouse model of infantile neuronal ceroid lipofuscinosis
    • Tamaki SJ, Jacobs Y, Dohse M, et al. Neuroprotection of host cells by human central nervous system stem cells in a mouse model of infantile neuronal ceroid lipofuscinosis. Cell Stem Cell 2009; 5: 310-319.
    • (2009) Cell Stem Cell , vol.5 , pp. 310-319
    • Tamaki, S.J.1    Jacobs, Y.2    Dohse, M.3
  • 65
    • 0035976969 scopus 로고    scopus 로고
    • Acid sphingomyelinase-deficient macrophages have defective cholesterol trafficking and efflux
    • Leventhal A, Chen W, Tall AR, Tabas I. Acid sphingomyelinase-deficient macrophages have defective cholesterol trafficking and efflux. J Biol Chem 2001; 276: 44976-44983.
    • (2001) J Biol Chem , vol.276 , pp. 44976-44983
    • Leventhal, A.1    Chen, W.2    Tall, A.R.3    Tabas, I.4
  • 66
    • 0032840719 scopus 로고    scopus 로고
    • Sphingomyelin-cholesterol interactions in biological and model membranes
    • Slotte J. Sphingomyelin-cholesterol interactions in biological and model membranes. Chem Phys Lipids 1999; 102: 13-27.
    • (1999) Chem Phys Lipids , vol.102 , pp. 13-27
    • Slotte, J.1
  • 67
    • 0025368184 scopus 로고
    • Very low levels of high density lipoprotein cholesterol in four sibs of a family with non-neuropathic Niemann-Pick disease and sea-blue histiocytosis
    • Viana M, Giugliani R, Leite VH, et al. Very low levels of high density lipoprotein cholesterol in four sibs of a family with non-neuropathic Niemann-Pick disease and sea-blue histiocytosis. J Med Genet 1990; 27: 499-504.
    • (1990) J Med Genet , vol.27 , pp. 499-504
    • Viana, M.1    Giugliani, R.2    Leite, V.H.3
  • 68
    • 0034976568 scopus 로고    scopus 로고
    • Patterned cerebellar Purkinje cell death in a transgenic mouse model of Niemann Pick type A/B disease
    • Sarna J, Miranda SR, Schuchman EH, Hawkes R. Patterned cerebellar Purkinje cell death in a transgenic mouse model of Niemann Pick type A/B disease. Eur J Neurosci 2001; 13: 1873-1880.
    • (2001) Eur J Neurosci , vol.13 , pp. 1873-1880
    • Sarna, J.1    Miranda, S.R.2    Schuchman, E.H.3    Hawkes, R.4
  • 69
    • 0032570597 scopus 로고    scopus 로고
    • Lysenin, a novel sphingomyelin-specific binding protein
    • Yamaji A, Sekizawa Y, Emoto K, et al. Lysenin, a novel sphingomyelin-specific binding protein. J Biol Chem 1998; 273: 5300-5306.
    • (1998) J Biol Chem , vol.273 , pp. 5300-5306
    • Yamaji, A.1    Sekizawa, Y.2    Emoto, K.3
  • 70
    • 0026572112 scopus 로고
    • Quantitative correlation between the residual activity of beta-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease
    • Leinekugel P, Michel S, Conzelmann E, Sandhoff K. Quantitative correlation between the residual activity of beta-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease. Hum Genet 1992; 88: 513-523.
    • (1992) Hum Genet , vol.88 , pp. 513-523
    • Leinekugel, P.1    Michel, S.2    Conzelmann, E.3    Sandhoff, K.4
  • 71
    • 33847681505 scopus 로고    scopus 로고
    • Injection of mouse and human neural stem cells into neonatal Niemann-Pick A model mice
    • Sidman R, Li J, Stewart GR, et al. Injection of mouse and human neural stem cells into neonatal Niemann-Pick A model mice. Brain Res 2006; 1140: 195-204.
    • (2006) Brain Res , vol.1140 , pp. 195-204
    • Sidman, R.1    Li, J.2    Stewart, G.R.3
  • 72
    • 33751019158 scopus 로고    scopus 로고
    • Neuronal ceroid lipofuscinoses therapeutic strategies: past, present and future
    • Hobert J, Dawson G. Neuronal ceroid lipofuscinoses therapeutic strategies: past, present and future. Biochim Biophys Acta 2006; 1762: 945-953.
    • (2006) Biochim Biophys Acta , vol.1762 , pp. 945-953
    • Hobert, J.1    Dawson, G.2
  • 73
    • 33645701362 scopus 로고    scopus 로고
    • HuCNS-SC (StemCells)
    • Taupin P. HuCNS-SC (StemCells). Curr Opin Mol Ther 2006; 8: 156-163.
    • (2006) Curr Opin Mol Ther , vol.8 , pp. 156-163
    • Taupin, P.1
  • 74
    • 44349136847 scopus 로고    scopus 로고
    • Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA
    • Worgall S, Sondhi D, Hackett NR, et al. Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther 2008; 19: 463-374.
    • (2008) Hum Gene Ther , vol.19 , pp. 374-463
    • Worgall, S.1    Sondhi, D.2    Hackett, N.R.3
  • 75
    • 69249220421 scopus 로고    scopus 로고
    • Stem cell-based cell therapy in neurological diseases: a review
    • Kim SU, de Vellis, J. Stem cell-based cell therapy in neurological diseases: a review. J Neurosci Res 2009; 87: 2183-2200.
    • (2009) J Neurosci Res , vol.87 , pp. 2183-2200
    • Kim, S.U.1    de Vellis, J.2
  • 76
    • 47749105005 scopus 로고    scopus 로고
    • Stem cell therapies for the lysosomal storage diseases - the quintessential neurodegenerative diseases
    • Schwartz PH, Brick DJ. Stem cell therapies for the lysosomal storage diseases - the quintessential neurodegenerative diseases. Curr Stem Cell Res Ther 2008; 3: 88-98.
    • (2008) Curr Stem Cell Res Ther , vol.3 , pp. 88-98
    • Schwartz, P.H.1    Brick, D.J.2
  • 77
    • 42149113096 scopus 로고    scopus 로고
    • Central nervous system therapy for lysosomal storage disorders
    • Enns GM, Huhn, SL. Central nervous system therapy for lysosomal storage disorders. Neurosurg Focus 2008; 24: 1-12.
    • (2008) Neurosurg Focus , vol.24 , pp. 1-12
    • Enns, G.M.1    Huhn, S.L.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.