期刊
BIOCHEMICAL SOCIETY TRANSACTIONS
卷 42, 期 -, 页码 1270-1274出版社
PORTLAND PRESS LTD
DOI: 10.1042/BST20140168
关键词
amyotrophic lateral sclerosis; drug screening; induced pluripotent stem cell; induced neural progenitor; non-neuronal cell
资金
- International Outgoing Marie Curie Fellowship
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder affecting the motor nerves. At present, there is no effective therapy for this devastating disease and only one Food and Drug Administration (FDA)-approved drug, riluzole, is known to moderately extend survival. In the last decade, the field of ALS has made a remarkable leap forward in understanding some of the genetic causes of this disease and the role that different cell types play in the degenerative mechanism affecting motor neurons. In particular, astrocytes have been implicated in disease progression, and multiple studies suggest that these cells are valuable therapeutic targets. Recent technological advancements have provided new tools to generate astrocytes from ALS patients either from post-mortem biopsies or from skin fibroblasts through genetic reprogramming. The advent of induced pluripotent stem cell (iPSC) technology and the newly developed induced neural progenitor cells (iNPCs) have created unprecedented exciting opportunities to unravel the mechanisms involved in neurodegeneration and initiate high-throughput drug screenings.
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