Human-induced pluripotent stem cells: Potential for neurodegenerative diseases

Research output: Research - peer-reviewArticle

Abstract

The cell biology of human neurodegenerative diseases has been difficult to study till recently. The development of human induced pluripotent stem cell (iPSC) models has greatly enhanced our ability to model disease in human cells. Methods have recently been improved, including increasing reprogramming efficiency, introducing non-viral and non-integrating methods of cell reprogramming, and using novel gene editing techniques for generating genetically corrected lines from patient-derived iPSCs, or for generating mutations in control cell lines. In this review, we highlight accomplishments made using iPSC models to study neurodegenerative disorders such as Huntington's disease, Parkinson's disease, Amyotrophic Lateral Sclerosis, Fronto-Temporal Dementia, Alzheimer's disease, Spinomuscular Atrophy and other polyglutamine diseases. We review disease-related phenotypes shown in patient-derived iPSCs differentiated to relevant neural subtypes, often with stressors or cell "aging", to enhance disease-specific phenotypes. We also discuss prospects for the future of using ofiPSCmodelsofneurodegenerative disorders, includingscreeningandtesting of therapeutic compounds, and possibly of cell transplantation in regenerative medicine. The new iPSC models have the potential to greatly enhance our understanding of pathogenesis and to facilitate the development of novel therapeutics.

LanguageEnglish (US)
JournalHuman Molecular Genetics
Volume23
Issue numberR1
DOIs
StatePublished - 2014

Fingerprint

Induced Pluripotent Stem Cells
Neurodegenerative Diseases
Phenotype
Therapeutics
Aptitude
Regenerative Medicine
Cell Aging
Huntington Disease
Cell Transplantation
Amyotrophic Lateral Sclerosis
Atrophy
Parkinson Disease
Cell Biology
Dementia
Alzheimer Disease
Efficiency
Cell Line
Mutation
Cellular Reprogramming
Gene Editing

ASJC Scopus subject areas

  • Genetics
  • Genetics(clinical)
  • Molecular Biology
  • Medicine(all)

Cite this

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title = "Human-induced pluripotent stem cells: Potential for neurodegenerative diseases",
abstract = "The cell biology of human neurodegenerative diseases has been difficult to study till recently. The development of human induced pluripotent stem cell (iPSC) models has greatly enhanced our ability to model disease in human cells. Methods have recently been improved, including increasing reprogramming efficiency, introducing non-viral and non-integrating methods of cell reprogramming, and using novel gene editing techniques for generating genetically corrected lines from patient-derived iPSCs, or for generating mutations in control cell lines. In this review, we highlight accomplishments made using iPSC models to study neurodegenerative disorders such as Huntington's disease, Parkinson's disease, Amyotrophic Lateral Sclerosis, Fronto-Temporal Dementia, Alzheimer's disease, Spinomuscular Atrophy and other polyglutamine diseases. We review disease-related phenotypes shown in patient-derived iPSCs differentiated to relevant neural subtypes, often with stressors or cell {"}aging{"}, to enhance disease-specific phenotypes. We also discuss prospects for the future of using ofiPSCmodelsofneurodegenerative disorders, includingscreeningandtesting of therapeutic compounds, and possibly of cell transplantation in regenerative medicine. The new iPSC models have the potential to greatly enhance our understanding of pathogenesis and to facilitate the development of novel therapeutics.",
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