HD research: Treatment & prevention
A biochemical approach to Huntington's disease
Erich E. Wanker, Ph.D Max-Planck Institute for Molecular Genetics, Berlin
"We are conducting a search for chemical compounds that inhibit or slow the formation of polyglutamine-containing aggregates. To do this, we developed a simple and sensitive membrane filter assay.
The aggregates are insoluble and are trapped by a filter membrane, while non-aggregated (normal) huntingtin proteins pass through the filter. We have automated the membrane filter assay to screen, in a reasonable time, large chemical libraries (1,500 different chemicals) for compounds that will inhibit huntingtin aggregation in the test tube. Our experimental work in combination with the theroretical studies should result in the isolation of chemical aggregation inhibitors, which can then be tested in mammalian cells (cell models), in a Drosophila melanogaster (fly) model and in HD transgenic mice.
Furthermore, we suggest that this project will provide valuable information for the design of therapeutics for HD and other polyglutamine disorders".
- This article is reprinted from HDSA's research newsletter, Toward a Cure.
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