Article
Disruption and therapeutic rescue of autophagy in a human neuronal model of Niemann Pick type C1
21 Mar 2012
Abstract excerpt
An unresolved issue about many neurodegenerative diseases is why neurons are particularly sensitive to defects in ubiquitous cellular processes. One example is Niemann Pick type C1, caused by defects in cholesterol trafficking in all cells, but where neurons are preferentially damaged. Understanding this selective failure is limited by the difficulty in obtaining live human neurons from affected patients. To...
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