Selective knockdown of mutant SOD1 in Schwann cells ameliorates disease in G85R mutant SOD1 transgenic mice

Neurobiology of Disease

Volumn 48, Issue 1, 2012, Pages 52-57

  Wang, Lijun ^a   Pytel, Peter ^a   Feltri, M Laura ^b   Wrabetz, Lawrence ^b   Roos, Raymond P ^a  

^a UNIVERSITY OF CHICAGO MEDICAL CENTER   (United States)

^b UNIVERSITY AT BUFFALO   (United States)

Author keywords

Amyotrophic lateral sclerosis; Familial amyotrophic lateral sclerosis; Motor neuron disease; Mutant superoxide dismutase type 1; Non cell autonomous degeneration; Schwann cells; Transgenic mice

Indexed keywords

COPPER ZINC SUPEROXIDE DISMUTASE; CRE RECOMBINASE; MYELIN PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL SURVIVAL; CONTROLLED STUDY; DISEASE COURSE; ENZYME ACTIVITY; GENE EXPRESSION; GENE SILENCING; HUMAN; MOUSE; NEUROTOXICITY; NONHUMAN; PATHOLOGY; PRIORITY JOURNAL; PROTEIN EXPRESSION; SCHWANN CELL; TRANSGENIC MOUSE;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; DISEASE MODELS, ANIMAL; MICE; MICE, TRANSGENIC; MOTOR NEURONS; SCHWANN CELLS; SPINAL CORD; SUPEROXIDE DISMUTASE;

EID: 84863737109     PISSN: 09699961     EISSN: 1095953X     Source Type: Journal    
DOI: 10.1016/j.nbd.2012.05.014     Document Type: Article

References (30)

1. Audet J.N., et al. Wild-type human SOD1 overexpression does not accelerate motor neuron disease in mice expressing murine Sod1 G86R. Neurobiol. Dis. 2010, 40:245-250.
2. Barber S.C., Shaw P.J. Oxidative stress in ALS: key role in motor neuron injury and therapeutic target. Free Radic. Biol. Med. 2010, 48:629-641.
3. Boillee S., et al. Onset and progression in inherited ALS determined by motor neurons and microglia. Science 2006, 312:1389-1392.
4. Borchelt D.R., et al. Axonal transport of mutant superoxide dismutase 1 and focal axonal abnormalities in the proximal axons of transgenic mice. Neurobiol. Dis. 1998, 5:27-35.
5. Bruijn L.I., et al. ALS-linked SOD1 mutant G85R mediates damage to astrocytes and promotes rapidly progressive disease with SOD1-containing inclusions. Neuron 1997, 18:327-338.
6. Deng H.X., et al. Conversion to the amyotrophic lateral sclerosis phenotype is associated with intermolecular linked insoluble aggregates of SOD1 in mitochondria. Proc. Natl. Acad. Sci. U. S. A. 2006, 103:7142-7147.
7. Feltri M.L., et al. P0-Cre transgenic mice for inactivation of adhesion molecules in Schwann cells. Ann. N. Y. Acad. Sci. 1999, 883:116-123.
8. Feltri M.L., et al. Conditional disruption of beta 1 integrin in Schwann cells impedes interactions with axons. J. Cell Biol. 2002, 156:199-209.
9. Fischer L.R., et al. Amyotrophic lateral sclerosis is a distal axonopathy: evidence in mice and man. Exp. Neurol. 2004, 185:232-240.
10. Fischer L.R., et al. Absence of SOD1 leads to oxidative stress in peripheral nerve and causes a progressive distal motor axonopathy. Exp. Neurol. 2011, 233:163-171.
11. Guo W., et al. An ALS-associated mutation affecting TDP-43 enhances protein aggregation, fibril formation and neurotoxicity. Nat. Struct. Mol. Biol. 2011, 18:822-830.
12. Harraz M.M., et al. SOD1 mutations disrupt redox-sensitive Rac regulation of NADPH oxidase in a familial ALS model. J. Clin. Invest. 2008, 118:659-670.
13. Ilieva H., et al. Non-cell autonomous toxicity in neurodegenerative disorders: ALS and beyond. J. Cell Biol. 2009, 187:761-772.
14. Israelson A., et al. Misfolded mutant SOD1 directly inhibits VDAC1 conductance in a mouse model of inherited ALS. Neuron 2010, 67:575-587.
15. Keller A.F., et al. Live imaging of amyotrophic lateral sclerosis pathogenesis: disease onset is characterized by marked induction of GFAP in Schwann cells. Glia 2009, 57:1130-1142.
16. Lin M.T., Beal M.F. Mitochondrial dysfunction and oxidative stress in neurodegenerative diseases. Nature 2006, 443:787-795.
17. Lobsiger C.S., et al. Schwann cells expressing dismutase active mutant SOD1 unexpectedly slow disease progression in ALS mice. Proc. Natl. Acad. Sci. U. S. A. 2009, 106:4465-4470.
18. Park H.T., Feltri M.L. Rac1 GTPase controls myelination and demyelination. BioArchitecture 2011, 1:110-113.
19. Pun S., et al. Selective vulnerability and pruning of phasic motoneuron axons in motoneuron disease alleviated by CNTF. Nat. Neurosci. 2006, 9:408-419.
20. Rosen D.R., et al. Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis [published erratum appears in Nature 1993 Jul 22;364(6435):362] [see comments]. Nature 1993, 362:59-62.
21. Rothstein J.D. Current hypotheses for the underlying biology of amyotrophic lateral sclerosis. Ann. Neurol. 2009, 65:S3-S9.
22. Simpson E.P., et al. Oxidative Stress: a common denominator in the pathogenesis of amyotrophic lateral sclerosis. Curr. Opin. Rheumatol. 2003, 15:730-736.
23. Turner B.J., et al. Dismutase-competent SOD1 mutant accumulation in myelinating Schwann cells is not detrimental to normal or transgenic ALS model mice. Hum. Mol. Genet. 2010, 19:815-824.
24. Vande Velde C., et al. Misfolded SOD1 associated with motor neuron mitochondria alters mitochondrial shape and distribution prior to clinical onset. PLoS One 2011, 6:e22031.
25. Viader A., et al. Schwann cell mitochondrial metabolism supports long-term axonal survival and peripheral nerve function. J. Neurosci. 2011, 31:10128-10140.
26. Wang L., et al. Wild-type SOD1 overexpression accelerates disease onset of a G85R SOD1 mouse. Hum. Mol. Genet. 2009, 18:1642-1651.
27. Wang L., et al. The effect of mutant SOD1 dismutase activity on non-cell autonomous degeneration in familial amyotrophic lateral sclerosis. Neurobiol. Dis. 2009, 35:234-240.
28. Wang L., et al. Astrocyte loss of mutant SOD1 delays ALS disease onset and progression in G85R transgenic mice. Hum. Mol. Genet. 2011, 20:286-293.
29. Winklhofer K.F., et al. The two faces of protein misfolding: gain- and loss-of-function in neurodegenerative diseases. EMBO J. 2008, 27:336-349.
30. Yamanaka K., et al. Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis. Nat. Neurosci. 2008, 11:251-253.