Programmed cells from basic neuroscience to therapy

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Recent advances in Programming Somatic Cell (PSC), including induced Pluripotent Stem Cells (iPS) and Induced Neuronal phenotypes (iN), have transformed our experimental landscape, enabling the study of human neuronal function and neurodegenerative and neurodevelopmental diseases in live human neurons. Reprogramming cells from patients with neurological disorders allows researchers to investigate molecular pathways specific to various neuron subtypes, such as dopaminergic neurons in Parkinson’s Disease and motor neurons in Amyotrophic Lateral Sclerosis. Identifying disease-specific molecular signatures in live human brain cells paves the way for early intervention therapies and new diagnostic tools. Once the neurological phenotype is established in vitro, the “disease-in-a-dish” approach facilitates drug screening to alleviate disease-specific conditions. Therapeutic drugs may target generalized pathways or be tailored for individual patients, supporting personalized medicine. However, several challenges must be addressed before PSC technology can be widely applied for clinically relevant modeling of neurological diseases. These include variability in PSC generation methods, individual differences, epigenetic/genetic instability, and the need for disease-relevant neuron subtypes. Current differentiation protocols are evolving, but further development is essential to enhance the generation and monitoring of specific neuron subt