FURI | Spring 2025

Designing a pegRNA to Examine the Impact of BIN1 Variants in HiPSC-Based Models of Alzheimer’s Disease

Alzheimer’s disease (AD), the most common cause of dementia, was officially listed as the‬‭ sixth-leading cause of death in the United States, with an estimated 6.9 million Americans age‬‭ 65 and older having passed away due to the condition. The BIN1 locus contains the second-most significant genetic risk factor for‬ late-onset AD, which has not been studied extensively on human-induced pluripotent stem cell (hiPSC)‬‭ models. In this study, the use of hiPSC provides a versatile system to study risk modifying BIN1‬ variants. From this project, designed pegRNA can be used in gene therapy to restore wild-type‬‭ BIN1 variant rs744373; thus, decreasing the risk of AD.