Reverse engineering human neurodegenerative disease using pluripotent stem cell technology

Ying Liu, Wenbin Deng

Research output: Contribution to journalReview articlepeer-review

14 Scopus citations


With the technology of reprogramming somatic cells by introducing defined transcription factors that enables the generation of induced pluripotent stem cells(iPSCs) with pluripotency comparable to that of embryonic stem cells (ESCs),it has become possible to use this technology to produce various cell sand tissues that have been difficult to obtain from living bodies.This advancement is bringing for thrapid progress in iPSC-based disease modeling, drugs creening,and regenerative medicine.More and more studies have demonstrated that phenotypes of adult-on set neurodegenerative disorders could be rather faith fully recapitulated in iPSC-derived neural cell cultures.More over,despite the adult- on set nature of the diseases,pathogenicphe notypes and cellular abnormalities of tenexist in early developmental stages,providing new windows of opportunity for understanding mechanisms underlying neurodegenerative disorders and for discovering new medicines. The cell reprogramming technology enable a reverse engineering approach for modeling the cellular degenerative phenotypes of a wide range of human disorders. An excellent example is the study of the human neurodegenerative disease amyotrophic lateral sclerosis (ALS) using iPSCs. ALS is a progressive neurodegenerative disease characterized by the loss of upper and lower motor neurons (MNs), culminating in muscle wasting and death from respiratory failure. The iPSC approach provides innovative cell culture plat- forms to serve as ALS patient-derived model systems. Researchers have converted iPSCs derived from ALS patients into MNs and various types of glial cells, all of which are involved in ALS, to study the disease. The iPSC technology could be used to determine the role ofspecific genetic factors to track down what'swrong in the neurodegenerative disease process in the disease-in-a-dish model. Mean while,parallel experiments of targeting the same specific genes in human ESCs couldal so be performed to control and to complement the iPSC-based approach for ALS disease modeling studies.

Original languageEnglish (US)
Pages (from-to)30-41
Number of pages12
JournalBrain Research
StatePublished - May 1 2016


  • Glia
  • Induced pluripotent stem cells
  • Lou Gehrig disease
  • Motor neurons

ASJC Scopus subject areas

  • Neuroscience(all)
  • Molecular Biology
  • Developmental Biology
  • Clinical Neurology


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