Gene Editing in Human Induced Pluripotent Stem Cell Using Prime Editing

The exploration and application of pluripotent stem cells (PSCs) play an indispensable role in stem cell research, human developmental studies, and clinical translation. Particularly since the introduction of induced PSCs (iPSCs) in 2006, the realm of stem cells has been increasingly utilized in areas such as the screening of disease-causing genes, investigation into pathogenic mechanisms, and therapeutic research. However, traditional gene-editing tools, characterized by their limited efficiency, high costs, and poor specificity, have significantly impeded the progression of stem cell applications. The advent of the prime editing (PE) system, heralded as a potent gene-editing tool, has notably bolstered the applicative potential of stem cells. In this chapter, we delve into the utility of PE in spinal muscular atrophy iPSCs as a case study to address its applications and challenges from a methodological perspective.