The dynamics of long-term transgene expression in engrafted neural stem cells

Journal of Comparative Neurology

Volumn 515, Issue 1, 2009, Pages 83-92

  Lee, Pean Pyo ^a,b   Tsai, David J ^a   Park, Kook In ^c   Harvey, Alan R ^d   Snyder, Evan Y ^a,b  

^a BURNHAM INSTITUTE   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c Yonsei University College of Medicine   (South Korea)

^d UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

Author keywords

CNS gene therapy; FISH; Neural transplantation; Viral vectors

Indexed keywords

BETA GALACTOSIDASE; BETA TUBULIN; GLIAL FIBRILLARY ACIDIC PROTEIN; NEURON SPECIFIC NUCLEAR PROTEIN;

ANIMAL CELL; ANIMAL TISSUE; CELL LEVEL; CELL NUCLEUS; CONFERENCE PAPER; CONTROLLED STUDY; DOWN REGULATION; EMBRYO; ENGRAFTMENT; FLUORESCENCE IN SITU HYBRIDIZATION; GENE EXPRESSION; GENE INSERTION; IMMUNOHISTOCHEMISTRY; IN VIVO STUDY; MOUSE; NEURAL STEM CELL; NONHUMAN; PRIORITY JOURNAL; STEM CELL TRANSPLANTATION; TRANSGENE;

ANIMALS; ANIMALS, NEWBORN; BRAIN TISSUE TRANSPLANTATION; CELL CULTURE TECHNIQUES; CELL DIFFERENTIATION; CELL PROLIFERATION; CLONE CELLS; FEMALE; GENE DOSAGE; GENE EXPRESSION REGULATION; GENES, REPORTER; GENETIC VECTORS; GRAFT SURVIVAL; MALE; MICE; NEURONS; RETROVIRIDAE; STEM CELL TRANSPLANTATION; STEM CELLS; TIME; TRANSFECTION; TRANSGENES;

EID: 67650882734     PISSN: 00219967     EISSN: 10969861     Source Type: Journal    
DOI: 10.1002/cne.21957     Document Type: Conference Paper

References (37)

