Astrocytes from familial and sporadic ALS patients are toxic to motor neurons

Nature Biotechnology

Volumn 29, Issue 9, 2011, Pages 824-828

  Haidet Phillips, Amanda M ^a,b   Hester, Mark E ^a   Miranda, Carlos J ^a   Meyer, Kathrin ^a   Braun, Lyndsey ^a   Frakes, Ashley ^a,b   Song, Sungwon ^a,c   Likhite, Shibi ^a,c   Murtha, Matthew J ^a,c   Foust, Kevin D ^a   Rao, Meghan ^a   Eagle, Amy ^a   Kammesheidt, Anja ^d   Christensen, Ashley ^d   Mendell, Jerry R ^a,b   Burghes, Arthur H M ^c   Kaspar, Brian K ^a,c  

^a THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b THE OHIO STATE UNIVERSITY COLLEGE OF MEDICINE   (United States)

^c OHIO STATE UNIVERSITY   (United States)

^d AMBRY GENETICS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALS PATIENTS; AMYOTROPHIC LATERAL SCLEROSIS; AUTONOMOUS COMPONENTS; IN-VITRO; MOTOR NEURON DISEASE; MOTOR NEURONS; POTENTIAL THERAPIES; RODENT MODELS; SUPER OXIDE DISMUTASE;

BRAIN; DISEASE CONTROL; GENES; NEURONS; OXYGEN; TISSUE; TOXIC MATERIALS;

NEURODEGENERATIVE DISEASES;

COPPER ZINC SUPEROXIDE DISMUTASE;

AMYOTROPHIC LATERAL SCLEROSIS; ARTICLE; ASTROCYTE; CONTROLLED STUDY; CYTOTOXICITY; GENE MUTATION; HUMAN; HUMAN CELL; MOTONEURON; MOTOR NEURON DISEASE; NERVE CELL CULTURE; NERVE CELL NECROSIS; NEUROPROTECTION; NEUROTOXICITY; PRIORITY JOURNAL; SPINAL CORD;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; ASTROCYTES; BIOLOGICAL MARKERS; CELL DIFFERENTIATION; CELL LINE; COCULTURE TECHNIQUES; DISEASE MODELS, ANIMAL; EMBRYONIC STEM CELLS; GENE EXPRESSION REGULATION; HUMANS; IMMUNOHISTOCHEMISTRY; MESENCHYMAL STEM CELLS; MICE; MOTOR NEURONS; MUTATION; SEQUENCE ANALYSIS, DNA; SIGNAL TRANSDUCTION; SUPEROXIDE DISMUTASE;

RODENTIA;

EID: 80052783545     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt.1957     Document Type: Article

References (30)

