title: Induced Pluripotent Stem Cells for Disease Modeling and Drug Discovery in Neurodegenerative Diseases.
abstract: Although most neurodegenerative diseases have been closely related to aberrant accumulation of aggregation-prone proteins in neurons, understanding their pathogenesis remains incomplete, and there is no treatment to delay the onset or slow the progression of many neurodegenerative diseases. The availability of induced pluripotent stem cells (iPSCs) in recapitulating the phenotypes of several late-onset neurodegenerative diseases marks the new era in in vitro modeling. The iPSC collection represents a unique and well-characterized resource to elucidate disease mechanisms in these diseases and provides a novel human stem cell platform for screening new candidate therapeutics. Modeling human diseases using iPSCs has created novel opportunities for both mechanistic studies as well as for the discovery of new disease therapies. In this review, we introduce iPSC-based disease modeling in neurodegenerative diseases, such as Alzheimer's disease, Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis. In addition, we discuss the implementation of iPSCs in drug discovery associated with some new techniques.
authors.affiliation: Department of Neurology, Qingdao Municipal Hospital, Nanjing Medical University, Nanjing, China.
authors.forename: Lei
authors.initials: L
authors.lastname: Cao
authors.forename: Lan
authors.initials: L
authors.lastname: Tan
authors.forename: Teng
authors.initials: T
authors.lastname: Jiang
authors.forename: Xi-Chen
authors.initials: XC
authors.lastname: Zhu
authors.forename: Jin-Tai
authors.initials: JT
authors.lastname: Yu
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D055808:Drug Discovery; D023281:Genomics; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology*; D058990:Molecular Targeted Therapy; D019636:Neurodegenerative Diseases / Q000628:therapy*