SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice

Annals of Clinical and Translational Neurology

Volumn 2, Issue 2, 2015, Pages 167-184

  Dirren, Elisabeth ^a   Aebischer, Julianne ^a   Rochat, Cylia ^a   Towne, Christopher ^a   Schneider, Bernard L ^a   Aebischer, Patrick ^a  

^a EPFL   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

COPPER ZINC SUPEROXIDE DISMUTASE; MUSCLE PROTEIN;

ACTION POTENTIAL; AMPLITUDE MODULATION; AMYOTROPHIC LATERAL SCLEROSIS; ANALYSIS OF VARIANCE; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASTROCYTE; BEHAVIOR CHANGE; CONTROLLED STUDY; ELECTROMYOGRAPHY; ELECTROPHYSIOLOGICAL PROCEDURES; EVALUATION AND FOLLOW UP; FEMALE; FLUORESCENCE; GASTROCNEMIUS MUSCLE; GENE MUTATION; GENE OVEREXPRESSION; GENE SILENCING; GENE THERAPY; GENETIC TRANSFECTION; GLIA CELL; HISTOLOGY; HUMAN; HUMAN CELL; IMMOBILIZATION; INFLAMMATION; MICROGLIA; MICROSCOPY; MOTONEURON; MOTOR PERFORMANCE; MOUSE; MSOD1 MOUSE; MUSCLE INNERVATION; NEUROMUSCULAR FUNCTION; NEUROMUSCULAR JUNCTION; NONHUMAN; PLASMID; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; PROTEIN EXPRESSION; RNA INTERFERENCE; ROTAROD TEST; SEQUENCE ANALYSIS; SWIMMING; WESTERN BLOTTING;

EID: 84969438526     PISSN: None     EISSN: 23289503     Source Type: Journal    
DOI: 10.1002/acn3.162     Document Type: Article

References (21)

1. Rosen DR, Siddique T, Patterson D, et al. Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis. Nature 1993;362:59–62.
2. Al-Chalabi A, Jones A, Troakes C, et al. The genetics and neuropathology of amyotrophic lateral sclerosis. Acta Neuropathol 2012;124:339–352.
3. Rothstein JD. Current hypotheses for the underlying biology of amyotrophic lateral sclerosis. Ann Neurol 2009;65(suppl 1):S3–S9.
4. Gurney ME, Pu H, Chiu AY, et al. Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation. Science 1994;264:1772–1775.
5. Raoul C, Abbas-Terki T, Bensadoun JC, et al. Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat Med 2005;11:423–428.
6. Ralph GS, Radcliffe PA, Day DM, et al. Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat Med 2005;11:429–433.
7. Foust KD, Salazar DL, Likhite S, et al. Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Mol Ther 2013;21:2148–2159.
8. Lasiene J, Yamanaka K. Glial cells in amyotrophic lateral sclerosis. Neurol Res Int 2011;2011:718987.
9. Yamanaka K, Chun SJ, Boillee S, et al. Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis. Nat Neurosci 2008;11:251–253.
10. Wang L, Gutmann DH, Roos RP. Astrocyte loss of mutant SOD1 delays ALS disease onset and progression in G85R transgenic mice. Hum Mol Genet 2011;20:286–293.
11. Lepore AC, Rauck B, Dejea C, et al. Focal transplantation-based astrocyte replacement is neuroprotective in a model of motor neuron disease. Nat Neurosci 2008;11:1294–1301.
12. Boucherie C, Schafer S, Lavand'homme P, et al. Chimerization of astroglial population in the lumbar spinal cord after mesenchymal stem cell transplantation prolongs survival in a rat model of amyotrophic lateral sclerosis. J Neurosci Res 2009;87:2034–2046.
13. Dirren E, Towne CL, Setola V, et al. Intracerebroventricular injection of adeno-associated virus 6 and 9 vectors for cell type-specific transgene expression in the spinal cord. Hum Gene Ther 2014;25:109–120.
14. Scott S, Kranz JE, Cole J, et al. Design, power, and interpretation of studies in the standard murine model of ALS. Amyotroph Lateral Scler 2008;9:4–15.
15. Boillee S, Yamanaka K, Lobsiger CS, et al. Onset and progression in inherited ALS determined by motor neurons and microglia. Science 2006;312:1389–1392.
16. Pun S, Santos AF, Saxena S, et al. Selective vulnerability and pruning of phasic motoneuron axons in motoneuron disease alleviated by CNTF. Nat Neurosci 2006;9:408–419.
17. Kanning KC, Kaplan A, Henderson CE. Motor neuron diversity in development and disease. Annu Rev Neurosci 2010;33:409–440.
18. Henkel JS, Beers DR, Zhao W, Appel SH. Microglia in ALS: the good, the bad, and the resting. J Neuroimmune Pharmacol 2009;4:389–398.
19. Jaarsma D, Teuling E, Haasdijk ED, et al. Neuron-specific expression of mutant superoxide dismutase is sufficient to induce amyotrophic lateral sclerosis in transgenic mice. J Neurosci 2008;28:2075–2088.
20. Wang H, Yang B, Qiu L, et al. Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis. Hum Mol Genet 2014;23:668–681.
21. Freischmidt A, Muller K, Zondler L, et al. Serum microRNAs in patients with genetic amyotrophic lateral sclerosis and pre-manifest mutation carriers. Brain 2014;137(Pt 11):2938–2950.