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Marshak DR, Gardner RL, Gottlieb D. 2001. Stem cell biology. Cold Spiring
Harbor, NY: Cold Spiring Harbor Laboratory Press.
Taman K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, Yamanaka S. 2008.
Induction of pluripotent stem cells from adult human fibroblasts by defined
factors. Cell 131:861–872.
Kim SU. 2004. Human neural stem cells genetically modified for brain repair in
neurological disorders. Neuropathology 24:159–174.
Eriksson PS, Perfilieva E, Bjo ¨rk-Eriksson T, Alborn AM, Nordborg C, Peterson DA,
Gage FH. 1998. Neurogenesis in the adult human hippo- campus. Nat Med
4:1313–1317.
Kim SU, Nagai A, Nakagawa E, Choi HB, Bang JH, Lee HJ, Lee MA, Lee YB, Park IH.
2008b. Production and characterization of immortal human neural stem cell line
with multipotent differentiation property. Methods Mol Biol 438:103–121.
Lee HJ, Kim KS, Kim EJ, Choi HB, Lee KH, Park IH, Ko Y, Jeong SW, Kim SU. 2007a.
Brain transplantation of human neural stem cells promotes functional recovery in
mouse intracerebral hemorrhage stroke model. Stem Cells 25:211–224.
Goldman S. 2005. Stem and progenitor cell-based therapy of the human central
nervous system. Nat Biotechnol 7:862–871.
Agid Y. 1991. Parkinson’s disease: pathophysiology. Lancet 337:1321– 1324.
Lang AE, Lozano AM. 1998b. Parkinson’s disease. Second of two parts. N Engl J
Med 339:1130–1143.
Dunnett SB, Bjorklund A. 1999. Prospects for new restorative and neuroprotective treatments in Parkinson’s disease. Nature 399:A32–A39.
Hagell P, Schrag A, Piccini P, Jahanshahi M, Brown R, Rehncrona S, Widner H,
Brundin P, Rothwell JC, Odin P, Wenning GK, Morrish P, Gustavii B, Bjo ¨rklund A,
Brooks DJ, Marsden CD, Quinn NP, Lindvall O. 1999. Sequential bilateral
transplantation in Parkinson’s dis- ease: effects of the second graft. Brain
122:1121–1132.
Shim JW, Koh HC, Chang MY, Roh E, Choi CY, Oh YJ, Son H, Lee YS, Studer L, Lee
SH. 2004. Enhanced in vitro midbrain dopamine neuron differentiation,
dopaminergic function, neurite outgrowth, and 1-methyl-4-phenylpyridium
resistance in mouse embryonic stem cells overexpressing Bcl-xL. J Neurosci
24:843–852.
Kawasaki H, Mizuseki K, Nishikawa S, Kaneko S, Kuwana Y, Nakanishi S,
Nishikawa SI, Sasai Y. 2000. Induction of midbrain dopaminergic neurons from ES
cells by stromal cell-derived inducing activity. Neuron 28:31–40.
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 ES cells function in a Parkinson primate model. J
Clin Invest 115:102–108.
Redmond DE Jr, Bjugstad KB, Teng YD, Ourednik V, Ourednik J, Wakeman DR,
Parsons XH, Gonzalez R, Blanchard BC, Kim SU, Gu Z, Lipton SA, Markakis EA,
Roth RH, Elsworth JD, Sladek JR Jr, Sidman RL, Snyder EY. 2007. Behavioral
improvement in a primate Parkinson’s model is associated with multiple
homeostatic effects of human neural stem cells. Proc Natl Acad Sci USA
104:12175–12180.
Kang UJ, Fisher LJ, Joh TH, O’Malley KL, Gage FH. 1993. Regulation of dopamine
production by genetically modified primary fibroblasts. J Neurosci 13:5203–5211.
Harper PS. 1996. Huntington’s disease. Philadelphia: W.B. Saunders.
