Advances in the Application of Pluripotent Stem Cells in the Research and Treatment of Neurological Diseases

The development of induced pluripotent stem cell (iPSC) technology has opened new avenues for understanding and treating neurological disorders. iPSCs possess unlimited self-renewal and pluripotent differentiation capabilities, enabling their use in disease modeling, mechanism studies, and high-throughput drug screening. Compared with traditional treatments that mainly alleviate symptoms, iPSC-based approaches offer unique advantages in regenerative medicine by providing patient-specific cell sources for neuronal replacement, modulating immune responses, and promoting endogenous repair. Recent advances have demonstrated the application of iPSC-derived models in major neurological disorders, including Alzheimers disease (AD), Parkinsons disease (PD), and spinal cord injury (SCI). In AD research, iPSC-derived brain organoids have successfully reproduced key pathological hallmarks such as A deposition and Tau hyperphosphorylation, serving as valuable platforms for drug discovery. For PD, iPSC-derived dopaminergic neurons and midbrain-like organoids have revealed critical pathogenic mechanisms and shown potential in transplantation therapy. In SCI, preclinical studies highlight the ability of iPSC-derived neural progenitors and engineered grafts to promote axonal regeneration and functional recovery. This review summarizes current progress, discusses challenges such as tumorigenicity and immune rejection, and explores future strategies to accelerate clinical translation.