Altered Purkinje cell miRNA expression and SCA1 pathogenesis

Neurobiology of Disease

Volumn 54, Issue , 2013, Pages 456-463

  Rodriguez Lebron, Edgardo ^a   Liu, Gumei ^a,b   Keiser, Megan ^b   Behlke, Mark A ^c   Davidson, Beverly L ^a,b  

^a UNIVERSITY OF IOWA   (United States)

^b UNIVERSITY OF IOWA   (United States)

^c INTEGRATED DNA TECHNOLOGIES   (United States)

Author keywords

AAV; Ataxin 1; Cerebellum; MiR 150; MiRNA; Neurodegeneration; Polyglutamine; RNA interference; Spinocerebellar ataxia; Vegfa

Indexed keywords

ATAXIN 1; MESSENGER RNA; MICRORNA; MICRORNA 150; RGS PROTEIN; RGS8 PROTEIN; UNCLASSIFIED DRUG; VASCULOTROPIN A;

3' UNTRANSLATED REGION; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CEREBELLUM; CONTROLLED STUDY; GENE EXPRESSION; MOUSE; NEUROBLASTOMA CELL; NONHUMAN; PATHOGENESIS; PRIORITY JOURNAL; PROTEIN EXPRESSION; PURKINJE CELL; RNA INTERFERENCE; SPINOCEREBELLAR DEGENERATION; TRANSGENIC MOUSE;

ANIMALS; DISEASE MODELS, ANIMAL; GENE EXPRESSION REGULATION; IMMUNOHISTOCHEMISTRY; IN SITU HYBRIDIZATION; MICE; MICE, TRANSGENIC; MICRORNAS; NERVE TISSUE PROTEINS; NUCLEAR PROTEINS; POLYMERASE CHAIN REACTION; PURKINJE CELLS; RGS PROTEINS; SPINOCEREBELLAR ATAXIAS; VASCULAR ENDOTHELIAL GROWTH FACTOR A;

EID: 84876329320     PISSN: 09699961     EISSN: 1095953X     Source Type: Journal    
DOI: 10.1016/j.nbd.2013.01.019     Document Type: Article

References (39)

