Niemann Pick Disease
NPC1-GFP gene expression fixes defects linked to Niemann Pick disease
Published in Gene Therapy Weekly, October 27th, 2005
According to a study from the United States, "Niemann Pick type C (NPC) disease is an autosomal recessive disorder characterized by abnormal cholesterol metabolism and accumulation in lysosomal and endosomal compartments. Although peripheral organs are affected, the progressive neurodegeneration in the brain is typically most deleterious, leading to dystonia, ataxia, seizures, and premature death."
"Although the two genes underlying this disorder in humans and mouse models of the disease have been identified (NPC1 in...
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Source: Gene Therapy Weekly (2005-10-27)
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