Icahn School of Medicine at Mount Sinai
New York, New York
1993 Zenith Fellow
Discovering the Genetics of Alzheimer's Disease and Other Dementias
Dr. Alison Goate has played a critical role in our understanding of how genetics influence the development of Alzheimer's and other dementias by identifying new risk genes, understanding how they interact with other genes, and investigating how they perturb cellular systems. Dr. Goate is responsible for identifying the first familial Alzheimer's gene mutations and additional mutations responsible for early-onset Alzheimer's and frontotemporal dementia. One of her ultimate goals has been to identify novel molecular targets that could be effective in changing the progress of Alzheimer's.
By identifying genes that cause dementia or increase risk, biochemical pathways that play an important role in the disease process can be identified and become targets for drug therapies. Her identification of the familial genes in Alzheimer's has led us to much of our understanding of how beta-amyloid is formed and helped pave the way for the development of today's leading drug candidates for treating Alzheimer's disease. Additionally, Dr. Goate's lab has pioneered the use of endophenotypes to link an individual's specific observable traits to natural genetic variations that may increase or decrease risk for Alzheimer's and other dementias. Through these new techniques, Dr. Goate's lab is investigating how gene variations could protect against Alzheimer's. We know not everybody gets this disease, so are there genetic mechanisms that could protect individuals? Her lab is using the advanced genetic methods developed by Dr. Goate to search for and identify these protective gene candidates.
Dr. Goate has nearly 250 publications on Alzheimer's and other dementias, with more than 400 publications in the genetics field. Since her 1993 Zenith Award, she has received over $10 million in National Institutes of Health (NIH) funding related to Alzheimer's and other dementias research. Recently she became a new member of the prestigious National Academy of Medicine, the highest honor in Medicine. She also received the Khalid Iqbal Lifetime Achievement Award in Alzheimer's Disease Research (from the Alzheimer's Association) and was elected to the American Association for the Advancement of Science. Dr. Goate is truly a pioneer and leader in the fields of genetics and neurodegenerative diseases.