Programmed Cells from Basic Neuroscience to Therapy

摘要

Studies of human brain and neuronal function in phenotypically normal as well as neurological and psychiatric patients have been performed using noninvasive imaging methods. However, the spatial and temporal limitations do not permit single cell/neuron resolution. In addition, genomic and molecular studies of neurological and psychiatric patients are conducted on postmortem tissues often representing the end-stage of life and disease or from peripheral tissues and biopsies, and blood. The recent advances in programming somatic cells (PSC), including induced pluripotent stem cells (iPS) and induced neuronal phenotypes (iN), have changed the experimental landscape and opened new possibilities. These advances have provided an important tool for the study of human neuronal function as well as neurodegenerative and neurodevelopmental diseases in live human neurons in a controlled environment. Researchers are just beginning to take advantage of the many implications of studying developing neurons from living humans in vitro. For example, reprogramming cells from patients with neurological diseases allows the study of molecular pathways particular to specific subtypes of neurons, such as dopaminergic neurons in Parkinson's disease, motor neurons for amyolateral sclerosis, or myelin for multiple sclerosis.