Human pluripotent stem cell–derived models and drug screening in CNS precision medicine

• Published in: Annals of the New York Academy of Sciences Vol. 1471; no. 1; pp. 18 - 56
• Main Authors: Silva, M. Catarina, Haggarty, Stephen J.
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01.07.2020
• Subjects: Aging; Automation; Biomarkers; Cell culture; Cell differentiation; Cell Differentiation - drug effects; Central nervous system; Central Nervous System - drug effects; Central Nervous System - pathology; Central Nervous System Diseases - drug therapy; Central Nervous System Diseases - genetics; Central Nervous System Diseases - pathology; Drug development; Drug Discovery; Drug Evaluation, Preclinical; Drug metabolism; Drug screening; Etiology; Humans; human‐induced pluripotent stem cells; Induced Pluripotent Stem Cells - drug effects; Inhibitory postsynaptic potentials; Medicine; neurodegenerative disorders; Neurological diseases; Neurons - drug effects; neuroscience; Optimization; Pathophysiology; Phenotypes; Pluripotency; Precision Medicine; psychiatric disorders; screening; Stem cells; Target recognition; Therapeutic applications; Toxicity; Toxicity testing; screening; drug discovery; human-induced pluripotent stem cells; neurodegenerative disorders; neuroscience; psychiatric disorders

Development of effective therapeutics for neurological disorders has historically been challenging partly because of lack of accurate model systems in which to investigate disease etiology and test new therapeutics at the preclinical stage. Human stem cells, particularly patient‐derived induced plur...