Bachoud-Le ´vi AC, Re ´my P, Nguyen JP, Brugie `res P, Lefaucheur JP, Bourdet C,
Baudic S, Gaura V, Maison P, Haddad B, Boisse ´ MF, Grandmougin T, Je ´ny R,
Bartolomeo P, Dalla Barba G, Degos JD, Lisovoski F, Ergis AM, Pailhous E, Cesaro
P, Hantraye P, Peschanski M. 2000. Motor and cognitive improvements in
patients with Hunting- ton’s disease after neural transplnatation. Lancet
356:1975–1979.
Freeman TB, Cicchetti F, Hauser RA, Deacon TW, Li XJ, Hersch SM, Nauert GM,
Sanberg PR, Kordower JH, Saporta S, Isacson O. 2000. Transplanted fetal striatum
in Huntington’s disease: phenotypic devel- opment and lack of pathology. Proc
Natl Acad Sci USA 97:13877– 13882.
Huntington’s Disease Collaborative Research Group. 1993. A novel gene
containing a trinucleotide repeat that is expanded and unstable on Hun- tington’s
disease chromosomes. Cell 72:971–983.
Roberts TJ, Price J, Williams SC, Modo M. 2006. Preservation of striatal tissue and
behavioral function after neural stem cell transplantation in a rat model of
Huntington’s disease. Neuroscience 139:1187–1199.
Ryu JK, Kim J, Cho SJ, Hatori K, Nagai A, Choi HB, Lee MC, McLar- non JG, Kim SU.
2004. Proactive transplantation of human neural stem cells blocks neuronal cell
death in rat model of Huntington disease. Neurobiol Dis 16:68–77.
Pe ´rez-Navarro E, Canudas AM, Akerund P, Alberch J, Arenas E. 2000. Brainderived neurotrophic factor, NT-3 and NT-3/4 prevent the death of striatal
projection neurons in rodent model of Huntington’s disease. J Neurochem
75:2190–2199
Yang CR, Yu RK. 2009. Intracerebral transplantation of neural stem cells
combined with trehalose ingetion alleviates pathology in a mouse model of
Huntington’s disease. J Neurosci Res 87:26–33.
Hudson AJ. 1990. Amyotrophic lateral sclerosis: concepts in pathogenesis and
etiology. Toronto: University of Toronto Press.
Bouillee S, Van de Velde C, Cleveland DW. 2006. ALS: a disease of motor neurons
and their nonneuronal neighbors. Neuron 52:39–59.
Li P, Tessler A, Han SS, Fischer I, Rao MS, Selzer ME. 2005. Fate of immortalized
human neuronal progenitor cells transplanted in rat spinal cord. Arch Neurol
62:223–229.
Kerr DA, Llado ´ J, Shamblott MJ, Maragakis NJ, Irani DN, Crawford TO, Krishnan C,
Dike S, Gearhart JD, Rothstein JD. 2003. Human embryonic germ cell derivatives
facilitate motor recovery of rats with diffuse motor neuron injury. J Neurosci
23:5131–5140.
Garbuzova-Davis S, Willing AE, Milliken M, Saporta S, Zigova T, Cahill DW,
Sanberg PR. 2002. Positive effect of transplantation of hNT neu- rons (NTera 2/D1
cell-line) in a model of familial amyotrophic lateral sclerosis. Exp Neurol 174:169–
180.
Klein SM, Behrstock S, McHugh J, Hoffmann K, Wallace K, Suzuki M, Aebischer P,
Svendsen CN. 2005. GDNF delivery using human neural progenitor cells in a rat
model of ALS. Hum Gene Ther 16:509–521.
Hwang DH, Lee HJ, Seok JI, Kim BG, Joo IS, Kim SU. 2009. Intrathe- cal
transplantation of human neural stem cells over-expressing VEGF provide
behavioral improvement, disease onset delay and survival extension in
transgenic ALS mice. Gene Ther (in press).
Coyle JT, Price DL, DeLong MR. 1983. Alzheimer’s disease: a disorder of cortical
cholinergic innervation. Science 219:1184–1190.
Bales KR, Tzavara ET, Wu S, Wade MR, Bymaster FP, Paul SM, Nomikos GG. 2006.