1. Di Giorgio, F.P., Boulting, G.L., Bobrowicz, S. & Eggan, K.C. Human embryonic stem cell-derived motor neurons are sensitive to the oxic effect of glial cells carrying an ALS-causing mutation. Cell Stem Cell 3, 637-648 (2008).
2. Yamanaka, K. et al. Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis. Nat. Neurosci. 11, 51-253 (2008).
3. Marchetto, M.C. et al. Non-cell-autonomous effect of human SOD1 G37R astrocytes on motor neurons derived from human embryonic stem cells. Cell Stem Cell 3, 649-657 (2008).
4. Di Giorgio, F.P., Carrasco, M.A., Siao, M.C., Maniatis, T. & Eggan, K. Non-cell autonomous effect of glia on motor neurons in an embryonic stem cell-based ALS model. Nat. Neurosci. 10, 608-614 (2007). (Pubitemid 46652442)
5. Nagai, M. et al. Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons. Nat. Neurosci. 10, 615-622 (2007). (Pubitemid 46652435)
6. Brown, R.H. Jr. Amyotrophic lateral sclerosis. Insights from genetics. Arch. Neurol. 54, 1246-1250 (1997). (Pubitemid 27452239)
7. Ilieva, H., Polymenidou, M. & Cleveland, D.W. Non-cell autonomous toxicity in neurodegenerative disorders: ALS and beyond. J. Cell Biol. 187, 761-772 (2009).
8. Boillee, S., Van de Velde, C. & Cleveland, D.W. ALS: a disease of motor neurons and their non-neuronal neighbors. Neuron 52, 39-59 (2006). (Pubitemid 44466358)
9. Boillee, S. et al. Onset and progression in inherited ALS determined by motor neurons and microglia. Science 312, 1389-1392 (2006). (Pubitemid 43839552)
10. Clement, A.M. et al. Wild-type non-neuronal cells extend survival of SOD1 mutant motor neurons in ALS mice. Science 302, 113-117 (2003). (Pubitemid 37210700)
11. Beers, D.R. et al. Wild-type microglia extend survival in PU.1 knockout mice with familial amyotrophic lateral sclerosis. Proc. Natl. Acad. Sci. USA 103, 16021-16026 (2006). (Pubitemid 44657585)
12. Boulting, G.L. et al. A functionally characterized test set of human induced pluripotent stem cells. Nat. Biotechnol. 29, 279-286 (2011).
13. Dimos, J.T. et al. Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons. Science 321, 1218-1221 (2008).
14. Glass, C.K., Saijo, K., Winner, B., Marchetto, M.C. & Gage, F.H. Mechanisms underlying inflammation in neurodegeneration. Cell 140, 918-934 (2010).
15. Dodge, J.C. et al. Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity. Mol. Ther. 16, 1056-1064 (2008). (Pubitemid 351737070)
16. Palmer, T.D. et al. Cell culture. Progenitor cells from human brain after death. Nature 411, 42-43 (2001). (Pubitemid 32428181)
17. Ray, J. & Gage, F.H. Differential properties of adult rat and mouse brain-derived neural stem/progenitor cells. Mol. Cell. Neurosci. 31, 560-573 (2006). (Pubitemid 43358027)
18. Cahoy, J.D. et al. A transcriptome database for astrocytes, neurons, and oligodendrocytes: a new resource for understanding brain development and function. J. Neurosci. 28, 264-278 (2008).
19. Wichterle, H., Lieberam, I., Porter, J.A. & Jessell, T.M. Directed differentiation of embryonic stem cells into motor neurons. Cell 110, 385-397 (2002).
20. Dodge, J.C. et al. AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice. Mol. Ther. 18, 2075-2084 (2010).
21. Lobsiger, C.S., Boillee, S. & Cleveland, D.W. Toxicity from different SOD1 mutants dysregulates the complement system and the neuronal regenerative response in ALS motor neurons. Proc. Natl. Acad. Sci. USA 104, 7319-7326 (2007). (Pubitemid 47185903)
22. Wang, R., Yang, B. & Zhang, D. Activation of interferon signaling pathways in spinal cord astrocytes from an ALS mouse model. Glia 59, 946-958 (2011).
23. Bendotti, C. et al. Inter- and intracellular signaling in amyotrophic lateral sclerosis: role of p38 mitogen-activated protein kinase. Neurodegener. Dis. 2, 128-134 (2005). (Pubitemid 43827913)
24. Miller, T.M. et al. Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis. Ann. Neurol. 57, 773-776 (2005). (Pubitemid 40628872)
25. Bosco, D.A. et al. Wild-type and mutant SOD1 share an aberrant conformation and a common pathogenic pathway in ALS. Nat. Neurosci. 13, 1396-1403 (2010).
26. Gruzman, A. et al. Common molecular signature in SOD1 for both sporadic and familial amyotrophic lateral sclerosis. Proc. Natl. Acad. Sci. USA 104, 12524-12529 (2007). (Pubitemid 47206171)
27. Zetterstrom, P., Graffmo, K.S., Andersen, P.M., Brannstrom, T. & Marklund, S.L. Proteins that bind to misfolded mutant superoxide ismutase-1 in spinal cords from transgenic ALS model mice. J. Biol. Chem. 286, 20130-20136 (2011).
28. Foust, K.D. et al. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat. Biotechnol. 27, 59-65 (2009).
29. Lepore, A.C. et al. Focal transplantation-based astrocyte replacement is neuroprotective in a model of motor neuron disease. Nat. eurosci. 11, 1294-1301 (2008).
30. Hester, M.E. et al. Rapid and efficient generation of functional motor neurons from human pluripotent stem cells using gene elivered transcription factor codes. Mol. Ther. (in the press).