Hereditary Disease Foundation
New Therapies: Screening in Animal Models
March 28, 1999. New York, New York
Report Prepared by Ai Yamamoto
Abstract
Since the publication of the first transgenic model of Huntington's Disease (HD) by Gillian Bates and colleagues1, a flurry of work has led to the creation of a second generation of models to better understand the disease. These numerous mouse models, together with in vitro systems, provide an excellent opportunity to study possible therapeutic agents. Before this can be done, however, it is necessary to establish the phenotypes that not only simulate HD, but can be most efficiently studied. Furthermore, the differences and similarities between all of the models must be ascertained, for it is still unclear which, if any of the models, truly represent HD.
In the meeting "New Therapies: Screening in Animal Models" held on March 28, 1999, laboratories that have generated the different mouse models, statisticians and experts in clinical trials were brought together to determine how most efficiently to begin drug screening. Each mouse model, as well as a primate model, was discussed at length and the following issues were addressed:
1. Which compounds should be given priority to be screened, and how should we determine which compound is worth screening?
2. How should the screens be designed such that not only is the greatest amount of information extracted, but the different models can be directly compared?
3. Should the screening between the various groups be formally organized?
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