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Nature Medicine 10, 675 - 676 (2004)
doi:10.1038/nm0704-675
Brain on steroids resists neurodegeneration
- Mark P. Burns and Karen Duff are at the Nathan Kline Institute, New York University, Orangeburg, New York 10962, USA. e-mail: duff@nkri.rfmh.org
Abstract
A single injection of the neurosteroid allopregnanolone can delay the onset of neurological symptoms, decrease neuronal cell death and double the lifespan of mice with Niemann–Pick type C disease (pages 704–711).
Niemann–Pick type C (NP-C) disease is a fatal, autosomal-recessive cholesterol transport disorder that affects 1:150,000 individuals of all ethnicities. In the cells of these individuals, faulty cholesterol trafficking from the endosomal system leads to the accumulation of gangliosides and unesterified cholesterol in lysosomes and gangliosides in the central nervous system.
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