Human stem cells for modeling neurological disorders: accelerating the drug discovery pipeline.
The availability of human stem cells heralds a new era for modeling normal and pathologic tissues and developing therapeutics. For example, the in vitro recapitulation of normal and aberrant neurogenesis holds significant promise as a tool for de novo modeling of neurodevelopmental and neurodegenerative diseases. Translational applications include deciphering brain development, function, pathologies, traditional medications, and drug discovery for novel pharmacotherapeutics. For the latter, human stem cell-based assays represent a physiologically relevant and high-throughput means to assess toxicity and other undesirable effects early in the drug development pipeline, avoiding late-stage attrition whilst expediting proof-of-concept of genuine drug candidates. Here we consider the potential of human embryonic, adult, and induced pluripotent stem cells for studying neurological disorders and preclinical drug development.
Crook
Jeremy Micah
JM
Stem Cell Disease Models Laboratory, Institute of Medical Biology, Singapore 138648, Singapore. jeremy.crook@imb.a-star.edu.sg
Kobayashi
Nao Rei
NR
D000818:Animals; D002454:Cell Differentiation; D055808:Drug Discovery / Q000379:methods; D004353:Drug Evaluation, Preclinical / Q000379:methods; D006801:Humans; D009422:Nervous System Diseases / Q000188:drug therapy*; D055495:Neurogenesis; D039904:Pluripotent Stem Cells / Q000166:cytology / Q000378:metabolism; D044968:Regenerative Medicine / Q000379:methods; D033581:Stem Cell Transplantation; D013234:Stem Cells / Q000166:cytology / Q000378:metabolism*