Studying the role of <i>CASP8</i> GGGAGA repeat expansion in Alzheimer's disease

We successfully developed patient-derived models to investigate the role of the CASP8-GGGAGA-AD-R1 repeat expansion in AD. Through CRISPR/Cas9, we demonstrated efficient excision of the mutation in HEK293 cells. Moving forward, we will apply this system to patient models to assess whether removing the mutation can mitigate disease phenotypes, providing insights into AD mechanisms and potential therapeutic strategies.