This chapter focuses on Alzheimer's disease (AD), the most common age-related dementia, and on the value of experimental models for understanding disease mechanisms and for insights into experimental therapeutics. Characteristic intracellular and extracellular protein aggregates, implicated in pathogenic processes, are critical elements of this pathology. Targeting of genes encoding proteins thought to be implicated in disease pathways has provided new understanding of the roles of specific gene products in AD and the potential of these proteins as therapeutic targets. The value of these targets for new treatment strategies is being tested in model systems and, once safety and efficacy are assured, in human trials. This chapter illustrates the clinical, pathological, biochemical, and genetic features of the human illness. Furthermore, it discusses selected aspects namely, the biology of proteins implicated in pathogenesis of disease, the value of genetically engineered models, the identification of new therapeutic targets, and experimental treatments in models.
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