A screen to identify cellular modulators of soluble levels of an amyotrophic lateral sclerosis (ALS)-causing mutant SOD1

Journal of Biomolecular Screening

Volumn 16, Issue 9, 2011, Pages 974-985

  Somalinga, Balajee R ^a   Miller, Gregory A ^b   Malik, Hiba T ^a   Wigley, W Christian ^b   Thomas, Philip J ^a  

^a UNIVERSITY OF TEXAS AT DALLAS   (United States)

^b Reata Pharmaceuticals Inc   (United States)

Author keywords

amyotrophic lateral sclerosis; cDNA expression cloning screen; protein solubility assay; superoxide dismutase 1

Indexed keywords

COMPLEMENTARY DNA; GENE PRODUCT; SUPEROXIDE DISMUTASE;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMAL TISSUE; ARTICLE; CELL LEVEL; CLONE; CONTROLLED STUDY; FLUORESCENCE ACTIVATED CELL SORTER; HIGH THROUGHPUT SCREENING; HUMAN; HUMAN CELL; MAMMAL CELL; MOUSE; MUTANT; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN FOLDING; SOLUBILITY; SPINAL CORD; VALIDATION PROCESS;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; CELL LINE; GENE EXPRESSION REGULATION; GENE LIBRARY; GENES, REPORTER; HEK293 CELLS; HELA CELLS; HIGH-THROUGHPUT SCREENING ASSAYS; HUMANS; MICE; MICE, INBRED C57BL; MUTATION; ORGAN SPECIFICITY; PROMOTER REGIONS, GENETIC; REPRODUCIBILITY OF RESULTS; SOLUBILITY; SUPEROXIDE DISMUTASE;

MAMMALIA;

EID: 80053540457     PISSN: 10870571     EISSN: 1552454X     Source Type: Journal    
DOI: 10.1177/1087057111418505     Document Type: Article

References (43)

