Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications.
Michael Xavier DossAgapios SachinidisPublished in: Cells (2019)
Induced pluripotent stem cell (iPSC)-based disease modelling and the cell replacement therapy approach have proven to be very powerful and instrumental in biomedical research and personalized regenerative medicine as evidenced in the past decade by unraveling novel pathological mechanisms of a multitude of monogenic diseases at the cellular level and the ongoing and emerging clinical trials with iPSC-derived cell products. iPSC-based disease modelling has sparked widespread enthusiasm and has presented an unprecedented opportunity in high throughput drug discovery platforms and safety pharmacology in association with three-dimensional multicellular organoids such as personalized organs-on-chips, gene/base editing, artificial intelligence and high throughput "omics" methodologies. This critical review summarizes the progress made in the past decade with the advent of iPSC discovery in biomedical applications and regenerative medicine with case examples and the current major challenges that need to be addressed to unleash the full potential of iPSCs in clinical settings and pharmacology for more effective and safer regenerative therapy.
Keyphrases
- high throughput
- single cell
- induced pluripotent stem cells
- artificial intelligence
- stem cells
- replacement therapy
- cell therapy
- drug discovery
- clinical trial
- machine learning
- big data
- deep learning
- randomized controlled trial
- mesenchymal stem cells
- high glucose
- dna methylation
- gene expression
- genome wide
- human health
- tissue engineering