Cystamine and intrabody co-treatment confers additional benefits in a fly model of Huntington's disease

Neurobiology of Disease

Volumn 40, Issue 1, 2010, Pages 130-134

  Bortvedt, S F ^a,b,d   McLear, J A ^b,e   Messer, A ^b,c   Ahern Rindell, A J ^a   Wolfgang, W J ^b,c  

^a UNIVERSITY OF PORTLAND   (United States)

^b WADSWORTH CENTER   (United States)

^c STATE UNIVERSITY OF NEW YORK   (United States)

^d UNIVERSITY OF NORTH CAROLINA   (United States)

^e UTICA COLLEGE   (United States)

Author keywords

Combinatorial therapy; Neurodegenerative disease; Polyglutamine; ScFv

Indexed keywords

C 121509; C4 HUNTINGTIN SINGLE CHAIN FRAGMENT VARIABLE ANTIBODY; CYSTAMINE; HUNTINGTIN; SINGLE CHAIN FRAGMENT VARIABLE ANTIBODY; UNCLASSIFIED DRUG;

ARTICLE; COMBINATION CHEMOTHERAPY; CONTROLLED STUDY; DEVELOPMENTAL STAGE; DROSOPHILA; DRUG EFFICACY; FEEDING; FEMALE; HUNTINGTON CHOREA; LARVAL STAGE; LONGEVITY; NERVE DEGENERATION; NEUROPROTECTION; NONHUMAN; PHOTORECEPTOR; PRIORITY JOURNAL; SURVIVAL RATE; TRANSGENE;

ANIMALS; ANIMALS, GENETICALLY MODIFIED; ANTIBODIES; CYSTAMINE; DISEASE MODELS, ANIMAL; DROSOPHILA MELANOGASTER; DRUG THERAPY, COMBINATION; FEMALE; GENE THERAPY; HUMANS; HUNTINGTON DISEASE; MALE; MODELS, GENETIC; NERVE DEGENERATION; NERVE TISSUE PROTEINS; NUCLEAR PROTEINS; PHOTORECEPTOR CELLS, INVERTEBRATE; SURVIVAL ANALYSIS; TREATMENT OUTCOME;

EID: 77955843583     PISSN: 09699961     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nbd.2010.04.007     Document Type: Article

References (41)

