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Can modifying a gene variation of APOE reduce Alzheimer’s risk?
Claire Clelland, M.D, Ph.D.
University of California, San Francisco
San Francisco, CA - United States
Background
The apolipoprotein E (APOE) gene provides instructions for making ApoE, a protein believed to help carry fats throughout the body. There are several genetic variations of APOE, including APOE-e2, APOE-e3 and APOE-e4. Studies have shown that in some populations, individuals who possess APOE-e4 have an increased risk for developing Alzheimer’s, compared to individuals with other APOE variants, but the mechanisms linking APOE-e4 and Alzheimer’s risk are not yet understood.
Dr. Claire Clelland and colleagues propose that a gene editing system called CRISPR can be used as a novel therapeutic approach to reduce Alzheimer’s risk in individuals with the APOE-e4 variant.
Research Plan
Dr. Clelland and colleagues will use a specialized type of stem cell called induced pluripotent stem cells (iPSCs) that can be “reprogrammed” into any type of cell in the body, including brain cells, and grown in laboratory dishes. They will use iPSCs from individuals with the APOE-e4 variant and test whether their CRISPR technique can successfully remove the variant from iPSCs that have been reprogrammed into different types of cells in the brain, including nerve cells, astrocytes, and microglia.
Impact
If successful, the results may pave the way for future studies of this novel gene therapy in high-risk individuals with the APOE-e4 variant.
The New Investigator Program Award (NIAP) is jointly funded by the Alzheimer's Association and National Alzheimer’s Coordinating Center.
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