Neural stem cell transplantation benefits a monogenic neurometabolic disorder during the symptomatic phase of disease

Stem Cells

Volumn 27, Issue 9, 2009, Pages 2362-2370

  Jeyakumar, Mylvaganam ^a   Lee, Jean Pyo ^b,c   Sibson, Nicola R ^a   Lowe, John P ^a   Stuckey, Daniel J ^a   Tester, Katie ^a   Fu, Gerald ^b   Newlin, Robin ^b   Smith, David A ^a   Snyder, Evan Y ^b,c   Platt, Frances M ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b BURNHAM INSTITUTE   (United States)

^c UNIVERSITY OF CALIFORNIA SAN DIEGO   (United States)

Author keywords

Adult brain; Behavior chemokines; Inflammation; Lysosomal storage disorders; Magnetic resonance imaging; Metabolic cross correction; Migration; Neural stem cell therapy; Neurodegenerative disease; Sandhoff disease

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE; CHEMOKINE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN FUNCTION; CELL DIFFERENTIATION; CELL FUNCTION; CELL MIGRATION; COGNITION; CONTROLLED STUDY; DISEASE COURSE; ENCEPHALITIS; ENGRAFTMENT; ENZYME ACTIVITY; HIPPOCAMPUS; INJECTION SITE; LIFESPAN; LYSOSOME; METABOLIC DISORDER; MONOGENIC DISORDER; MOUSE; NEURAL STEM CELL; NEURAL STEM CELL TRANSPLANTATION; NEUROLOGIC DISEASE; NEUROPATHOLOGY; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PHENOTYPE; PROTEIN EXPRESSION; SANDHOFF DISEASE;

ANIMALS; CELL LINE; CENTRAL NERVOUS SYSTEM; IMMUNOHISTOCHEMISTRY; LYSOSOMES; MICE; MICE, MUTANT STRAINS; MICROSCOPY, CONFOCAL; NEURONS; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; SANDHOFF DISEASE; STEM CELL TRANSPLANTATION;

MUS;

EID: 70349869180     PISSN: 10665099     EISSN: None     Source Type: Journal    
DOI: 10.1002/stem.163     Document Type: Article

References (36)