1. Agrawal N., et al. Identification of combinatorial drug regimens for treatment of Huntington's disease using Drosophila. Proc. Natl. Acad. Sci. U. S. A. 2005, 102:3777-3781.
2. Apostol B.L., et al. A cell-based assay for aggregation inhibitors as therapeutics of polyglutamine-repeat disease and validation in Drosophila. Proc. Natl. Acad. Sci. U. S. A. 2003, 100:5950-5955.
3. Arrasate M., et al. Inclusion body formation reduces levels of mutant huntingtin and the risk of neuronal death. Nature 2004, 431:805-810.
4. Bailey C.D., Johnson G.V. Tissue transglutaminase contributes to disease progression in the R6/2 Huntington's disease mouse model via aggregate-independent mechanisms. J. Neurochem. 2005, 92:83-92.
5. Bodner R.A., et al. Pharmacological promotion of inclusion formation: a therapeutic approach for Huntington's and Parkinson's diseases. Proc. Natl. Acad. Sci. U. S. A. 2006, 103:4246-4251.
6. Borrell-Pages M., et al. Cystamine and cysteamine increase brain levels of BDNF in Huntington disease via HSJ1b and transglutaminase. J. Clin. Invest. 2006, 116:1410-1424.
7. Dedeoglu A., et al. Therapeutic effects of cystamine in a murine model of Huntington's disease. J. Neurosci. 2002, 22:8942-8950.
8. Dubinsky R., Gray C. CYTE-I-HD: phase I dose finding and tolerability study of cysteamine (Cystagon) in Huntington's disease. Mov. Disord. 2006, 21:530-533.
9. Fox J.H., et al. Cystamine increases l-cysteine levels in Huntington's disease transgenic mouse brain and in a PC12 model of polyglutamine aggregation. J. Neurochem. 2004, 91:413-422.
10. Gu X., et al. Serines 13 and 16 are critical determinants of full-length human mutant huntingtin induced disease pathogenesis in HD mice. Neuron 2009, 64:828-840.
11. Harper S.Q., et al. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc. Natl. Acad. Sci. U. S. A. 2005, 102:5820-5825.
12. Huntington's Disease Collaborative Research Group A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group Cell 1993, 72:971-983.
13. Karpuj M.V., et al. Prolonged survival and decreased abnormal movements in transgenic model of Huntington disease, with administration of the transglutaminase inhibitor cystamine. Nat. Med. 2002, 8:143-149.
14. Karpuj M.V., et al. Transglutaminase aggregates huntingtin into nonamyloidogenic polymers, and its enzymatic activity increases in Huntington's disease brain nuclei. Proc. Natl. Acad. Sci. U. S. A. 1999, 96:7388-7393.
15. Kazemi-Esfarjani P., Benzer S. Genetic suppression of polyglutamine toxicity in Drosophila. Science 2000, 287:1837-1840.
16. Lecerf J.M., et al. Human single-chain Fv intrabodies counteract in situ huntingtin aggregation in cellular models of Huntington's disease. Proc. Natl. Acad. Sci. U. S. A. 2001, 98:4764-4769.
17. Lesort M., et al. Cystamine inhibits caspase activity Implications for the treatment of polyglutamine disorders. J. Biol. Chem. 2003, 278:3825-3830.
18. Lorand L., Conrad S.M. Transglutaminases. Mol. Cell. Biochem. 1984, 58:9-35.
19. Marsh J.L., et al. Fly models of Huntington's disease. Hum. Mol. Genet. 2003, 12(Spec No 2):R187-R193.
20. Marsh J.L., Thompson L.M. Drosophila in the study of neurodegenerative disease. Neuron 2006, 52:169-178.
21. McLear J.A., et al. Combinational approach of intrabody with enhanced Hsp70 expression addresses multiple pathologies in a fly model of Huntington's disease. FASEB J. 2008, 22:2003-2011.
22. Messer A., et al. Developing intrabodies for the therapeutic suppression of neurodegenerative pathology. Expert Opin. Biol. Ther. 2009, 9:1189-1197.
23. Miller T.W., Messer A. Intrabody applications in neurological disorders: progress and future prospects. Mol. Ther. 2005, 12:394-401.
24. Miller T.W., et al. A human single-chain Fv intrabody preferentially targets amino-terminal Huntingtin's fragments in striatal models of Huntington's disease. Neurobiol. Dis. 2005, 19:47-56.
25. Morton A.J., et al. A combination drug therapy improves cognition and reverses gene expression changes in a mouse model of Huntington's disease. Eur. J. NeuroSci. 2005, 21:855-870.
26. Ross C.A., Poirier M.A. Protein aggregation and neurodegenerative disease. Nat. Med. 2004, (10 Suppl):S10-S17.
27. Ryu J.K., et al. Combined minocycline plus pyruvate treatment enhances effects of each agent to inhibit inflammation, oxidative damage, and neuronal loss in an excitotoxic animal model of Huntington's disease. Neuroscience 2006, 141:1835-1848.
28. Sarkar S., et al. A rational mechanism for combination treatment of Huntington's disease using lithium and rapamycin. Hum. Mol. Genet. 2008, 17:170-178.
29. Schilling G., et al. Coenzyme Q10 and remacemide hydrochloride ameliorate motor deficits in a Huntington's disease transgenic mouse model. Neurosci. Lett. 2001, 315:149-153.
30. Southwell A.L., et al. Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease. J. Neurosci. 2009, 29:13589-13602.
31. Stack E.C., et al. Combination therapy using minocycline and coenzyme Q10 in R6/2 transgenic Huntington's disease mice. Biochim. Biophys. Acta 2006, 1762:373-380.
32. Steffan J.S., et al. Histone deacetylase inhibitors arrest polyglutamine-dependent neurodegeneration in Drosophila. Nature 2001, 413:739-743.
33. Stocks M. Intracellular antibodies: a revolution waiting to happen?. Curr. Opin. Mol. Ther. 2006, 8:17-23.
34. Tam S., et al. The chaperonin TRiC blocks a huntingtin sequence element that promotes the conformational switch to aggregation. Nat. Struct. Mol. Biol. 2009, 16:1279-1285.
35. Thompson L.M., et al. IKK phosphorylates Huntingtin and targets it for degradation by the proteasome and lysosome. J. Cell Biol. 2009, 187:1083-1099.
36. Truant R., et al. Huntington's disease: revisiting the aggregation hypothesis in polyglutamine neurodegenerative diseases. FEBS J. 2008, 275:4252-4262.
37. Van Raamsdonk J.M., et al. Cystamine treatment is neuroprotective in the YAC128 mouse model of Huntington disease. J. Neurochem. 2005, 95:210-220.
38. Wang C.E., et al. Suppression of neuropil aggregates and neurological symptoms by an intracellular antibody implicates the cytoplasmic toxicity of mutant huntingtin. J. Cell Biol. 2008, 181:803-816.
39. Williamson T.E., et al. Modulation of polyglutamine conformations and dimer formation by the N-terminus of huntingtin. J. Mol. Biol. 2010, 396:1295-1309.
40. Wolfgang W.J., et al. Suppression of Huntington's disease pathology in Drosophila by human single-chain Fv antibodies. Proc. Natl. Acad. Sci. U. S. A. 2005, 102:11563-11568.
41. Yang L., et al. Combination therapy with coenzyme Q10 and creatine produces additive neuroprotective effects in models of Parkinson's and Huntington's diseases. J. Neurochem. 2009, 109:1427-1439.