Cholinergc dysfunction in a mouse model of Alz- heimer disease is reversed by
an anti-Ab antibody. J Clin Invest 116:825–832.
Marr RA, Rockenstein E, Mukherjee A, Kindy MS, Hersh LB, Gage FH, Verma IM,
Masliah E. 2003. Neprilysin gene transfer reduces human amyloid patholgy in
trasgenic mice. J Neurosci 23:1992–1996.
Tuszynski MH, Thal L, Pay M, Salmon DP, U HS, Bakay R, Patel P, Blesch A,
Vahlsing HL, Ho G, Tong G, Potkin SG, Fallon J, Hansen L, Mufson EJ, Kordower JH,
Gall C, Conner J. 2005. A phase 1 clini- cal trial of nerve growth factor gene
therapy for Alzheimer disease. Nat Med 11:551–555.
Ebers GC. 1988. Multiple scelrosis and other demyelinating diseases. In: Asbury
A, McKhann G, McDonald W, editors. Diseases of the nervous system.
Philadelphia: W.B. Saunders. p 1268–1291.
Ben-Hur T, Einstein O, Mizrachi-Kol R, Ben-Menachem O, Reinhartz E, Karussis D,
Abramsky O. 2003. Transplanted multipotential neural progenitor cells migrate
into the inflamed white matter in response to experimental allergic encephalitis.
Glia 41:73–80.
Windrem MS, Nunes MC, Rashbaum WK, Schwartz TH, Goodman RA, McKhann G,
Roy NS, Goldman SA. 2004. Fetal and adult human oligodendrocyte progenitor
cell isolates myelinate the congenitally dys- myelinated brain. Nat Med 10:93–97.
Yandava B, Billinghurst L, Snyder E. 1999. Global cell replacement is feasible via
neural stem cell transplantation: evidence from the dysmye- linated shiverer
mouse brain. Proc Natl Acad Sci USA 96:7029–7034.
Copray S, Balasubramaniyan V, Levenga J, de Bruijn J, Liem R, Bod- deke E. 2006.
Olig2 overexpression induces the in vitro differentiation of neural stem cells into
mature oligodendrocytes. Stem Cells 24:1001– 1010.
Marshall J, Thomas DJ. 1988. Cerebrovascular disease. In: Asbury A, McKhann G,
McDonald W, editors. Diseases of the nervous system. Philadelphia: W.B.
Saunders. p 1101–1135.
Saporta S, Borlongan CV, Sanberg PR. 1999. Neural transplantation of human
teratocarcinoma neurons into ischemic rats. A quantitative dose- response
analysis of cell survival and behavioral recovery. Neuroscience 180:519–525.
Savitz SI, Rosenbaum DM, Dinsmore JH, Wechsler LR, Caplan LR. 2002. Cell
transplantation for stroke. Ann Neurol 52:266–275.
Kondziolka D, Wechsler L, Goldstein S, Meltzer C, Thulborn KR, Gebel J, Jannetta
P, DeCesare S, Elder EM, McGrogan M, Reitman MA, Bynum L. 2000.
Transplantation of cultured human neuronal cells for patients with stroke.
Neurology 55:565–569.
Chen J, Li Y, Katakowski M, Chen X, Wang L, Lu D, Lu M, Gautam SC, Chopp M.
2003. Intravenous bone marrow stromal cell therapy reduces apoptosis and
promotes endogenous cell proliferation after stroke in female rat. J Neurosci Res
73:778–786.
Kurozumi K, Nakamura K, Tamiya T, Kawano Y, Kobune M, Hirai S, Uchida H,
Sasaki K, Ito Y, Kato K, Honmou O, Houkin K, Date I, Hamada H. 2004. BDNF
gene-modified mesenchymal stem cells pro- mote functional recovery and
reduce infarct size in the rat middle cere- bral artery occlusion model. Mol Ther
9:189–197.
Nagai A, Kim WK, Lee HJ, Jeong HS, Kim KS, Hong SH, Park IH, Kim SU. 2007.
Multilineage potential of stable human mesenchymal stem cell line derived from
fetal marrow. PLoS ONE 2/e1272:1–18.
