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Hereditary Disease Foundation
"Huntingtin Action: Up & Down the Axon"
June 27-28, 1999
Playa del Rey, CA
Report prepared by Tuan Hoang
Abstract
On June 27 and 28, 1999, the Hereditary Disease Foundation sponsored a workshop on Huntington's disease titled "Up and Down the Axon." The goals were to bring together over a dozen scientific investigators with diverse backgrounds and skills and to facilitate the exchange of ideas and perspectives in an open forum. The main topic was: "What are the functions of the normal and mutant Huntingtin proteins in the cell?" To address this question, the workshop began with a discussion of the localization of the normal Huntingtin protein (Htt). Several possible theories on the normal function of the protein were then proposed based on the protein's subcellular localization and association with axonal transport proteins. The localization of the mutant Htt protein in various in vivo and in vitro models was also reviewed. Eventually, the possible mechanism of cell dysfunction and cell death was discussed. Consideration of the seeding of protein aggregates was followed by our focus on new potential techniques such as gene arrays that can be utilized to elucidate the pathway of neuronal failure in HD. The workshop concluded with the participants proposing new directions in research to advance the understanding of the disease and to expedite the development of an effective treatment.
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