3. Disease modeling and drug development

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We have contributed disease modeling and drug development using disease-specific iPSC lines on ring chromosomes (Bershteyn*, Hayashi* et al., Nature 2014: *equally contributed), Fibrodysplasia Ossificans Progressiva (Hayashi et al PNAS 2016; Matsumoto*, Hayashi* et al., OJRD 2013), Long QT syndromes (Spencer et al., Stem Cell Reports 2014). Our team continues to conduct research projects on basic medicine and drug development using disease-specific iPSC lines provided by RIKEN cell bank.

  • We develop the differentiation-induction system toward specific disease-targeted cell types if the induction protocols are not established.
  • We identify the abnormal cellular phenotypes recapitulating the disease using the differentiated cells from iPSCs in vitro, by comparing the results between disease-specific iPSCs and healthy-donor iPSCs (or isogenic control iPSCs)
  • After we establish the assay system described above, we perform the experiments to search for the responsible genes and screening drug candidates.