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Reference Report
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| Citation | Apostol, B.L., Kazantsev, A., Raffioni, S., Illes, K., Pallos, J., Bodai, L., Slepko, N., Bear, J.E., Gertler, F.B., Hersch, S., Housman, D.E., Marsh, J.L., Thompson, L.M. (2003). A cell-based assay for aggregation inhibitors as therapeutics of polyglutamine-repeat disease and validation in Drosophila. Proc. Natl. Acad. Sci. U.S.A. 100(10): 5950--5955. (Export to RIS) | ||
| FlyBase ID | FBrf0159306 | ||
| Publication Type | Research paper | ||
| PubMed ID | 12730384 | ||
| PubMed Abstract | The formation of polyglutamine-containing aggregates and inclusions are hallmarks of pathogenesis in Huntington's disease that can be recapitulated in model systems. Although the contribution of inclusions to pathogenesis is unclear, cell-based assays can be used to screen for chemical compounds that affect aggregation and may provide therapeutic benefit. We have developed inducible PC12 cell-culture models to screen for loss of visible aggregates. To test the validity of this approach, compounds that inhibit aggregation in the PC12 cell-based screen were tested in a Drosophila model of polyglutamine-repeat disease. The disruption of aggregation in PC12 cells strongly correlates with suppression of neuronal degeneration in Drosophila. Thus, the engineered PC12 cells coupled with the Drosophila model provide a rapid and effective method to screen and validate compounds. | ||
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| Language of Publication | English | ||
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| Publication Type | Journal | ||
| Abbreviation | Proc. Natl. Acad. Sci. U.S.A. | ||
| Title | Proceedings of the National Academy of Sciences of the United States of America | ||
| Publication Year | 1915- | ||
| ISBN/ISSN | 0027-8424 | ||
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Data from Reference
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| Genes (3)
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