Abstract: Alzheimer's disease (AD) is a complex neurodegenerative disorder characterized by progressive cognitive decline and memory loss, significantly affecting the quality of life for millions of people worldwide. Current therapeutic options primarily focus on symptomatic relief, with limited effectiveness in addressing the underlying pathophysiology. Recent advancements in gene editing technologies, particularly CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats), offer promising opportunities to enhance stem cell therapy for AD. This review explores the potential of gene editing to target genetic risk factors associated with AD, such as APOE4 (Apolipoprotein E) and TREM2 (Triggering Receptor Expressed on Myeloid Cells 2), and to improve the differentiation and functionality of induced pluripotent stem cells (iPSCs) derived from AD patients. By creating more accurate disease models and humanized animal systems, researchers can gain a deeper understanding of the mechanisms of AD and evaluate novel therapeutic strategies. Moreover, combining gene editing with stem cell therapy may facilitate cell replacement therapies and the development of exosome-based treatments that modulate neuroinflammation and promote neuroprotection. Despite the promise of these approaches, challenges, such as off-target effects, delivery efficiency, and ethical considerations, remain significant hurdles. This review aims to provide a comprehensive overview of the current state of research, emphasizing the potential of gene editing technologies to revolutionize stem cell therapy for AD and improve patient outcomes.
Shima Mehrabadi (Tue,) studied this question.