Harbor, NY: Cold Spiring Harbor Laboratory Press.
Taman K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, Yamanaka S. 2008.
Induction of pluripotent stem cells from adult human fibroblasts by defined
factors. Cell 131:861–872.
Kim SU. 2004. Human neural stem cells genetically modified for brain repair in
neurological disorders. Neuropathology 24:159–174.
Eriksson PS, Perfilieva E, Bjo ¨rk-Eriksson T, Alborn AM, Nordborg C, Peterson DA,
Gage FH. 1998. Neurogenesis in the adult human hippo- campus. Nat Med
4:1313–1317.
Kim SU, Nagai A, Nakagawa E, Choi HB, Bang JH, Lee HJ, Lee MA, Lee YB, Park IH.
2008b. Production and characterization of immortal human neural stem cell line
with multipotent differentiation property. Methods Mol Biol 438:103–121.
Lee HJ, Kim KS, Kim EJ, Choi HB, Lee KH, Park IH, Ko Y, Jeong SW, Kim SU. 2007a.
Brain transplantation of human neural stem cells promotes functional recovery in
mouse intracerebral hemorrhage stroke model. Stem Cells 25:211–224.
Goldman S. 2005. Stem and progenitor cell-based therapy of the human central
nervous system. Nat Biotechnol 7:862–871.
Agid Y. 1991. Parkinson’s disease: pathophysiology. Lancet 337:1321– 1324.
Lang AE, Lozano AM. 1998b. Parkinson’s disease. Second of two parts. N Engl J
Med 339:1130–1143.
Dunnett SB, Bjorklund A. 1999. Prospects for new restorative and neuroprotective treatments in Parkinson’s disease. Nature 399:A32–A39.
Hagell P, Schrag A, Piccini P, Jahanshahi M, Brown R, Rehncrona S, Widner H,
Brundin P, Rothwell JC, Odin P, Wenning GK, Morrish P, Gustavii B, Bjo ¨rklund A,
Brooks DJ, Marsden CD, Quinn NP, Lindvall O. 1999. Sequential bilateral
transplantation in Parkinson’s dis- ease: effects of the second graft. Brain
122:1121–1132.
Shim JW, Koh HC, Chang MY, Roh E, Choi CY, Oh YJ, Son H, Lee YS, Studer L, Lee
SH. 2004. Enhanced in vitro midbrain dopamine neuron differentiation,
dopaminergic function, neurite outgrowth, and 1-methyl-4-phenylpyridium
resistance in mouse embryonic stem cells overexpressing Bcl-xL. J Neurosci
24:843–852.
Kawasaki H, Mizuseki K, Nishikawa S, Kaneko S, Kuwana Y, Nakanishi S,
Nishikawa SI, Sasai Y. 2000. Induction of midbrain dopaminergic neurons from ES
cells by stromal cell-derived inducing activity. Neuron 28:31–40.
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 ES cells function in a Parkinson primate model. J
Clin Invest 115:102–108.
Redmond DE Jr, Bjugstad KB, Teng YD, Ourednik V, Ourednik J, Wakeman DR,
Parsons XH, Gonzalez R, Blanchard BC, Kim SU, Gu Z, Lipton SA, Markakis EA,
Roth RH, Elsworth JD, Sladek JR Jr, Sidman RL, Snyder EY. 2007. Behavioral
improvement in a primate Parkinson’s model is associated with multiple
homeostatic effects of human neural stem cells. Proc Natl Acad Sci USA
104:12175–12180.
Kang UJ, Fisher LJ, Joh TH, O’Malley KL, Gage FH. 1993. Regulation of dopamine
production by genetically modified primary fibroblasts. J Neurosci 13:5203–5211.
Harper PS. 1996. Huntington’s disease. Philadelphia: W.B. Saunders.
