title: Drug discovery using induced pluripotent stem cell models of neurodegenerative and ocular diseases.
abstract: The revolution of induced pluripotent stem cell (iPSC) technology provides a platform for development of cell therapy, disease modeling and drug discovery. Recent technological advances now allow us to reprogram a patient's somatic cells into induced pluripotent stem cells (iPSCs). Together with methods to differentiate these iPSCs into disease-relevant cell types, we are now able to model disease in vitro using iPSCs. Importantly, this represents a robust in vitro platform using patient-specific cells, providing opportunity for personalized precision medicine. Here we provide a review of advances using iPSC for drug development, and discuss the potential and limitations of iPSCs for drug discovery in neurodegenerative and ocular diseases. Emerging technologies that can facilitate the search for new drugs by assessment using in vitro disease models will also be discussed, including organoid differentiation, organ-on-chip, direct reprogramming and humanized animal models.
authors.affiliation: Centre for Eye Research Australia & Ophthalmology, Department of Surgery, University of Melbourne, Australia.
authors.forename: Sandy S C
authors.initials: SSC
authors.lastname: Hung
authors.affiliation: Centre for Eye Research Australia & Ophthalmology, Department of Surgery, University of Melbourne, Australia.
authors.forename: Shahnaz
authors.initials: S
authors.lastname: Khan
authors.affiliation: Monash Micro Imaging, Monash University, Australia.
authors.forename: Camden Y
authors.initials: CY
authors.lastname: Lo
authors.affiliation: Centre for Eye Research Australia & Ophthalmology, Department of Surgery, University of Melbourne, Australia; Menzies Institute for Medical Research, School of Medicine, University of Tasmania, Australia.
authors.forename: Alex W
authors.initials: AW
authors.lastname: Hewitt
authors.affiliation: Centre for Eye Research Australia & Ophthalmology, Department of Surgery, University of Melbourne, Australia. Electronic address: wongcb@unimelb.edu.au.
authors.forename: Raymond C B
authors.initials: RCB
authors.lastname: Wong
mesh_terms: D000818:Animals; D055808:Drug Discovery; D005128:Eye Diseases / Q000188:drug therapy; D006801:Humans; D057026:Induced Pluripotent Stem Cells; D019636:Neurodegenerative Diseases / Q000188:drug therapy
keywords: Disease modeling; Drug discovery; High throughput screening; Induced pluripotent stem cells; Organ-on-chip; Organoid