Drug discovery using induced pluripotent stem cell models of neurodegenerative and ocular diseases

Sandy S.C. Hung, Shahnaz Khan, Camden Y. Lo, Alex W. Hewitt, Raymond C.B. Wong

Research output: Contribution to journalReview ArticleResearchpeer-review

24 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)32-43
Number of pages12
JournalPharmacology and Therapeutics
Publication statusPublished - 1 Sep 2017


  • Disease modeling
  • Drug discovery
  • High throughput screening
  • Induced pluripotent stem cells
  • Organ-on-chip
  • Organoid

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