Bachoud-Le ´vi AC, Re ´my P, Nguyen JP, Brugie `res P, Lefaucheur JP, Bourdet C,
Baudic S, Gaura V, Maison P, Haddad B, Boisse ´ MF, Grandmougin T, Je ´ny R,
Bartolomeo P, Dalla Barba G, Degos JD, Lisovoski F, Ergis AM, Pailhous E, Cesaro
P, Hantraye P, Peschanski M. 2000. Motor and cognitive improvements in
patients with Hunting- ton’s disease after neural transplnatation. Lancet
356:1975–1979.
Freeman TB, Cicchetti F, Hauser RA, Deacon TW, Li XJ, Hersch SM, Nauert GM,
Sanberg PR, Kordower JH, Saporta S, Isacson O. 2000. Transplanted fetal striatum
in Huntington’s disease: phenotypic devel- opment and lack of pathology. Proc
Natl Acad Sci USA 97:13877– 13882.
Huntington’s Disease Collaborative Research Group. 1993. A novel gene
containing a trinucleotide repeat that is expanded and unstable on Hun- tington’s
disease chromosomes. Cell 72:971–983.
Roberts TJ, Price J, Williams SC, Modo M. 2006. Preservation of striatal tissue and
behavioral function after neural stem cell transplantation in a rat model of
Huntington’s disease. Neuroscience 139:1187–1199.
Ryu JK, Kim J, Cho SJ, Hatori K, Nagai A, Choi HB, Lee MC, McLar- non JG, Kim SU.
2004. Proactive transplantation of human neural stem cells blocks neuronal cell
death in rat model of Huntington disease. Neurobiol Dis 16:68–77.
Pe ´rez-Navarro E, Canudas AM, Akerund P, Alberch J, Arenas E. 2000. Brainderived neurotrophic factor, NT-3 and NT-3/4 prevent the death of striatal
projection neurons in rodent model of Huntington’s disease. J Neurochem
75:2190–2199
Yang CR, Yu RK. 2009. Intracerebral transplantation of neural stem cells
combined with trehalose ingetion alleviates pathology in a mouse model of
Huntington’s disease. J Neurosci Res 87:26–33.
Hudson AJ. 1990. Amyotrophic lateral sclerosis: concepts in pathogenesis and
etiology. Toronto: University of Toronto Press.
Bouillee S, Van de Velde C, Cleveland DW. 2006. ALS: a disease of motor neurons
and their nonneuronal neighbors. Neuron 52:39–59.
Li P, Tessler A, Han SS, Fischer I, Rao MS, Selzer ME. 2005. Fate of immortalized
human neuronal progenitor cells transplanted in rat spinal cord. Arch Neurol
62:223–229.
Kerr DA, Llado ´ J, Shamblott MJ, Maragakis NJ, Irani DN, Crawford TO, Krishnan C,
Dike S, Gearhart JD, Rothstein JD. 2003. Human embryonic germ cell derivatives
facilitate motor recovery of rats with diffuse motor neuron injury. J Neurosci
23:5131–5140.
Garbuzova-Davis S, Willing AE, Milliken M, Saporta S, Zigova T, Cahill DW,
Sanberg PR. 2002. Positive effect of transplantation of hNT neu- rons (NTera 2/D1
cell-line) in a model of familial amyotrophic lateral sclerosis. Exp Neurol 174:169–
180.
Klein SM, Behrstock S, McHugh J, Hoffmann K, Wallace K, Suzuki M, Aebischer P,
Svendsen CN. 2005. GDNF delivery using human neural progenitor cells in a rat
model of ALS. Hum Gene Ther 16:509–521.
Hwang DH, Lee HJ, Seok JI, Kim BG, Joo IS, Kim SU. 2009. Intrathe- cal
transplantation of human neural stem cells over-expressing VEGF provide
behavioral improvement, disease onset delay and survival extension in
transgenic ALS mice. Gene Ther (in press).
Coyle JT, Price DL, DeLong MR. 1983. Alzheimer’s disease: a disorder of cortical
cholinergic innervation. Science 219:1184–1190.
Bales KR, Tzavara ET, Wu S, Wade MR, Bymaster FP, Paul SM, Nomikos GG. 2006.
Cholinergc dysfunction in a mouse model of Alz- heimer disease is reversed by
an anti-Ab antibody. J Clin Invest 116:825–832.
