Induced Pluripotent Stem Cell Technology: A Paradigm Shift in Medical Science for Drug Screening and Disease Modeling

Curr Med Chem. 2017;24(39):4368-4398. doi: 10.2174/0929867324666170727100508.

Abstract

Background: Induced Pluripotent Stem Cell (IPSC) Technology is the most advanced research as it offers an attractive alternative for establishing patient-specific IPSCs to recapitulate phenotypes of not only monogenic diseases (viz. Thalassaemia, Sickle cell anemia, Haemophilia, Tay-Sachs disease), but also late-onset polygenic diseases (viz. Parkinson's disease, Alzheimer's disease, schizophrenia). Over the hindsight, numerous studies of the past and current scientists have led to the production, maturation and understanding of induced pluripotent stem cell technology and its use in basic and clinical research.

Methods: A systematic search of peer-reviewed scientific literature and clinical trials in public databases were carried out to summarize the evidence on the use of IPSC.

Results: Current review sheds light upon the use of patient-derived iPSC models in drug toxicity, screening and discovery which have been derived after referring to more than 200 articles in literature. Furthermore, their use as disease models was also studied signifying the versatility of iPSC lines.

Conclusion: Through this review, we describe the advent of iPSC technology, where we comprehensively cover the generation of iPSCs and their characterization along with their prospective applications using IPSC banks in disease modeling and drug screening.

Keywords: disease modeling; drug discovery; iPSC; pluripotency; reprogramming; therapy; transcription factors.

Publication types

  • Review

MeSH terms

  • Alzheimer Disease / therapy
  • Anemia, Sickle Cell / therapy
  • Animals
  • Disease Models, Animal*
  • Drug Evaluation, Preclinical
  • Hemophilia A / therapy
  • Humans
  • Induced Pluripotent Stem Cells*
  • Parkinson Disease / therapy
  • Schizophrenia / therapy
  • Stem Cell Transplantation*
  • Tay-Sachs Disease / therapy
  • Thalassemia / therapy