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Cells 2018, 7(5), 38; https://doi.org/10.3390/cells7050038

Patient-Derived iPSCs and iNs—Shedding New Light on the Cellular Etiology of Neurodegenerative Diseases

1
Department of Biochemistry, Yong Loo Lin School of Medicine, National University of Singapore, Singapore 117597, Singapore
2
NUS Graduate School for Integrative Sciences and Engineering, National University of Singapore, Singapore 117597, Singapore
Received: 20 April 2018 / Revised: 7 May 2018 / Accepted: 7 May 2018 / Published: 8 May 2018
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Abstract

Induced pluripotent stem cells (iPSCs) and induced neuronal (iN) cells are very much touted in terms of their potential promises in therapeutics. However, from a more fundamental perspective, iPSCs and iNs are invaluable tools for the postnatal generation of specific diseased cell types from patients, which may offer insights into disease etiology that are otherwise unobtainable with available animal or human proxies. There are two good recent examples of such important insights with diseased neurons derived via either the iPSC or iN approaches. In one, induced motor neurons (iMNs) derived from iPSCs of Amyotrophic lateral sclerosis/Frontotemporal dementia (ALS/FTD) patients with a C9orf72 repeat expansion revealed a haploinsufficiency of protein function resulting from the intronic expansion and deficiencies in motor neuron vesicular trafficking and lysosomal biogenesis that were not previously obvious in knockout mouse models. In another, striatal medium spinal neurons (MSNs) derived directly from fibroblasts of Huntington’s disease (HD) patients recapitulated age-associated disease signatures of mutant Huntingtin (mHTT) aggregation and neurodegeneration that were not prominent in neurons differentiated indirectly via iPSCs from HD patients. These results attest to the tremendous potential for pathologically accurate and mechanistically revealing disease modelling with advances in the derivation of iPSCs and iNs. View Full-Text
Keywords: induced pluripotent stem cells (iPSCs); induced neuronal (iN) cells; C9ORF72; Huntingtin; amyotrophic lateral sclerosis (ALS); Huntington’s disease; neurodegenerative diseases induced pluripotent stem cells (iPSCs); induced neuronal (iN) cells; C9ORF72; Huntingtin; amyotrophic lateral sclerosis (ALS); Huntington’s disease; neurodegenerative diseases
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).
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Tang, B.L. Patient-Derived iPSCs and iNs—Shedding New Light on the Cellular Etiology of Neurodegenerative Diseases. Cells 2018, 7, 38.

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