1. Allantaz F., et al. Expression profiling of human immune cell subsets identifies miRNA-mRNA regulatory relationships correlated with cell type specific expression. PLoS One 2012, 7:e29979.
2. Banfi S., et al. Identification and characterization of the gene causing type 1 spinocerebellar ataxia. Nat. Genet. 1994, 7:513-520.
3. Barmack N.H., et al. Climbing fibers induce microRNA transcription in cerebellar Purkinje cells. Neuroscience 2010, 171:655-665.
4. Barnes J.A., et al. Abnormalities in the climbing fiber-Purkinje cell circuitry contribute to neuronal dysfunction in ATXN1[82Q] mice. J. Neurosci. 2011, 31:12778-12789.
5. Bilen J., et al. MicroRNA pathways modulate polyglutamine-induced neurodegeneration. Mol. Cell. 2006, 24:157-163.
6. Boudreau R.L., et al. Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo. Mol. Ther. 2009, 17:169-175.
7. Boudreau R.L., et al. Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. Mol. Ther. 2011, 19:2169-2177.
8. Burright E.N., et al. SCA1 transgenic mice: a model for neurodegeneration caused by an expanded CAG trinucleotide repeat. Cell 1995, 82:937-948.
9. Crespo-Barreto J., et al. Partial loss of ataxin-1 function contributes to transcriptional dysregulation in spinocerebellar ataxia type 1 pathogenesis. PLoS Genet. 2010, 6:e1001021.
10. Cvetanovic M., et al. Vascular endothelial growth factor ameliorates the ataxic phenotype in a mouse model of spinocerebellar ataxia type 1. Nat. Med. 2011, 17:1445-1447.
11. Durr A. Autosomal dominant cerebellar ataxias: polyglutamine expansions and beyond. Lancet Neurol. 2010, 9:885-894.
12. Fernandez-Funez P., et al. Identification of genes that modify ataxin-1-induced neurodegeneration. Nature 2000, 408:101-106.
13. Friedman R.C., et al. Most mammalian mRNAs are conserved targets of microRNAs. Genome Res. 2009, 19:92-105.
14. Gatchel J.R., et al. The insulin-like growth factor pathway is altered in spinocerebellar ataxia type 1 and type 7. Proc. Natl. Acad. Sci. U. S. A. 2008, 105:1291-1296.
15. Grimson A., et al. MicroRNA targeting specificity in mammals: determinants beyond seed pairing. Mol. Cell. 2007, 27:91-105.
16. Hua Z., et al. MiRNA-directed regulation of VEGF and other angiogenic factors under hypoxia. PLoS One 2006, 1:e116.
17. Kim V.N., et al. Biogenesis of small RNAs in animals. Nat. Rev. Mol. Cell Biol. 2009, 10:126-139.
18. Lam Y.C., et al. ATAXIN-1 interacts with the repressor Capicua in its native complex to cause SCA1 neuropathology. Cell 2006, 127:1335-1347.
19. Lee Y., et al. miR-19, miR-101 and miR-130 co-regulate ATXN1 levels to potentially modulate SCA1 pathogenesis. Nat. Neurosci. 2008, 11:1137-1139.
20. Lewis B.P., et al. Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets. Cell 2005, 120:15-20.
21. Lim J., et al. Opposing effects of polyglutamine expansion on native protein complexes contribute to SCA1. Nature 2008, 452:713-718.
22. Lin X., et al. Polyglutamine expansion down-regulates specific neuronal genes before pathologic changes in SCA1. Nat. Neurosci. 2000, 3:157-163.
23. Liu N., et al. The microRNA miR-34 modulates ageing and neurodegeneration in Drosophila. Nature 2012, 482:519-523.
24. Matilla-Duenas A., et al. Cellular and molecular pathways triggering neurodegeneration in the spinocerebellar ataxias. Cerebellum 2010, 9:148-166.
25. Orr H.T. Cell biology of spinocerebellar ataxia. J. Cell Biol. 2012, 197:167-177.
26. Orr H.T., Zoghbi H.Y. Trinucleotide repeat disorders. Annu. Rev. Neurosci. 2007, 30:575-621.
27. Packer A.N., et al. The bifunctional microRNA miR-9/miR-9* regulates REST and CoREST and is downregulated in Huntington's disease. J. Neurosci. 2008, 28:14341-14346.
28. Persengiev S., et al. Genome-wide analysis of miRNA expression reveals a potential role for miR-144 in brain aging and spinocerebellar ataxia pathogenesis. Neurobiol. Aging 2011, 32(2316):e17-e27.
29. Qian Z., et al. Climbing fiber activity reduces 14-3-3-theta regulated GABA(A) receptor phosphorylation in cerebellar Purkinje cells. Neuroscience 2012, 201:34-45.
30. Schaefer A., et al. Cerebellar neurodegeneration in the absence of microRNAs. J. Exp. Med. 2007, 204:1553-1558.
31. Seidel K., et al. Brain pathology of spinocerebellar ataxias. Acta Neuropathol. 2012, 124:1-21.
32. Serra H.G., et al. Gene profiling links SCA1 pathophysiology to glutamate signaling in Purkinje cells of transgenic mice. Hum. Mol. Genet. 2004, 13:2535-2543.
33. Serra H.G., et al. RORalpha-mediated Purkinje cell development determines disease severity in adult SCA1 mice. Cell 2006, 127:697-708.
34. Takahashi T., et al. Polyglutamine diseases: where does toxicity come from? What is toxicity? Where are we going?. J. Mol. Cell Biol. 2010, 2:180-191.
35. Tsou W.L., et al. Splice isoform-specific suppression of the Cav2.1 variant underlying spinocerebellar ataxia type 6. Neurobiol. Dis. 2011, 43:533-542.
36. Verbeek D.S., van de Warrenburg B.P. Genetics of the dominant ataxias. Semin. Neurol. 2011, 31:461-469.
37. Xia H., et al. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat. Med. 2004, 10:816-820.
38. Ye W., et al. The effect of central loops in miRNA:MRE duplexes on the efficiency of miRNA-mediated gene regulation. PLoS One 2008, 3:e1719.
39. Zoghbi H.Y., Orr H.T. Pathogenic mechanisms of a polyglutamine-mediated neurodegenerative disease, spinocerebellar ataxia type 1. J. Biol. Chem. 2009, 284:7425-7429.