期刊
MOLECULAR AND CELLULAR NEUROSCIENCE
卷 64, 期 -, 页码 44-50出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.mcn.2014.12.005
关键词
Spinal muscular atrophy; iPSCs; Cell models; Neuromuscular diseases
资金
- Ministry of Health [GR-2009-1483560, GR-2010-2309463]
- Ministry of Health: FIRB [RBFR08RV86]
- Cariplo grant [2012-0513]
Motor neuron diseases, as the vast majority of neurodegenerative disorders in humans, are incurable conditions that are challenging to study in vitro, owing to the obstacles in obtaining the cell types majorly involved in the pathogenesis. Recent advances in stem cell research, especially in the development of induced pluripotent stem cell (iPSC) technology, have opened up the possibility of generating a substantial amount of disease-specific neuronal cells, including motor neurons and glial cells. The present review analyzes the practical implications of iPSCs, generated from fibroblasts of patients affected by spinal muscular atrophy (SMA), and discusses the challenges in the development and optimization of in vitro disease models. Research on patient-derived disease-specific cells may shed light on the pathological processes behind neuronal dysfunction and death in SMA, thus providing new insights for the development of novel effective therapies. (C) 2014 Elsevier Inc All rights reserved.
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