1. Wraith JE. Lysosomal disorders. Semin Neonatol 2002;7:75-83.
2. Wada R, Tiff CJ, Proia, RL. Microglial activation precedes acute neurodegeneration in Sandhoff disease and is suppressed by bone marrow transplantation. Proc Natl Acad Sci USA 2000;97:10954-10959. (Pubitemid 30751260)
3. Myerowitz R, Lawson D, Mizukami H et al. Molecular pathophysiology in Tay-Sachs and Sandhoff diseases as revealed by gene expression profiling. Hum Mol Genet 2002;11:1343-1350. (Pubitemid 34555532)
4. Jeyakumar M, Thomas R, Elliot-Smith E et al. Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis. Brain 2003;126:974-987. (Pubitemid 36372994)
5. Sango K, Yamanaka S, Hoffmann A et al. Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism. Nat Genet 1995;11:170-176.
6. Phaneuf D, Wakamatsu N, Huang JQ et al. Dramatically different phenotypes in mouse models of human Tay-Sachs and Sandhoff diseases. Hum Mol Genet 1996;5:1-14. (Pubitemid 26018268)
7. Jeyakumar M, Dwek RA, Butters TD et al. Storage solutions: treating lysosomal disorders of the brain. Nat Rev Neurosci 2005;6:713-725. (Pubitemid 44773245)
8. Suzuki K, Iseki E, Togo T et al. Neuronal and glial accumulation of alpha- and beta-synucleins in human lipidoses. Acta Neuropathol (Berl) 2007;114:481-489. (Pubitemid 47521504)
9. Suzuki K, Iseki E, Katsuse O et al. Neuronal accumulation of alpha-and beta-synucleins in the brain of a GM2 gangliosidosis mouse model. Neuroreport 2003;14:551-554. (Pubitemid 36528988)
10. Pluchino S, Zanotti L, Rossi B et al. Neurosphere-derived multipotent precursors promote neuroprotection by an immunomodulatory mechanism. Nature 2005;436:266-271. (Pubitemid 41021278)
11. Ourednik J, Ourednik V, Lynch WP et al. Neural stem cells display an inherent mechanism for rescuing dysfunctional neurons. Nat Biotechnol 2002;20:1103-1110. (Pubitemid 35285438)
12. 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. (Pubitemid 46559825)
13. Wraith JE. Clinical aspects and diagnosis. In: Platt FM, Walkley SU (ed). Lysosomal disorders of the brain. Oxford: Oxford University Press, 2004:50-77.
14. Ryder EF, Snyder EY, Cepko CL. Establishment and characterization of multipotent neural cell lines using retrovirus vector-mediated oncogene transfer. J Neurobiol 1990;21:356-375.
15. Snyder EY, Deitcher DL, Walsh C et al. Multipotent neural cell lines can engraft and participate in development of mouse cerebellum. Cell 1992;68:33-51.
16. Snyder EY, Taylor RM, Wolfe JH. Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature 1995;374:367-370.
17. Snyder EY. Immortalized neural stem cells: insights into development; prospects for gene therapy and repair. Proc Assoc Am Physicians 1995;107:195-204.
18. Rosario CM, Yandava BD, Kosaras B et al. Differentiation of engrafted multipotent neural progenitors towards replacement of missing granule neurons in meander tail cerebellum may help determine the locus of mutant gene action. Development 1997;124:4213-4224. (Pubitemid 27496857)
19. Jeyakumar M, Norflus F, Tifft CJ et al. Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation. Blood 2001;97:327-329. (Pubitemid 32061278)
20. Jeyakumar M, Butters TD, Cortina-Borja M et al. Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin. Proc Natl Acad Sci USA 1999;96:6388-6393. (Pubitemid 29256675)
21. Andersson U, Smith D, Jeyakumar M et al. Improved outcome of N-butyldeoxygalactonojirimycin- mediated substrate reduction therapy in a mouse model of Sandhoff disease. Neurobiol Dis 2004;16:506-515. (Pubitemid 38942983)
22. Jeyakumar M, Smith DA, Williams IM et al. NSAIDs increase survival in the Sandhoff disease mouse: synergy with N-butyldeoxynojirimycin. Ann Neurol 2004;56:642-649. (Pubitemid 39540744)
23. Hoehn M, Kustermann E, Blunk J et al. Monitoring of implanted stem cell migration in vivo: a highly resolved in vivo magnetic resonance imaging investigation of experimental stroke in rat. Proc Natl Acad Sci USA 2002;99:16267-16272. (Pubitemid 35462784)
24. Neville DC, Coquard V, Priestman DA et al. Analysis of fluorescently labeled glycosphingolipid-derived oligosaccharides following ceramide glycanase digestion and anthranilic acid labeling. Anal Biochem 2004;331:275-282. (Pubitemid 38953078)
25. Zhang SC, Fedoroff S. Expression of stem cell factor and c-kit receptor in neural cells after brain injury. Acta Neuropathol (Berl) 1999;97:393-398. (Pubitemid 29134520)
26. 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. (Pubitemid 46559825)
27. Sun L, Lee J, Fine HA. Neuronally expressed stem cell factor induces neural stem cell migration to areas of brain injury. J Clin Invest 2004;113:1364-1374. (Pubitemid 39069935)
28. Martino G, Pluchino S. The therapeutic potential of neural stem cells. Nat Rev Neurosci 2006;7:395-406.
29. Bae JS, Furuya S, Ahn SJ et al. Neuroglial activation in Niemann- Pick type C mice is suppressed by intracerebral transplantation of bone marrow-derived mesenchymal stem cells. Neurosci Lett 2005;381:234-236. (Pubitemid 40719163)
30. Meng XL, Shen JS, Ohashi T et al. 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. (Pubitemid 37193713)
31. Karolewski BA, Wolfe JH. Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII. Mol Ther 2006;14:14-24.
32. Taylor RM, Lee JP, Palacino JJ et al. Intrinsic resistance of neural stem cells to toxic metabolites may make them well suited for cell non-autonomous disorders: evidence from a mouse model of Krabbe leukodystrophy. J Neurochem 2006;97:1585-1599.
33. Klein D, Schmandt T, Muth-Kohne E et al. Embryonic stem cell-based reduction of central nervous system sulfatide storage in an animal model of metachromatic leukodystrophy. Gene Ther 2006;13:1686-1695. (Pubitemid 44849033)
34. Pellegatta S, Tunici P, Poliani PL et al. The therapeutic potential of neural stem/progenitor cells in murine globoid cell leukodystrophy is conditioned by macrophage/microglia activation. Neurobiol Dis 2006;21:314-323. (Pubitemid 43099855)
35. 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. (Pubitemid 39557637)
36. Proia RL, Wu YP. Blood to brain to the rescue. J Clin Invest 2004;113:1108-1110.