Alzheimer's Disease: Genome-wide Clues for Novel Therapies

Alzheimer's disease (AD) is strongly influenced by genetic factors such as the APP, PSEN1, PSEN2 and APOE. Since 2005, we have led the Alzheimer's Genome Project, which in 2008, reported novel AD genes including CD33, one of several genes involved in the innate immune system of the brain that have been associated with AD risk. We are characterizing these genes and screening for novel therapies in a three-dimensional human stem cell-derived neural culture system that recapitulates plaque and tangle pathology of AD.The elucidation of the genes and functional variants influencing risk for AD should continue to enhance our understanding of AD etiology and pathogenesis. Ultimately, these genes will be used to predict risk for AD and guide the development of novel therapies for the effective treatment and prevention of this terrible disease.