1. Anfinsen C. B. Principles That Govern the Folding of Protein Chains. Science. 1973 ; 181: 223-230
2. Dobson C. M. Protein Folding and Misfolding. Nature. 2003 ; 426: 884-890 (Pubitemid 38056880)
3. Thomas P. J., Qu B. H., Pedersen P. L. Defective Protein Folding as a Basis of Human Disease. Trends Biochem. Sci. 1995 ; 20: 456-459
4. Cheng S. H., Gregory R. J., Marshall J., Paul S., Souza D. W., White G. A., O'Riordan C. R., Smith A. E. Defective Intracellular Transport and Processing of CFTR Is the Molecular Basis of Most Cystic Fibrosis. Cell. 1990 ; 63: 827-834 (Pubitemid 120035055)
5. Lomas D. A., Evans D. L., Finch J. T., Carrell R. W. The Mechanism of Z Alpha 1-Antitrypsin Accumulation in the Liver. Nature. 1992 ; 357: 605-607
6. Hardy J., Selkoe D. J. The Amyloid Hypothesis of Alzheimer's Disease: Progress and Problems on the Road to Therapeutics. Science. 2002 ; 297: 353-356 (Pubitemid 34790756)
7. Polymeropoulos M. H., Lavedan C., Leroy E., Ide S. E., Dehejia A., Dutra A., Pike B., Root H., Rubenstein J., Boyer R., et al. Mutation in the Alpha-Synuclein Gene Identified in Families with Parkinson's Disease. Science. 1997 ; 276: 2045-2047 (Pubitemid 27443610)
8. Spillantini M. G., Crowther R. A., Jakes R., Hasegawa M., Goedert M. Alpha-Synuclein in Filamentous Inclusions of Lewy Bodies from Parkinson's Disease and Dementia with Lewy Bodies. Proc. Natl. Acad. Sci. U. S. A. 1998 ; 95: 6469-6473 (Pubitemid 28249037)
9. Waldo G. S., Standish B. M., Berendzen J., Terwilliger T. C. Rapid Protein-Folding Assay Using Green Fluorescent Protein. Nat. Biotechnol. 1999 ; 17: 691-695 (Pubitemid 29316148)
10. Cabantous S., Rogers Y., Terwilliger T. C., Waldo G. S. New Molecular Reporters for Rapid Protein Folding Assays. PLoS One. 2008 ; 3: e2387
11. Wigley W. C., Stidham R. D., Smith N. M., Hunt J. F., Thomas P. J. Protein Solubility and Folding Monitored In Vivo by Structural Complementation of a Genetic Marker Protein. Nat. Biotechnol. 2001 ; 19: 131-136 (Pubitemid 32144440)
12. Stidham R. D., Wigley W. C., Hunt J. F., Thomas P. J. Assessment of Protein Folding/Solubility in Live Cells. Methods Mol. Biol. 2003 ; 205: 155-169
13. Galarneau A., Primeau M., Trudeau L. E., Michnick S. W. Beta-Lactamase Protein Fragment Complementation Assays as In Vivo and In Vitro Sensors of Protein Protein Interactions. Nat. Biotechnol. 2002 ; 20: 619-622 (Pubitemid 34595158)
14. Chun W., Waldo G. S., Johnson G. V. Split GFP Complementation Assay: A Novel Approach to Quantitatively Measure Aggregation of tau In Situ: Effects of GSK3beta Activation and Caspase 3 Cleavage. J. Neurochem. 2007 ; 103: 2529-2539 (Pubitemid 350173576)
15. Murakami G., Inoue H., Tsukita K., Asai Y., Amagai Y., Aiba K., Shimogawa H., Uesugi M., Nakatsuji N., Takahashi R. Chemical Library Screening Identifies a Small Molecule That Downregulates SOD1 Transcription for Drugs to Treat Amyotrophic Lateral Sclerosis. J. Biomol. Screen. 2011 ; 16: 405-414
16. Broom W. J., Auwarter K. E., Ni J., Russel D. E., Yeh L. A., Maxwell M. M., Glicksman M., Kazantsev A. G., Brown R. H. Two Approaches to Drug Discovery in SOD1-Mediated ALS. J. Biomol. Screen. 2006 ; 11: 729-735 (Pubitemid 44562313)
17. Dangond F., Hwang D., Camelo S., Pasinelli P., Frosch M. P., Stephanopoulos G., Brown R. H., Gullans S. R. Molecular Signature of Late-Stage Human ALS Revealed by Expression Profiling of Postmortem Spinal Cord Gray Matter. Physiol. Genomics. 2004 ; 16: 229-239 (Pubitemid 38420040)
18. Ferraiuolo L., Heath P. R., Holden H., Kasher P., Kirby J., Shaw P. J. Microarray Analysis of the Cellular Pathways Involved in the Adaptation to and Progression of Motor Neuron Injury in the SOD1 G93A Mouse Model of Familial ALS. J. Neurosci. 2007 ; 27: 9201-9219 (Pubitemid 47312070)
19. Kirby J., Halligan E., Baptista M. J., Allen S., Heath P. R., Holden H., Barber S. C., Loynes C. A., Wood-Allum C. A., Lunec J., et al. Mutant SOD1 Alters the Motor Neuronal Transcriptome: Implications for Familial ALS. Brain. 2005 ; 128: 1686-1706 (Pubitemid 41373687)
20. Rosen D. R. Mutations in Cu/Zn Superoxide Dismutase Gene Are Associated with Familial Amyotrophic Lateral Sclerosis. Nature. 1993 ; 364: 362 (Pubitemid 23265284)
21. McCord J. M., Fridovich I. Superoxide Dismutase: An Enzymic Function for Erythrocuprein (Hemocuprein). J. Biol. Chem. 1969 ; 244: 6049-6055
22. Lindberg M. J., Tibell L., Oliveberg M. Common Denominator of Cu/Zn Superoxide Dismutase Mutants Associated with Amyotrophic Lateral Sclerosis: Decreased Stability of the apo State. Proc. Natl. Acad. Sci. U. S. A. 2002 ; 99: 16607-16612 (Pubitemid 36034020)
