2025/06/09 by Moawiah M. Naffaa · 1 voice · 1 citation
Biochemistry, Genetics and Molecular Biology · Engineering · #3D Printing in Biomedical Research #CRISPR and Genetic Engineering #Pluripotent Stem Cells Research
paper · doi:10.4103/regenmed.regenmed-d-25-00013
openalex publication_date 2025/06/09 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/14
Induced pluripotent stem cell technology has significantly advanced regenerative medicine, providing an invaluable platform for modeling neurodevelopmental disorders and facilitating the development of novel therapeutic strategies. This article discusses the potential of induced pluripotent stem cells to unravel the cellular mechanisms underlying neurodevelopmental disorders, which, despite their genetic diversity, share common pathological features. Traditional models have struggled to replicate human-specific phenotypes, whereas induced pluripotent stem cell-based models, including two-dimensional cultures and three-dimensional organoids, offer more accurate representations of neural development and disease. The article explores advances in reprogramming and differentiation protocols that have enabled the generation of patient-specific induced pluripotent stem cell models, while acknowledging the challenges that persist, such as genomic instability and reprogramming inefficiencies. Additionally, the integration of CRISPR/Cas9 gene editing and patient-derived models has led to precision therapies targeting specific genetic mutations, including small molecules, gene editing, and antisense oligonucleotides. By combining in vitro and in vivo approaches, induced pluripotent stem cell-induced pluripotent stem cell models have expanded our understanding of neurodevelopmental disorder mechanisms, including aging, sex differences, and epigenetic regulation. However, challenges in model reproducibility and physiological complexity remain, and the article emphasizes efforts to address these limitations through optimized differentiation protocols, robust quality control, and ethical sourcing. Ultimately, induced pluripotent stem cells hold transformative potential for elucidating neurodevelopmental disorder pathogenesis and advancing personalized therapies for these complex disorders.