1. Abdellatif AA, Pelt JL, Benton RL, Howard RM, Tsoulfas P, Ping P, Xu XM, Whittemore SR. 2006. Gene delivery to the spinal cord: comparison between lentiviral, adenoviral, and retroviral vector delivery systems. J Neurosci Res 84:553-567.
2. Breneman JW, Swiger RR, Ramsey MJ, Minkler JL, Eveleth JG, Langlois RA, Tucker JD. 1995. The development of painting probes for dualcolor and multiple chromosome analysis in the mouse. Cytogenet Cell Genet 68:197-202.
3. Cartwright P, McLean C, Sheppard A, Rivett D, Jones K, Dalton S. 2005. LIF/STAT3 controls ES cell self-renewal and pluripotency by a Myc-dependent mechanism. Development 132:885-896.
4. Coggeshall RE, Lekan HA. 1996. Methods for determining numbers of cells and synapses: a case for more uniform standards of review. J Comp Neurol 364:6-15.
5. Couwenhoven RI, Luo W, Snead ML. 1990. Co-localization of EGF transcripts and peptides by combined immunohistochemistry and in situ hybridization. J Histochem Cytochem 38:1853-1857.
6. Ellis J. 2005. Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum Gene Ther 16:1241-1246.
7. Flax JD, Aurora S, Yang C, Simonin C, Wills AM, Billinghurst LL, Jendoubi M, Sidman RL, Wolfe JH, Kim SU, Snyder EY. 1998. Engraftable human neural stem cells respond to developmental cues, replace neurons, and express foreign genes. Nat Biotechnol 16:1033-1039.
8. Guillery RW, Herrup K. 1997. Quantification without pontification: choosing a method for counting objects in sectioned tissues. J Comp Neurol 386:2-7.
9. Gussoni E, Wang Y, Fraefel C, Miller RG, Blau HM, Geller AI, Kunkel LM. 1996. A method to codetect introduced genes and their products in gene therapy protocols. Nat Biotechnol 14:1012-1016.
10. Gussoni E, Blau HM, Kunkel LM. 1997. The fate of individual myoblasts after transplantation into muscles of DMD patients. Nat Med 3:970-977.
11. Harvey AR, Fan Y, Beilharz MW, Grounds MD. 1992. Survival and migration of transplanted male glia in adult female mouse brains monitored by a Y-chromosome-specific probe. Brain Res Mol Brain Res 12:339-343.
12. Held PK, Olivares EC, Aguilar CP, Finegold M, Calos MP, Grompe M. 2005. In vivo correction of murine hereditary tyrosinemia type I by phiC31 integrase-mediated gene delivery. Mol Ther 11:399- 408.
13. Jakobsson J, Lundberg C. 2006. Lentiviral vectors for use in the central nervous system. Mol Ther 13:484- 493.
14. Kaplan PL, Simon S, Cartwright CA, Eckhart W. 1987. cDNA cloning with a retrovirus expression vector: generation of a pp60C-src cDNA clone. J Virol 61(5):1731-1734.
15. Knoepfler PS, Cheng PF, Eisenman RN. 2002. N-myc is essential during neurogenesis for the rapid expansion of progenitor cell populations and the inhibition of neuronal differentiation. Genes Dev 16:2699-2712.
16. Lacorazza HD, Flax JD, Snyder EY, 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.
17. Lee JP, Jeyakumar M, Gonzalez R, Takahashi H, Lee PJ, Baek RC, Clark D, Rose H, Fu G, Clarke J, McKercher S, Meerloo J, Muller FJ, Park KI, Butters TD, Dwek RA, Schwartz P, Tong G, Wenger D, Lipton SA, Seyfried TN, Platt FM, Snyder EY. 2007. Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease. Nat Med 13:439-447.
18. Light W, Vernon AE, Lasorella A, Iavarone A, LaBonne C. 2005. Xenopus Id3 is required downstream of Myc for the formation of multipotent neural crest progenitor cells. Development 132:1831-1841.
19. Maragakis NJ, Rao MS, Llado J, Wong V, Xue H, Pardo A, Herring J, Kerr D, Coccia C, Rothstein JD. 2005. Glial restricted precursors protect against chronic glutamate neurotoxicity of motor neurons in vitro. Glia 50:145-159.
20. Murphy MJ, Wilson A, Trumpp A. 2005. More than just proliferation: Myc function in stem cells. Trends Cell Biol 15:128-137.
21. Nienhuis AW, Dunbar CE, Sorrentino BP. 2006. Genotoxicity of retroviral integration in hematopoietic cells. Mol Ther 13:1031-1049.
22. Palmer TD, Rosman GJ, Osborne WR, Miller AD. 1991. Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc Natl Acad Sci U S A 88:1330-1334.
23. Park K, Hack MA, Ourednik J, Yandava B, Flax JD, Stieg PE, Gullans S, Jensen FE, Sidman RL, Ourednik V, Snyder EY. 2006. Acute injury directs the migration, proliferation, and differentiation of solid organ stem cells: evidence from the effect of hypoxia-ischemia in the CNS on clonal "reporter" neural stem cells. Exp Neurol 199:156-178.
24. Parker MA, Anderson JK, Corliss DA, Abraria VE, Sidman RL, Park KI, Teng YD, Cotanche DA, Snyder EY. 2005. Expression profile of an operationally-defined neural stem cell clone. Exp Neurol 194:320-332.
25. Philippe S, Sarkis C, Barkats M, Mammeri H, Ladroue C, Petit C, Mallet J, Serguera C. 2006. Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression in vitro and in vivo. Proc Natl Acad Sci U S A 103:17684-17689.
26. Price J, Turner D, Cepko C. 1987. Lineage analysis in the vertebrate nervous system by retrovirus-mediated gene transfer. Proc Natl Acad Sci U S A 84:156-160.
27. Rosario CM, Yandava BD, Kosaras B, Zurakowski D, Sidman RL, Snyder EY. 1997. 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 124:4213-4224.
28. Rosenqvist N, Jakobsson J, Lundberg C. 2005. Inhibition of chromatin condensation prevents transgene silencing in a neural progenitor cell line transplanted to the rat brain. Cell Transplant 14:129-138.
29. Ryder EF, Snyder EY, Cepko CL. 1990. Establishment and characterization of multipotent neural cell lines using retrovirus vector-mediated oncogene transfer. J Neurobiol 21:356-375.
30. Snyder EY, Deitcher DL, Walsh C, Arnold-Aldea S, Hartwieg EA, Cepko CL. 1992. Multipotent neural cell lines can engraft and participate in development of mouse cerebellum. Cell 68:33-51.
31. Snyder EY, Taylor RM, Wolfe JH. 1995. Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature 374:367-370.
32. Symons NA, Danielsen N, Harvey AR. 2001. Migration of cells into and out of peripheral nerve isografts in the peripheral and central nervous systems of the adult mouse. Eur J Neurosci 14:522-532.
33. Takahashi K, Yamanaka S. 2006. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 126:663-676.
34. Taylor RM, Lee JP, Palacino JJ, Bower KA, Li J, Vanier MT, Wenger DA, Sidman RL, Snyder EY. 2006. 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 97:1585-1599.
35. Vroemen M, Weidner N, Blesch A. 2005. Loss of gene expression in lentivirus- and retrovirus-transduced neural progenitor cells is correlated to migration and differentiation in the adult spinal cord. Exp Neurol 195:127-139.
36. Weimann JM, Charlton CA, Brazelton TR, Hackman RC, Blau HM. 2003. Contribution of transplanted bone marrow cells to Purkinje neurons in human adult brains. Proc Natl Acad Sci U S A 100:2088-2093.
37. Zagon IS, McLaughlin PJ. 1979. Morphological identification and biochemical characterization of isolated brain cell nuclei from the developing rat cerebellum. Brain Res 170:443-457.