23. Bruijn L. I., Houseweart M. K., Kato S., Anderson K. L., Anderson S. D., Ohama E., Reaume A. G., Scott R. W., Cleveland D. W. Aggregation and Motor Neuron Toxicity of an ALS-Linked SOD1 Mutant Independent from Wild-Type SOD1. Science. 1998 ; 281: 1851-1854 (Pubitemid 28450505)
24. Rowland L. P., Shneider N. A. Amyotrophic Lateral Sclerosis. N. Engl. J. Med. 2001 ; 344: 1688-1700 (Pubitemid 32479968)
25. Reaume A. G., Elliott J. L., Hoffman E. K., Kowall N. W., Ferrante R. J., Siwek D. F., Wilcox H. M., Flood D. G., Beal M. F., Brown R. H., et al. Motor Neurons in Cu/Zn Superoxide Dismutase-Deficient Mice Develop Normally but Exhibit Enhanced Cell Death after Axonal Injury. Nat. Genet. 1996 ; 13: 43-47 (Pubitemid 26139292)
26. Cleveland D. W. From Charcot to SOD1: Mechanisms of Selective Motor Neuron Death in ALS. Neuron. 1999 ; 24: 515-520
27. Broom W. J., Greenway M., Sadri-Vakili G., Russ C., Auwarter K. E., Glajch K. E., Dupre N., Swingler R. J., Purcell S., Hayward C., et al. 50bp Deletion in the Promoter for Superoxide Dismutase 1 (SOD1) Reduces SOD1 Expression In Vitro and May Correlate with Increased Age of Onset of Sporadic Amyotrophic Lateral Sclerosis. Amyotroph. Lateral Scler. 2008 ; 9: 229-237
28. Zhang J. H., Chung T. D., Oldenburg K. R. A Simple Statistical Parameter for Use in Evaluation and Validation of High Throughput Screening Assays. J. Biomol. Screen. 1999 ; 4: 67-73 (Pubitemid 29278954)
29. Hampel F. R. The Influence Curve and Its Role in Robust Estimation. J. Am. Stat. Assoc. 1974 ; 69: 383-393
30. KambujP. A.Engineering High-Throughput Protein Stability Assays for Drug Discovery. M.S. thesis, University of Texas at Arlington, 2003.
31. Kabuta T., Suzuki Y., Wada K. Degradation of Amyotrophic Lateral Sclerosis-Linked Mutant Cu,Zn-Superoxide Dismutase Proteins by Macroautophagy and the Proteasome. J. Biol. Chem. 2006 ; 281: 30524-30533 (Pubitemid 44582109)
32. Gray P. A., Fu H., Luo P., Zhao Q., Yu J., Ferrari A., Tenzen T., Yuk D. I., Tsung E. F., Cai Z., et al. Mouse Brain Organization Revealed through Direct Genome-Scale TF Expression Analysis. Science. 2004 ; 306: 2255-2257 (Pubitemid 40024462)
33. Altschul S. F., Gish W., Miller W., Myers E. W., Lipman D. J. Basic Local Alignment Search Tool. J. Mol. Biol. 1990 ; 215: 403-410
34. Ullmann A., Jacob F., Monod J. Characterization by In Vitro Complementation of a Peptide Corresponding to an Operator-Proximal Segment of the Beta-Galactosidase Structural Gene of Escherichia coli. J. Mol. Biol. 1967 ; 24: 339-343
35. Forlani G., Baldassa S., Lavagni P., Sturani E., Zippel R. The Guanine Nucleotide Exchange Factor RasGRF1 Directly Binds Microtubules via DHPH2-Mediated Interaction. FEBS J. 2006 ; 273: 2127-2138
36. Binet S., Meininger V. Modifications of Microtubule Proteins in ALS Nerve Precede Detectable Histologic and Ultrastructural Changes. Neurology. 1988 ; 38: 1596-1600
37. Lin H., Zhai J., Schlaepfer W. W. RNA-Binding Protein Is Involved in Aggregation of Light Neurofilament Protein and Is Implicated in the Pathogenesis of Motor Neuron Degeneration. Hum. Mol. Genet. 2005 ; 14: 3643-3659 (Pubitemid 41754665)
38. Topp J. D., Gray N. W., Gerard R. D., Horazdovsky B. F. Alsin Is a Rab5 and Rac1 Guanine Nucleotide Exchange Factor. J. Biol. Chem. 2004 ; 279: 24612-24623 (Pubitemid 38725328)
39. Buratti E., Dork T., Zuccato E., Pagani F., Romano M., Baralle F. E. Nuclear Factor TDP-43 and SR Proteins Promote In Vitro and In Vivo CFTR Exon 9 Skipping. EMBO J. 2001 ; 20: 1774-1784 (Pubitemid 32299413)
40. Kwong L. K., Uryu K., Trojanowski J. Q., Lee V. M. TDP-43 Proteinopathies: Neurodegenerative Protein Misfolding Diseases without Amyloidosis. Neurosignals. 2008 ; 16: 41-51 (Pubitemid 350308320)
41. Sreedharan J., Blair I. P., Tripathi V. B., Hu X., Vance C., Rogelj B., Ackerley S., Durnall J. C., Williams K. L., Buratti E., et al. TDP-43 Mutations in Familial and Sporadic Amyotrophic Lateral Sclerosis. Science. 2008 ; 319: 1668-1672 (Pubitemid 351432505)
42. Murakami G., Inoue H., Tsukita K., Asai Y., Amagai Y., Aiba K., Shimogawa H., Uesugi M., Nakatsuji N., Takahashi R. Chemical Library Screening Identifies a Small Molecule That Downregulates SOD1 Transcription for Drugs to Treat Amyotrophic Lateral Sclerosis. J. Biomol. Screen. 2011 ; 16: 405-414
43. Sebolt-Leopold J. S. Development of Anticancer Drugs Targeting the MAP Kinase Pathway. Oncogene. 2000 ; 19: 6594-6599 (Pubitemid 32197697)