Marr RA, Rockenstein E, Mukherjee A, Kindy MS, Hersh LB, Gage FH, Verma IM,
Masliah E. 2003. Neprilysin gene transfer reduces human amyloid patholgy in
trasgenic mice. J Neurosci 23:1992–1996.
Tuszynski MH, Thal L, Pay M, Salmon DP, U HS, Bakay R, Patel P, Blesch A,
Vahlsing HL, Ho G, Tong G, Potkin SG, Fallon J, Hansen L, Mufson EJ, Kordower JH,
Gall C, Conner J. 2005. A phase 1 clini- cal trial of nerve growth factor gene
therapy for Alzheimer disease. Nat Med 11:551–555.
Ebers GC. 1988. Multiple scelrosis and other demyelinating diseases. In: Asbury
A, McKhann G, McDonald W, editors. Diseases of the nervous system.
Philadelphia: W.B. Saunders. p 1268–1291.
Ben-Hur T, Einstein O, Mizrachi-Kol R, Ben-Menachem O, Reinhartz E, Karussis D,
Abramsky O. 2003. Transplanted multipotential neural progenitor cells migrate
into the inflamed white matter in response to experimental allergic encephalitis.
Glia 41:73–80.
Windrem MS, Nunes MC, Rashbaum WK, Schwartz TH, Goodman RA, McKhann G,
Roy NS, Goldman SA. 2004. Fetal and adult human oligodendrocyte progenitor
cell isolates myelinate the congenitally dys- myelinated brain. Nat Med 10:93–97.
Yandava B, Billinghurst L, Snyder E. 1999. Global cell replacement is feasible via
neural stem cell transplantation: evidence from the dysmye- linated shiverer
mouse brain. Proc Natl Acad Sci USA 96:7029–7034.
Copray S, Balasubramaniyan V, Levenga J, de Bruijn J, Liem R, Bod- deke E. 2006.
Olig2 overexpression induces the in vitro differentiation of neural stem cells into
mature oligodendrocytes. Stem Cells 24:1001– 1010.
Marshall J, Thomas DJ. 1988. Cerebrovascular disease. In: Asbury A, McKhann G,
McDonald W, editors. Diseases of the nervous system. Philadelphia: W.B.
Saunders. p 1101–1135.
Saporta S, Borlongan CV, Sanberg PR. 1999. Neural transplantation of human
teratocarcinoma neurons into ischemic rats. A quantitative dose- response
analysis of cell survival and behavioral recovery. Neuroscience 180:519–525.
Savitz SI, Rosenbaum DM, Dinsmore JH, Wechsler LR, Caplan LR. 2002. Cell
transplantation for stroke. Ann Neurol 52:266–275.
Kondziolka D, Wechsler L, Goldstein S, Meltzer C, Thulborn KR, Gebel J, Jannetta
P, DeCesare S, Elder EM, McGrogan M, Reitman MA, Bynum L. 2000.
Transplantation of cultured human neuronal cells for patients with stroke.
Neurology 55:565–569.
Chen J, Li Y, Katakowski M, Chen X, Wang L, Lu D, Lu M, Gautam SC, Chopp M.
2003. Intravenous bone marrow stromal cell therapy reduces apoptosis and
promotes endogenous cell proliferation after stroke in female rat. J Neurosci Res
73:778–786.
Kurozumi K, Nakamura K, Tamiya T, Kawano Y, Kobune M, Hirai S, Uchida H,
Sasaki K, Ito Y, Kato K, Honmou O, Houkin K, Date I, Hamada H. 2004. BDNF
gene-modified mesenchymal stem cells pro- mote functional recovery and
reduce infarct size in the rat middle cere- bral artery occlusion model. Mol Ther
9:189–197.
Nagai A, Kim WK, Lee HJ, Jeong HS, Kim KS, Hong SH, Park IH, Kim SU. 2007.
Multilineage potential of stable human mesenchymal stem cell line derived from
fetal marrow. PLoS ONE 2/e1272:1–18.
