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Critical Reviews™ in Biomedical Engineering
SJR: 0.26 SNIP: 0.375 CiteScore™: 1.4

ISSN Imprimer: 0278-940X
ISSN En ligne: 1943-619X

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Critical Reviews™ in Biomedical Engineering

DOI: 10.1615/CritRevBiomedEng.v37.i4-5.20
pages 321-353

Stem Cell-Based Models and Therapies for Neurodegenerative Diseases

Shilpa Iyer
Department of Neurology, University of Virginia, Charlottesville, Virginia, USA
Khaled Alsayegh
Department of Human and Molecular Genetics, Virginia Commonwealth University, Richmond, Virginia, USA
Sheena Abraham
Department of Chemical & Life Science Engineering, Virginia Commonwealth University, Richmond, Virginia,USA
Raj R. Rao
Department of Human and Molecular Genetics, and Department of Chemical & Life Science Engineering, Virginia Commonwealth University, USA


Multiple neurodegenerative disorders typically result from irrevocable damage and improper functioning of specialized neuronal cells or populations of neuronal cells. These disorders have the potential to contribute to an already overburdened health care system unless the progression of neurodegeneration can be altered. Progress in understanding neurodegenerative cell biology has been hampered by a lack of predictive and, some would claim, relevant cellular models. Additionally, the research needed to develop new drugs and determine methods for repair or replacement of damaged neurons is severely hampered by the lack of an adequate in vitro human neuron cell-based model. In this context, pluripotent stem cells and neural progenitors and their properties—including unlimited proliferation, plasticity to generate other cell types, and a readily available source of cells—pose an excellent alternative to ex vivo primary cultures or established immortalized cell lines in contributing to our understanding of neurodegenerative cell biology and our ability to analyze the therapeutic or cytotoxic effects of chemicals, drugs, and xenobiotics. Many questions that define the underlying "genesis" of the neuronal death in these disorders also remain unanswered, with evidence suggesting a key role for mitochondrial dysfunction. The assessment of stem cells, neural progenitors, and engineered adult cells can provide useful insights into neuronal development and neurodegenerative processes. Finally, the potential for a combination of cell- and gene-based therapeutics for neurodegenerative disorders is also discussed.

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