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Time for a rare disease Moon Shot?
Why is it so difficult to translate biological discoveries into effective drugs for diseases that are triggered by a single faulty gene? Once upon a time we stamped out smallpox. We went to the Moon...
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Genus envy
In 1997, a breakthrough was made in rare/orphan disease research. An evolutionarily conserved gene called NPC1 was shown to be responsible for Niemann-Pick disease type C, a degenerative lysosomal...
View ArticleA Drosophila model of the Niemann-Pick type C lysosome storage disease:...
Niemann-Pick type C (NPC) disease is a fatal autosomal-recessive neurodegenerative disorder characterized by the inappropriate accumulation of unesterified cholesterol in aberrant organelles. The...
View ArticleNpc1 acting in neurons and glia is essential for the formation and...
Cholesterol availability is rate-limiting for myelination, and prior studies have established the importance of cholesterol synthesis by oligodendrocytes for normal CNS myelination. However, the...
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Off mice and men
Personalized disease models will speed up and open up rare disease drug discovery. Our collective experiment in government-cultivated biomedical research — the National Institutes of Health, or NIH –...
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