iPSを用いた筋萎縮性側索硬化症病態の解析と治療法の開発

抄録

Amyotrophic Lateral Sclerosis (ALS) is a devastating disease characterized by the progressive loss of motor neurons. A number of genes involved in the etiopathology of ALS have been identified, and studies in vivo and in vitro models have clarified the contribution of several genetic and physiological abnormalities to the disease onset and progression.

However, a complete understanding of the ALS molecular mechanisms has yet to be determined. In addition, no cure or effective treatment have been found for ALS.

In the 2006, in vitro technologies to model neurological disorders have undergone impressive developments. The development of induced pluripotent stem cells (iPSCs) enable researchers to make the patient–derived ALS model in vitro, which can reproduce the whole ALS pathology in a dish from onset to death. These iPSC–derived ALS models can also be a powerful and versatile tool for finding ALS therapeutic agents as well as basic research of ALS pathology.

Here, we will discuss the efforts so far to create iPSC–dependent, ALS patient–specific disease models. Furthermore, we will give an overview of how human iPSC–based in vitro models have been established and used, what discoveries they have led to, what outcomes they have returned to society/ALS–patient, and how the recent advances in iPSC technology would expand the field of ALS study.